A new $200 million collaboration aims to speed development of genetic cures for people in Africa with sickle cell disease (above) and, separately, HIV infection.

By Jon Cohen, Jocelyn KaiserOct. 23, 2019 , 5:00 PM

Two major U.S. biomedical research funders plan to each put at least $100 million over 4 years toward bringing cutting-edge, gene-based treatments to a part of the world that often struggles to provide access to even basic medicines: sub-Saharan Africa. The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation today announced the unusual collaboration to launch clinical trials for gene-based cures for HIV and sickle cell disease within the region in the coming decade.

The ambitious goal is to steer clear of expensive, logistically impractical strategies that require stem cell transplantation, and instead develop simpler, affordable ways of delivering genes or gene-editing drugs that can cure these diseases. Yes, this is audacious, NIH Director Francis Collins said during a press teleconference this morning on the project. But if we dont put our best minds, resources, and visions together right now, we would not live up to our mandate to bring the best science to those who are suffering.

After decades of work and setbacks, the traditional gene therapy approach of delivering DNA into the body to replace a defective gene or boost a proteins production is now reaching the clinic for several diseases, including inherited blindness, neuromuscular disease, and leukemia. Animal studies and some clinical trials have suggested that two diseases prevalent in Africa, HIV and sickle cell disease, can be treated by gene therapies or newer genome-editing tools such as CRISPR.

But in most cases, introducing those therapeutic genes or the components of a genome editor involves removing stem cells from the body, adding or modifying genes, then reinfusing the cells back into the body. That is essentially a stem cell transplant with ones own cells, an expensive procedure that is also typically risky because physicians wipe out most of a patients existing stem cells with chemotherapy so the corrected cells can engraft and grow. It remains out of reach for most people in sub-Saharan Africa, where few places have the medical infrastructure to support such intensive interventions.

Yet sub-Saharan Africa is home to about two-thirds of the 20 million people with sickle cell disease and the 38 million living with HIV. The NIH-Gates partnership is an incredible opportunity to find new therapies and possible cures for two diseases that affect millions of Africans and to make them available at affordable costs, said Matshidiso Moeti, who heads the Regional Office for Africa at the World Health Organization.

Anthony Fauci, director of NIHs National Institute of Allergy and Infectious Diseases, noted that if this collaboration pans out, it could also lead to enormous cost savings. If we do successfully achieve an HIV cure, it will ultimately be important not only for millions of individuals with HIV, but also will save hundreds of billions of dollars in health care costs, said Fauci, whose institute already funds a major HIV cure initiative.

In sickle cell disease, which involves a defect in the oxygen-carrying hemoglobin in red blood cells, several ongoing gene therapy and gene-editing clinical trials in the United States and Europe are either adding a new hemoglobin gene to cells or turning on the gene for a fetal form of the protein. Other clinical trials for HIV have used CRISPR or other genome editors in stem cells to cripple a receptor, CCR5, that the virus depends on to establish infection.

Instead of modifying a persons stem cells and transplanting them back, the new collaboration will seek to ferry a therapeutic gene or gene-editing tools directly into the body (in vivo) with vectorssuch as harmless viruses or nanoparticles, Collins said. The treatment itself would be similar to a simple blood transfusion. Although studies are already underway with viral vectors that deliver new genes to certain tissues in people, in vivo gene therapy has only been used to modify blood stem cells in animal models of certain diseases. Figuring out how to home in on and modify those cells in people is a big part of the collaborations plan, Collins said.

Hematologist Alexis Thompson of the Northwestern University Feinberg School of Medicine in Chicago, Illinois, who is involved with some sickle cell gene therapy trials, calls the NIH-Gates collaboration phenomenal.But, she says, a more urgent need is to expand efforts to screen newborns in Africa for sickle cell disease and treat them with antibiotics; at the moment, the majority die before age 5 from bacterial infections because the sickled cells impair the spleens ability to filter bacteria and make antibodies. Unless more children with sickle cell disease mutations survive longer, there will be few to be cured with the new gene-based treatments, Thompson says. Its almost being able to crawl or walk before you sprint.(Gates and NIH say they plan to support screening efforts outside of the new collaboration.)

For HIV, a big impetus for the cure push builds on two people infected with the AIDS virus who were cured with stem cell transplants. These two men each had blood cancers that required the transplants, which intentionally used blood from donors who had white blood cells with crippled CCR5 receptors. After the transplants, whatever HIV remained in these men could not enter new host cells, and their infections petered out. This new initiative hopes to speed development of direct injections of gene editor components that can target theCCR5gene in blood cells and cripple it. The potential beauty of in vivo gene editing is that it might be given ultimately as a single shot, curing everyone in a scalable manner, says Steven Deeks, a leading HIV cure researcher at the University of California, San Francisco.

The collaboration will also try to speed development of more experimental interventions that directly excise HIVs genetic material from a patients cells or allow people to artificially make superpotent antibodies against the virus. This might be science fiction now, but one day may be a real possibility, Deeks says.

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NIH and Gates Foundation lay out ambitious plan to bring gene-based treatments for HIV and sickle cell disease to Africa - Science Magazine

Let's play a game of word association. I'll go first.

Cell Therapy

What words spring to mind? CRISPR? Medicine? Genetic disorders? Cancer? Gene therapy?

What about green tea? Unlikely, I imagine.

But in a new study published today in Science Advances, researchers from East China Normal University have created an elegant system for activating genetically edited cells using green tea.1Realizing the promise of cell therapiesEngineered cell therapies, deemed the "next frontier" in modern medicine, contain specific cellular material that triggers a desired effect in vitro or in vivo. Such therapies are in development in laboratories across the globe for an array of different conditions, including acute myocardial infarction (heart attack), brain cancer, breast cancer, diabetes and liver diseases. They offer a novel avenue of therapeutics for patients suffering from diseases for which treatment options are limited.For their efficacious and safe use in the clinic, scientists need to be able to regulate the activity of these cells in vivo. Essentially, they require a "remote control". This has proven a major barrier for the delivery of cell therapies to patients. Initial work in this field has adopted antibiotics such as doxycycline or tetracycline as remote-control triggers for gene expression in the cells. However, regular use of antibiotics may result in antibiotic resistance and other adverse side effects.So, what alternatives exist?Haifeng Ye, Professor at East China Normal University, says "Ideal trigger molecules for clinical biomedical applications would be natural, non-toxic, highly soluble, inexpensive, and perhaps even beneficial to health."Previous studies have reported that remote control switches can be activated through the use of food or cosmetic preservatives, vanillic acid, benzoate and phloretin for example. These molecules do not naturally occur in food however, and the safety implications of their long-term use is not well known.A green solution?Nothing beats a good cup of tea. It is the second most popular beverage on the planet (following water) and can be found in the household cupboards of 80% of Americans. Tea is available in a variety of forms, including but not limited to black tea, oolong tea, white tea and green tea. A plethora of research studies have documented the numerous health benefits of green tea consumption, including anticarcinogenic, anti-inflammatory, antimicrobial, and antioxidant effects.The components of green tea most heavily researched with regards to health are the polyphenols, of which the most pertinent are flavonoids, and the most pertinent flavonoids are the catechins.2Post green-tea consumption, the tea catechins and phenolic acids undergo metabolic processing to form the antioxidant protocatechuic acid (PCA). In their latest study, Ye and team have utilized this antioxidant as a "remote control" for activating gene switches in cells. "PCA is a major tea catechin compound produced by humans following green tea consumption that has powerful antioxidant activity. Therefore, in this study, we showed the use of protocatechuic acid (we call it PCA), a metabolite after tea drinking, as a trigger molecule," Ye told Technology Networks.PCA-inducible gene switchesIn the study, the scientists engineered PCA-inducible gene switches in mammalian cells. Initially, they explored the potential for using PCA to monitor cell-based long-term therapies in vivo by integrating the genetic switch into HEK-293 cells and found that the cell line demonstrated reversible and tunable induction kinetics, which the authors regard as "excellent switching performance". This was characterized by negligible basal expression and nonsaturating increases in the transgene output over the course of a 15-day trial.Next, they microencapsulated and implanted the HEK cells into mice. Ye tells us, "The alginate-poly (L-lysine)-alginate-based encapsulation technology was used in our study for cell therapy. This clinically validated implant technology enables the free diffusion of metabolites, nutrients and proteins of lower molecular weights (<72 kDa) across the biocompatible capsule membrane while shielding their cellular content from physical contact with the hosts immune system. The implant technology has been successfully validated in human clinical trials and the performance of the material is continuously improved for clinical applications."The researchers found that, regardless of delivery method (intraperitoneal, oral intake from water, or oral intake from concentrated green tea), PCA could control the secretion of a reporter protein, SEAP, in a dose-dependent manner.Making CRISPR more crisp?CRISPR gene-editing shows promise in revolutionizing personalized medicine. A notable key issue with CRISPR, however, is the "off target" effects that limit its specificity. In this study, the scientists used the PCA-responsive cells to perform more targeted CRISPR gene editing: "By applying newly-designed fusion-protein-based PCA-controlled gene switches to Pol III promoters, we created trigger-inducible expression systems for gRNAs to program PCA-mediated CRISPR/Cas9-activity," says Ye.Exploring diabetes treatment with PCA-induced cell therapyYe and colleagues next tested the potential of the PCA remote control system for treating experimental diabetes using a mouse model. Using the switch, they engineered two different cell lines: one that enabled PCA-inducible expression of the reporter protein SEAP and insulin, and the other producing a short variant of human glucagon-like peptide 1 and SEAP. Implantation of these cells into mouse models of type 1 diabetes and type 2 diabetes mellitus resulted in restored homeostatic fasting blood glucose concentrations and glucose tolerance upon PCA injection.Recognizing that the translation of research findings from mouse models to humans in the clinic can be problematic, the scientists then decided to explore the PCA remote control switch efficacy in non-human primate models of diabetes. In parallel to the treatment efficacy observed in the type 2 diabetic mice, daily oral administration of PCA rapidly increased the expression of glucagon-like peptide 1 and restored glucose homeostasis in diabetic monkeys.In terms of safety, blood biochemical analyses related to inflammatory responses found that white blood cell count, lymphocytes, monocytes, eosinophils, and basophils, did not increase at any point during the treatment when compared with pre-treatment.The study findings certainly excite the authors, "Although there have not yet been preclinical studies for the application of engineered cellbased therapies in humans, this first-in-monkey study demonstrates the feasibility of safely and successfully scaling up a treatment strategy by controlling microencapsulated engineered cells to release therapeutic outputs from animals such as mice to larger NHPs. Therefore, this study substantiates the medical utility of concepts developed in synthetic biology," they note in the discussion of the paper.How much tea is too much tea?Hypothetically, if this therapy was to reach the clinic, I ponder over the possibility of an individual consuming "too much" green tea, and how this might impact the therapy. Ye is quick to inform me that this would not be an issue, "Only custom prepared concentrated green tea can activated the implanted designer cells. The normal green tea drinks cannot activate the implanted cells because of low concentration," he says.The future looks greenThe study is comprehensive, assessing the PCA "switch" in a variety of cell lines and mammalian models with a variety of control measures in place.Thus, in which direction will this research go next? I ask Ye, who tells me, " We will next focus on solving the following limitations:(1) The PCAON-switch was stably integrated into [the] genome by a "Sleeping Beauty" transposon system. Due to a random integration, unwanted insertional mutagenesis might occur. We will next consider using gene editing tools, such as CRISPR, to enable facile and permanent integration of the switch into the targeted genomic sequences in human cells without insertional mutagenesis;(2) The chassis of the HEK-293 cells are easily handled, transfected, and compatible to the PCAON-switch. For translational applications, they must also be safe (no side effects) in humans. Hence, we will test the therapeutic efficiency of the PCAON-switch in autologous parental cells from patients own mesenchymal stem cells, which may provide immunocompatible and noncarcinogenic autologous or allogeneic cell sources;(3) The lifespan of the designer cells inside the alginate microcapsules is an imperative issue. To realize long-term cell therapy, we will further improve the encapsulation technology."Haifeng Ye, Professor at East China Normal University, was speaking with Molly Campbell, Science Writer, Technology Networks.References:1. A green teatriggered genetic control system for treating diabetes in mice and monkeys," by J. Yin; L. Yang; K. Dong; J. Jiang; S. Xue; Y. Xu; X. Wang; H. Ye at East China Normal University in Shanghai, China; L. Mou; Y. Lu at First Affiliated Hospital of Shenzhen University in Shenzhen, China.2. Reygaert. 2018. Green Tea Catechins: Their Use in Treating and Preventing Infectious Diseases. Biomed Research International. doi: 10.1155/2018/9105261.

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Green Tea Acts as a "Remote Control" To Switch on Cell Therapy - Technology Networks

United Therapeutics received a building permit Tuesday for a $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4 million building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

Originally posted here:

United Therapeutics receives permit for cell therapy facility build-out at Mayo - Jacksonville Daily Record

NEW YORK, Oct. 24, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced, Chaim Lebovits, President and CEO, will serve as a Keynote Speaker at Cell Series UK.Cell Series UK, will be held October 29-30, 2019, at London Novotel West, London, UK. The Conference, organized by Oxford Global, is one of the foremost events in Europe focused on regenerative medicine and cellular innovation.

Ralph Kern MD, MHSc, Chief Operating and Chief Medical Officer of Brainstorm, who will also participate at Cell Series UK stated, We are very pleased to have Chaim Lebovits presenting at this prestigious conference where global leaders in stem cell and regenerative medicine will have the opportunity to learn more about NurOwn and the critical research being conducted by the Company. Mr. Lebovits Keynote Address, Stem Cell Therapeutic Approaches For ALS, will be presented to leading members of the scientific and business community including potential partners and investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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BrainStorm Cell Therapeutics President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 - Yahoo Finance

ImmuneCyte to Begin Operations in Fourth Quarter of 2019

Rancho Cordova, CA /PRNewswire/ - Cesca Therapeutics Inc. (Nasdaq: KOOL), a market leader in automated cell processing and autologous cell therapies for regenerative medicine, and ThermoGenesis, its wholly owned device subsidiary, today announced that the company has entered into a definitive joint venture agreement with HealthBanks Biotech (USA) Inc., one of the world's leading stem cell bank networks, to commercialize its proprietary cell processing platform, CAR-TXpress, for use in immune cell banking as well as for cell-based contract development and manufacturing services (CMO/CDMO). The joint venture will be named ImmuneCyte Life Sciences Inc. ("ImmuneCyte") and is expected to officially launch during the fourth quarter of 2019.

Under terms of the agreement, ImmuneCyte will initially be owned 80% by HealthBanks Biotech and 20% by Cesca. Cesca will contribute to ImmuneCyte exclusive rights to use ThermoGenesis' proprietary cell processing technology for the immune cell banking business and non-exclusive rights for other cell-based contract development and manufacturing services. Cesca will also contribute its clinical development assets to the joint venture, as the company has decided to discontinue these activities in order to focus exclusively on the device business.

Once operational, ImmuneCyte will be among the first immune cell banks in the U.S. to provide clients with the opportunity to bank their own healthy immune cells for future use as a resource for cell-based immunotherapies, such as dendritic cell and chimeric antigen receptor (CAR) T-cell therapies. ImmuneCyte will utilize ThermoGenesis' proprietary CAR-TXpress platform which allows for the isolation of different components from 200 ml of blood in cGMP compliant, closed system. Given that the CAR-TXpress platform can increase cell processing efficiency by up to 16-fold as compared with the traditional, labor-intensive ficoll gradient centrifugation-based cell processing method, ImmuneCyte is expected to offer customers an unparalleled competitive advantage, including an ability to store their own immune cells at a tangibly lower cost.

"The ImmuneCyte joint venture will be paramount to the execution of our strategy to become a preferred cell processing and manufacturing solution provider in the cell and gene therapy field," said Dr. Chris Xu, Chairman and Chief Executive Officer of Cesca Therapeutics. "CAR-T therapeutic research is advancing rapidly. Partnering with HealthBanks Biotech, one of the foremost stem cell bank networks, with an experienced team and an established global infrastructure, will offer customers the ability to preserve younger, healthier and uncontaminated immune cells for potential future use. By applying our proprietary CAR-TXpress technology to immune cell banking and other CDMO cellular manufacturing services, we will allow for the manufacture and production of more effective and less costly immunotherapies."

In 2017, the U.S. Food and Drug Administration (FDA) approved two CAR-T cell therapies, under breakthrough designation, for the treatment of advanced B cell leukemia and lymphomas. Both use autologous (a patient's own) immune T cells to fight cancer and have reported an over 80% response rate in the "no-option" patient group, for those who have failed both chemo- and radiation therapies. This has helped to spur massive global interest for the development of additional CAR-T immunotherapies1. By the end of September 2019, there were over 800 CAR-T cell clinical trials registered on the http://www.clinicaltrials.gov website, targeting a wide variety of blood cancers and solid tumors.

Although highly effective, several recent studies on the eligibility of patients to enroll in CAR-T clinical trials showed that as many as 30-50% of cancer patients may not be eligible to enroll or to get sufficient CAR-T cells manufactured for the therapy. Reasons may include: (1) the function of the immune system declines with age and can be negatively affected by other medical conditions, (2) most standard cancer therapies, such as chemotherapy and radiation, destroy the immune system, and (3) in many cases of advanced cancer, cancer cells will enter circulation, invade and interfere with the body's natural production of immune cells. According to a recently reported JULIE trial, a CAR-T clinical trial in relapsed or refractory diffuse large B-cell lymphoma (DLBCL), one-third of the 238 screened patients failed to be enrolled, and more than half of the 238 failed to receive the intended CAR-T therapy2,3. ImmuneCyte will offer customers the ability to preserve younger, healthier and uncontaminated immune cells, for potential future use in advanced cancer immunotherapy.

About HealthBanks Biotech (USA) Inc.HealthBanks Biotech, headquartered in Irvine, CA, is one of the leading stem cell bank networks in the world and offers services globally through its sister companies located in the United States and other regions and nations. HealthBanks Biotech is accredited by the FDA, AABB, and CAP. The HealthBanks Biotech group was originally founded in 2001 with a vision that stem cells and cell and gene therapies could transform modern medicine. HealthBanks Biotech is a subsidiary of Boyalife Group, Inc. (USA), an affiliate of Boyalife (Hong Kong) Limited, the largest stockholder of Cesca. For more information about HealthBanks Biotech (USA) Inc., please visit: http://www.healthbanks.us.

About ImmuneCyte Life Sciences Inc.ImmuneCyte will provide clients with the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood. For more information about ImmuneCyte Life Sciences Inc., please visit: http://www.immunecyte.com.

About Cesca Therapeutics Inc.Cesca Therapeutics develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. Its device division, ThermoGenesis develops, commercializes and markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology. The Company has developed a semi- automated, functionally closed CAR-TXpress platform to streamline the manufacturing process for the emerging CAR-T immunotherapy market. For more information about Cesca and ThermoGenesis, please visit: http://www.cescatherapeutics.com.

References:1. Facts About Chimeric Antigen Receptor (CAR) T-Cell Therapy, Leukemia and Lymphoma Society (2018). https://www.lls.org

2. Updated Analysis of JULIET Trial: Tisagenlecleucel in Relapsed or Refractory DLBCL (2018).

3. Eligibility Criteria for CAR-T Trials and Survival Rates in Chemorefractory DLBCL. Journal of Clinical Pathways (2018).

SOURCE: Cesca Therapeutics Inc.

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Cesca Therapeutics Forms Joint Venture With HealthBanks Biotech (USA) To Provide Immune Cell Banking And Cell Processing Service - Clinical Leader

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today presents updated long-term follow-up from the Phase 1/2 clinical trial of RP-L102 at the European Society of Cell and Gene Therapy (ESGCT) 27th Annual Congress in Barcelona, Spain. RP-L102 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA). The data are included in an oral presentation by Dr. Juan Bueren, Scientific Director of the FA gene therapy program and Head of the Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBERER / IIS-FJD, entitled, Gene Therapy for Patients with Fanconi Anaemia.

Two critical validations for an FA gene therapy product are: 1) stem cell engraftment in the absence of cytotoxic conditioning and 2) evidence of sustained clinical improvement. We are proud to report that the maturing long-term data from the patients treated with RP-L102 meet both of these requirements, said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. In all four patients, bone marrow MMC-resistance, a key measure of phenotypic reversal and engraftment, meets or exceeds the 10% threshold agreed to by the FDA and EMA for the upcoming registration-enabling Phase 2 trial, and all four patients now resemble FA mosaic patients as evaluated by peripheral T-cell chromosomal fragility assay. Remarkably, patients 02002 and 02006, who received what we consider adequate drug product similar to the upcoming Phase 2 trial, now demonstrate durable robust bone marrow MMC-resistance levels of approximately 60% and 32%, respectively, confirming phenotypic correction in long-term bone marrow stem and progenitor cells. Of note, each of the four initial patients continue to show evidence of a proliferative advantage, with ongoing increases in peripheral mononuclear cell VCNs. In addition, improvement or stabilization of peripheral blood counts, which had declined substantially prior to gene therapy, suggests a halt in bone marrow failure progression. In patient 02002, hemoglobin levels are now similar to those in the first year after birth, and all lineages in patients 02002 and 02006 are now stable or improving.

Dr. Shah continued, Preliminary VCN data from three additional patients who were treated with a viable drug product also show engraftment in a dose-dependent manner, consistent with the first four patients. With this progress to date, we look forward to the upcoming results from the first two patients receiving Process B of RP-L102, designed to enable consistent results with commercial-grade product.

The presentation described nine pediatric patients (ages 3-7 years) who received RP-L102 utilizing fresh or cryopreserved mobilized peripheral blood CD34+ cells that were transduced with the therapeutic vector. Four of these patients have been followed for more than 2 years (24-39 months for patients 02002, 02004, 02005, and 02006). The Phase 1/2 study of RP-L102 is an ongoing, open-label, single-center study designed to evaluate the safety and efficacy of Process A RP-L102 without the use of any conditioning regimen conventionally used in allogenic transplant.

Dr. Bueren noted, These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected HSCs in the absence of any conditioning regimen. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer. The ability to treat patients without the use of genotoxic conditioning and to restore blood cell counts is a life-altering advancement for patients and their families, as well as the scientific community which has dedicated over two decades to finding a minimally toxic alternative for FA patients.

Rocket expects initial data from the Phase 1 Process B trial of RP-L102 by year-end. The registration-enabling Phase 2 study in Spain is now enrolling, and additional global sites will follow.

Full results from the ESGCT presentation will be available online at the conclusion of the oral presentation: https://www.rocketpharma.com/esgct-presentations/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natures gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Presents First Evidence of Long-Term Improvement and Stabilization in Blood Counts and Durable Mosaicism in RP-L102 Process A...

A leading research firm, Zion Market Research added a latest industry report on "Global Stem Cell Therapy Market" consisting of 110+ pages during the forecast period and Stem Cell Therapy Market report offers a comprehensive research updates and information related to market growth, demand, opportunities in the global Stem Cell Therapy Market.

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The Stem Cell Therapy Market report provides in-depth analysis and insights into developments impacting businesses and enterprises on global and regional level. The report covers the global Stem Cell Therapy Market performance in terms of revenue contribution from various segments and includes a detailed analysis of key trends, drivers, restraints, and opportunities influencing revenue growth of the global consumer electronics market.This report studies the global Stem Cell Therapy Market size, industry status and forecast, competition landscape and growth opportunity. This research report categorizes the global Stem Cell Therapy Market by companies, region, type and end-use industry.

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The Stem Cell Therapy Market report mainly includes the major company profiles with their annual sales & revenue, business strategies, company major products, profits, industry growth parameters, industry contribution on global and regional level.This report covers the global Stem Cell Therapy Market performance in terms of value and volume contribution. This section also includes major company analysis of key trends, drivers, restraints, challenges, and opportunities, which are influencing the global Stem Cell Therapy Market. Impact analysis of key growth drivers and restraints, based on the weighted average model, is included in this report to better equip clients with crystal clear decision-making insights.

The Stem Cell Therapy Market research report mainly segmented into types, applications and regions.The market overview section highlights the Stem Cell Therapy Market definition, taxonomy, and an overview of the parent market across the globe and region wise.To provide better understanding of the global Stem Cell Therapy Market, the report includes in-depth analysis of drivers, restraints, and trends in all major regions namely, Asia Pacific, North America, Europe, Latin America and the Middle East & Africa, which influence the current market scenario and future status of the global Stem Cell Therapy Market over the forecast period.

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Major Company Profiles Covered in This Report:

Anterogen Co.,Ltd.,RTI SurgicalInc.,Pharmicell Co.,Ltd.,MEDIPOST Co.,Ltd.,JCR Pharmaceuticals Co.,Ltd.,Holostem Terapie Avanzate S.r.l.,NuVasiveInc.,and AlloSource.

Some of the major objectives of this report:

1) To provide detailed analysis of the market structure along with forecast of the various segments and sub-segments of the global Stem Cell Therapy Market.

2. To provide insights about factors affecting the market growth. To analyze the Stem Cell Therapy Market based on various factors- price analysis, supply chain analysis, porter five force analysis etc.

3. To provide historical and forecast revenue of the Stem Cell Therapy Market segments and sub-segments with respect to four main geographies and their countries- North America, Europe, Asia, and Rest of the World.

4. Country level analysis of the market with respect to the current market size and future prospective.

5. To provide country level analysis of the market for segment by application, product type and sub-segments.

6. To provide strategic profiling of key players in the market, comprehensively analyzing their core competencies, and drawing a competitive landscape for the market.

7. Track and analyze competitive developments such as joint ventures, strategic alliances, mergers and acquisitions, new product developments, and research and developments in the global Stem Cell Therapy Market.

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Zion Market Research is an obligated company. We create futuristic, cutting edge, informative reports ranging from industry reports, company reports to country reports. We provide our clients not only with market statistics unveiled by avowed private publishers and public organizations but also with vogue and newest industry reports along with pre-eminent and niche company profiles. Our database of market research reports comprises a wide variety of reports from cardinal industries. Our database is been updated constantly in order to fulfill our clients with prompt and direct online access to our database. Keeping in mind the clients needs, we have included expert insights on global industries, products, and market trends in this database. Last but not the least, we make it our duty to ensure the success of clients connected to usafter allif you do well, a little of the light shines on us.

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Global Stem Cell Therapy Market Global and Regional Analysis by Top Key Market Players, Key Regions, Product Segments, and Applications 2024 - Globe...

BOSTON and LONDON, Oct. 22, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The initial data show that cellular engraftment with OTL-200 using a cryopreserved formulation is similar to that observed using a fresh formulation with the longest patient having 12 months of follow-up since treatment. The data are being featured this week in a poster session at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Barcelona, Spain.

MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms, patients will not survive beyond their first decade of life.

These data compare the initial results of OTL-200 in the first four MLD patients treated using a cryopreserved formulation to a previously presented integrated analysis of 29 patients treated with a fresh formulation that demonstrated meaningful clinical outcomes. Hematopoietic stem cells are collected, purified and transduced in the same way for both formulations. For the cryopreserved formulation, following transduction, the gene-corrected cells are placed in a specific medium that allows them to be stably frozen. After successful testing and release, the cryopreserved cells are shipped to the site of care where they are thawed and administered to patients who have received conditioning.

Presenting the first supportive data on OTL-200 using a cryopreserved formulation represents a cross-functional effort involving our clinical, CMC and regulatory teams as we prepare for the upcoming European regulatory submission for MLD followed by a BLA in the U.S., said Mark Rothera, president and chief executive officer of Orchard. If approved, a cryopreserved formulation of OTL-200 would more readily facilitate global commercialization and patient access efforts, which are key elements in our mission to deliver potentially curative therapies to patients suffering from often-deadly rare diseases.

Mr. Rothera continued, With over 40 patients now treated using a cryopreserved formulation across our pipeline of six clinical-stage programs, we are confident our approach is supported by a robust set of evidence.

Study Results At the time of the analysis, four early-onset MLD patients (two late infantile and two early juvenile) have been treated with the cryopreserved formulation of OTL-200. All patients are alive and were followed for a minimum of one month, with the longest follow-up out to 12 months in the first patient treated (median follow-up of 0.38 years). The age at the time of treatment ranged from seven months to 42 months.

The initial results in patients receiving the cryopreserved formulation (n=4) demonstrated the following:

Figure 1. Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/83f41457-927b-4b1b-9ac2-9d48ac10353a

Figure 2. ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/393ca5f0-98ad-47f8-b723-35c5c6c08d8f

c = cryopreserved; f = fresh; Sbj. = subject

We are pleased that these initial data suggest that using gene-corrected cells that have been cryopreserved has a similar impact on clinical biomarkers for early-onset MLD patients as the OTL-200 fresh formulation, said Dr. Valeria Calbi, a hematologist at San Raffaele Scientific Institute and SR-Tiget and an investigator of the study. The four treated patients showed good levels of engraftment of gene-corrected cells and reconstitution of ARSA activity at multiple time points, as well as encouraging early trends in GMFM scores that we look forward to evaluating with additional follow-up. We believe that these data further support the positive benefit / risk profile of OTL-200 as a therapy with potential lifelong benefit for patients with MLD.

Next Steps for OTL-200 Orchard remains on track to submit a marketing authorization application, or MAA, in Europe for MLD in the first half of 2020, as well as a biologics licensing application, or BLA, in the U.S. approximately one year later.

About MLD and OTL-200Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About OrchardOrchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including OTL-200 for the treatment of metachromatic leukodystrophy, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, including OTL-200, and the likelihood that such data will be positive and support further clinical development and regulatory approval of its product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including OTL-200, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading Risk Factors in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Figure 1

Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

Figure 2

ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh; c = cryopreserved; f = fresh; Sbj. = subject

See the original post here:

Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation - BioSpace

24 October 2019

Most cancers kill because tumour cells spread, or metastasise beyond the primary site, for example breast, to invade other organs, brain being one. Now, a University of Southern California (USC), study has found that circulating tumour cells can actually target specific distant organs.

Their study of brain-invading breast cancer reveals that circulating tumour cells have a molecular signature indicating specific organ preferences.

The findings, which appear in Cancer Discovery, explain how tumour cells in the blood target a particular organ and may enable the development of treatments to prevent the spread of these metastatic cancers.

In this study breast cancer cells from the blood of breast cancer patients with metastatic tumours were isolated, expanded and grown in the lab.

Analysis of these cells identified regulator genes and proteins within the cells that apparently directed the cancers spread to the brain. The team were therefore able to predict that a patients breast cancer cells would eventually migrate to the brain.

Assistant professor of stem cell and regenerative medicine at the Keck School of Medicine at USC, Min Yu, also discovered that a protein on the surface of these brain-targeting tumour cells helps them to breech the blood brain barrier and lodge in brain tissue, while another protein inside the cells shield them from the brains immune response, enabling them to grow there.

We can imagine someday using the information carried by circulating tumour cells to improve the detection, monitoring and treatment of the spreading cancers, Yu said.

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People considering stem cell treatment for arthritis want to know Is it safe? and Is it effective?

Most stem cell therapy using adult stem cells is considered safe because the stem cells are collected from the patient, minimizing the risk of an unwanted reaction. The most common side effects are temporary swelling and pain.3

While most stem cell therapy for arthritis is considered safe, it does carry the same risks as any other medical procedure, such as a small risk of infection. Risk may be increased if:

See What Are Stem Cells?

Some research suggests stem cell therapy engaging in these kinds of practices may elevate the risk of tumors.4

As with most regenerative medicine treatments, research is ongoing, and FDA regulations are relatively new and subject to change.

Article continues below

Whether or not stem cells therapy is effective in treating osteoarthritis is a controversial subject among medical professionals, and research in the area is ongoing.

See Osteoarthritis Treatment

How researchers think stem cell therapy worksResearchers theorize5 that when applied to an arthritic joint, stem cells might:

See Osteoarthritis Symptoms and Signs

It may be none, one, two, or all three processes at are work.

Proponents vs criticsLike many relatively new treatments, stem cell therapy has proponents and critics.

Critics emphasize that there have been no large-scale, prospective, double-blind research studiesthe kind of clinical studies that medical professionals consider the gold standardto support stem cell therapy for arthritis.

Factors that affect stem cell therapy researchAnother challenge associated with current stem cell research is that there is no standard stem cell therapy for arthritis treatment. So the stem cell therapy in one study is not necessarily the same as the stem cell therapy in another study.

Differences can include:

These differences are further complicated by more unknowns. For example, how many stem cells are needed for a particular treatment? And how do we determine if a patients own stem cells are competent enough to aid in healing?

Many physicians combine the use of stem cells with platelet rich plasma, or PRP.

See Platelet-Rich Plasma (PRP) Therapy for Arthritis

PRP is derived from a sample of the patients blood. In the body, platelets secrete substances called growth factors and other proteins that regulate cell division, stimulate tissue regeneration, and promote healing. Like stem cell therapy, PRP therapy is sometimes used alone with the hopes of healing an arthritic joint.

See PRP Injection Preparation and Composition

Physicians who use PRP and stem cells together think that the PRP can help maximize the healing effects of stem cells.7,8 Research in this area is ongoing.

See Platelet-Rich Plasma Injection Procedure

Stem cell therapy can vary depending on the doctor performing it. People considering stem cell therapy for an arthritic knee or other joint are advised to ask their doctors questions, including:

Both doctors and patients can benefit from having a frank conversation and setting reasonable expectations.

See Arthritis Treatment Specialists

Complete Listing of References

Read more from the original source:

Is Stem Cell Therapy for Arthritis Safe and Effective?

Stem cell facts

What are stem cells?

Stem cells are cells that have the potential to develop into many different or specialized cell types. Stem cells can be thought of as primitive, "unspecialized" cells that are able to divide and become specialized cells of the body such as liver cells, muscle cells, blood cells, and other cells with specific functions. Stem cells are referred to as "undifferentiated" cells because they have not yet committed to a developmental path that will form a specific tissue or organ. The process of changing into a specific cell type is known as differentiation. In some areas of the body, stem cells divide regularly to renew and repair the existing tissue. The bone marrow and gastrointestinal tract are examples of areas in which stem cells function to renew and repair tissue.

The best and most readily understood example of a stem cell in humans is that of the fertilized egg, or zygote. A zygote is a single cell that is formed by the union of a sperm and ovum. The sperm and the ovum each carry half of the genetic material required to form a new individual. Once that single cell or zygote starts dividing, it is known as an embryo. One cell becomes two, two become four, four become eight, eight become sixteen, and so on, doubling rapidly until it ultimately grows into an entire sophisticated organism composed of many different kinds of specialized cells. That organism, a person, is an immensely complicated structure consisting of many, many, billions of cells with functions as diverse as those of your eyes, your heart, your immune system, the color of your skin, your brain, etc. All of the specialized cells that make up these body systems are descendants of the original zygote, a stem cell with the potential to ultimately develop into all kinds of body cells. The cells of a zygote are totipotent, meaning that they have the capacity to develop into any type of cell in the body.

The process by which stem cells commit to become differentiated, or specialized, cells is complex and involves the regulation of gene expression. Research is ongoing to further understand the molecular events and controls necessary for stem cells to become specialized cell types.

Stem Cells:One of the human body's master cells, with the ability to grow into any one of the body's more than 200 cell types.

All stem cells are unspecialized (undifferentiated) cells that are characteristically of the same family type (lineage). They retain the ability to divide throughout life and give rise to cells that can become highly specialized and take the place of cells that die or are lost.

Stem cells contribute to the body's ability to renew and repair its tissues. Unlike mature cells, which are permanently committed to their fate, stem cells can both renew themselves as well as create new cells of whatever tissue they belong to (and other tissues).

Why are stem cells important?

Stem cells represent an exciting area in medicine because of their potential to regenerate and repair damaged tissue. Some current therapies, such as bone marrow transplantation, already make use of stem cells and their potential for regeneration of damaged tissues. Other therapies that are under investigation involve transplanting stem cells into a damaged body part and directing them to grow and differentiate into healthy tissue.

Embryonic stem cells

During the early stages of embryonic development the cells remain relatively undifferentiated (immature) and appear to possess the ability to become, or differentiate, into almost any tissue within the body. For example, cells taken from one section of an embryo that might have become part of the eye can be transferred into another section of the embryo and could develop into blood, muscle, nerve, or liver cells.

Cells in the early embryonic stage are totipotent (see above) and can differentiate to become any type of body cell. After about seven days, the zygote forms a structure known as a blastocyst, which contains a mass of cells that eventually become the fetus, as well as trophoblastic tissue that eventually becomes the placenta. If cells are taken from the blastocyst at this stage, they are known as pluripotent, meaning that they have the capacity to become many different types of human cells. Cells at this stage are often referred to as blastocyst embryonic stem cells. When any type of embryonic stem cells is grown in culture in the laboratory, they can divide and grow indefinitely. These cells are then known as embryonic stem cell lines.

Fetal stem cells

The embryo is referred to as a fetus after the eighth week of development. The fetus contains stem cells that are pluripotent and eventually develop into the different body tissues in the fetus.

Adult stem cells

Adult stem cells are present in all humans in small numbers. The adult stem cell is one of the class of cells that we have been able to manipulate quite effectively in the bone marrow transplant arena over the past 30 years. These are stem cells that are largely tissue-specific in their location. Rather than typically giving rise to all of the cells of the body, these cells are capable of giving rise only to a few types of cells that develop into a specific tissue or organ. They are therefore known as multipotent stem cells. Adult stem cells are sometimes referred to as somatic stem cells.

The best characterized example of an adult stem cell is the blood stem cell (the hematopoietic stem cell). When we refer to a bone marrow transplant, a stem cell transplant, or a blood transplant, the cell being transplanted is the hematopoietic stem cell, or blood stem cell. This cell is a very rare cell that is found primarily within the bone marrow of the adult.

One of the exciting discoveries of the last years has been the overturning of a long-held scientific belief that an adult stem cell was a completely committed stem cell. It was previously believed that a hematopoietic, or blood-forming stem cell, could only create other blood cells and could never become another type of stem cell. There is now evidence that some of these apparently committed adult stem cells are able to change direction to become a stem cell in a different organ. For example, there are some models of bone marrow transplantation in rats with damaged livers in which the liver partially re-grows with cells that are derived from transplanted bone marrow. Similar studies can be done showing that many different cell types can be derived from each other. It appears that heart cells can be grown from bone marrow stem cells, that bone marrow cells can be grown from stem cells derived from muscle, and that brain stem cells can turn into many types of cells.

Peripheral blood stem cells

Most blood stem cells are present in the bone marrow, but a few are present in the bloodstream. This means that these so-called peripheral blood stem cells (PBSCs) can be isolated from a drawn blood sample. The blood stem cell is capable of giving rise to a very large number of very different cells that make up the blood and immune system, including red blood cells, platelets, granulocytes, and lymphocytes.

All of these very different cells with very different functions are derived from a common, ancestral, committed blood-forming (hematopoietic), stem cell.

Umbilical cord stem cells

Blood from the umbilical cord contains some stem cells that are genetically identical to the newborn. Like adult stem cells, these are multipotent stem cells that are able to differentiate into certain, but not all, cell types. For this reason, umbilical cord blood is often banked, or stored, for possible future use should the individual require stem cell therapy.

Induced pluripotent stem cells

Induced pluripotent stem cells (iPSCs) were first created from human cells in 2007. These are adult cells that have been genetically converted to an embryonic stem celllike state. In animal studies, iPSCs have been shown to possess characteristics of pluripotent stem cells. Human iPSCs can differentiate and become multiple different fetal cell types. iPSCs are valuable aids in the study of disease development and drug treatment, and they may have future uses in transplantation medicine. Further research is needed regarding the development and use of these cells.

Why is there controversy surrounding the use of stem cells?

Embryonic stem cells and embryonic stem cell lines have received much public attention concerning the ethics of their use or non-use. Clearly, there is hope that a large number of treatment advances could occur as a result of growing and differentiating these embryonic stem cells in the laboratory. It is equally clear that each embryonic stem cell line has been derived from a human embryo created through in-vitro fertilization (IVF) or through cloning technologies, with all the attendant ethical, religious, and philosophical problems, depending upon one's perspective.

What are some stem cell therapies that are currently available?

Routine use of stem cells in therapy has been limited to blood-forming stem cells (hematopoietic stem cells) derived from bone marrow, peripheral blood, or umbilical cord blood. Bone marrow transplantation is the most familiar form of stem cell therapy and the only instance of stem cell therapy in common use. It is used to treat cancers of the blood cells (leukemias) and other disorders of the blood and bone marrow.

In bone marrow transplantation, the patient's existing white blood cells and bone marrow are destroyed using chemotherapy and radiation therapy. Then, a sample of bone marrow (containing stem cells) from a healthy, immunologically matched donor is injected into the patient. The transplanted stem cells populate the recipient's bone marrow and begin producing new, healthy blood cells.

Umbilical cord blood stem cells and peripheral blood stem cells can also be used instead of bone marrow samples to repopulate the bone marrow in the process of bone marrow transplantation.

In 2009, the California-based company Geron received clearance from the U. S. Food and Drug Administration (FDA) to begin the first human clinical trial of cells derived from human embryonic stem cells in the treatment of patients with acute spinal cord injury.

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What are experimental treatments using stem cells and possible future directions for stem cell therapy?

Stem cell therapy is an exciting and active field of biomedical research. Scientists and physicians are investigating the use of stem cells in therapies to treat a wide variety of diseases and injuries. For a stem cell therapy to be successful, a number of factors must be considered. The appropriate type of stem cell must be chosen, and the stem cells must be matched to the recipient so that they are not destroyed by the recipient's immune system. It is also critical to develop a system for effective delivery of the stem cells to the desired location in the body. Finally, devising methods to "switch on" and control the differentiation of stem cells and ensure that they develop into the desired tissue type is critical for the success of any stem cell therapy.

Researchers are currently examining the use of stem cells to regenerate damaged or diseased tissue in many conditions, including those listed below.

References

REFERENCE:

"Stem Cell Information." National Institutes of Health.

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What Are Stem Cells? Research, Transplant, Therapy, Definition

Clinical research regarding stem cell therapy benefits has grown dramatically in recent decades. The most promising thing about stem cell therapy and similar prolotherapy treatments including PRP is that they offer relief for patients with chronic pain and difficult-to-heal injuries, all without medications or risky reconstructive surgeries. Today researchers are also uncovering ways to apply stem cell treatments for common chronic conditions such as heart disease,neurodegenerative diseases and diabetes.

The most common use of stem cell treatments in prolotherapy is managing pain. Most consider stem cell therapy to be a form of interventional pain-management, meaning its a minimally invasive technique. Treatment involves injecting stem cells (along with an anesthetic and sometimes other substances) around painful and damaged nerves, tendons, joints or muscle tissue.

What specific types of conditions can stem cell therapy help treat? Some of the most common include osteoarthritis knee pain, tennis elbow, shoulder pains or rotator cuff injuries, tendonitis, Achilles tendon injuries, ACL injuries and now cardiovascular diseases likeatherosclerosis.

There are now more options available to patients than ever before for various types of prolotherapy treatments, but the type of prolotherapyI recommend the most is the unique approach to stem cell therapy offered by the Regenexxclinic. I have personally visited the Regenexx clinic in the Cayman Islands to receive treatments performed by Dr. Chris Centeno, Dr. John Schultz and Dr. John Pitt for back and tendon injuries. The form of stem cell therapy offered by these doctors is considered to be one of themost thoroughly researched and effective in the world.

Stem cell therapy is a type of treatment option that uses a patients own stem cells to help repair damaged tissue and repair injuries. Its usually performed relatively quickly through injections, and is a simple outpatient or in office procedure.

This type of treatment has also been found to help:

According to the National Institute of Health,

Stem cells are important for living organisms for many reasons. In the 3- to 5-day-old embryo, called a blastocyst, the inner cells give rise to the entire body of the organism, including all of the many specialized cell types and organs such as the heart, lungs, skin, sperm, eggs and other tissues. In some adult tissues, such as bone marrow, muscle, and brain, discrete populations of adult stem cells generate replacements for other cells that are lost through normal wear and tear, injury, or disease.

The California Stem Cell Agency reports that there is no limit to the types of diseases that could be treated with stem cell research. Because of their amazing abilities to help with regrowth, stem cell therapy treatments are now being used (or continuously researched) in regards to treating:

Stem cells are usually taken from one of two areas in the patients body: bone marrow or adipose (fat) tissue in their upper thigh/abdomen. Because its common to remove stem cells from areas of stored body fat, some refer to stem cell therapy as Adipose Stem Cell Therapy in some cases. (1)

Once stem cells from removed from one of these locations, they are placed in a centrifuge machine that spins them very, very quickly and concentrates the substances that are most valuable (including up to seven different types of natural growth factors). The sample of concentrated stem cells is then injected directly into the patients affected, painful area allowing the cells growth factors to go to work immediately, building new skin cells, connective tissue and so on.

What exactly makes stem cells so beneficial and gives stem cell injections the power to do this healing? Stem cells have the following unique characteristics, uses and healing abilities:

The type of stem cells being used in the most cutting-edge orthopedic practices including those offered at the Regenexx clinic mentioned above are called Mesenchymal stem cells (MSCs). A growing body of research shows that MSCs have the capability of differentiating and forming new orthopedic tissues that make up muscle, bones, cartilage and tendons, ligaments and adipose tissue. (3)

Research suggests that in treating orthopedic problems,fat-derived MSCs tend to under-perform bone marrow derived stem cells, therefore bone derived is the preferred method. (4) This is especially true when bone marrow cells are dramatically concentrated using advanced centrifuge equipment. Certain studies have found that these advanced samples can contain up to 25 different growth factors and other beneficial rebuilding substances.

In studies regarding orthopedic care such as those used for cartilage replacement,bone repairand soft-tissue repair bone marrow stem cells injections have been found to: reduce chronic pain, heal stubborn injuries, improve functionality and return patients to their normal routine sometimes within just one week.

Wondering if MSCs for orthopedic injuries are safe? There is no evidence of overgrowth of MSCs in damaged tissue or reason to believe theres risk for tumor growth. Advanced clinics such as Regenexx actually count cells before injecting them and carefully monitor progress. According to research used by Regenexx, MSCs safely stop proliferating once they physically contact each other, because this signals to them that the affected area has reached its full potential in growth. (5)

Cardiovascular diseases can deprive heart tissue of oxygen and cause scar tissue to form which changes blood flow/blood pressure. Research suggests that stem cells taken from adult bone marrow have the ability to differentiate into those needed to repair the heart and blood vessels, thanks to the secretion of multiple growth factors. Several ways in which stem cell therapy is now being used and further researched in regards to improving recovery of heart disease are:

Although more research is needed to assess the safety and efficacy of this approach, stem cell types used in heart disease treatment include: embryonic stem (ES) cells, cardiac stem cells,myoblasts (muscle stem cells), adult bone marrow-derived cells, umbilical cord blood cells, mesenchymal cells (bone marrow-derived cells) and endothelial progenitor cells (these form the interior lining of blood vessels).

Studies have found that stem cell treatments can help improve the growth of healthy new skin tissue, improve collagen production, stimulate hair growth after loss or incisions, and help replace scar tissue with newly formed healthy tissue.

One of the ways stem cells help facilitate wound healing is by increasingcollagen concentrations in the skin, which shrinks as it matures and thereby strengthens and tightens the damaged area. This same mechanism also applies to treating connective tissue injuries related to collagen/cartilage loss, such as those caused by osteoarthritis or overuses that affect ligaments or tendons.

Recent progress in the treatment of diseases like Parkinsons, Huntingtons, Alzheimers and stroke recovery show that transplanted adult stem cells can be used to form new brain cells, neurons and synapses following cognitive degeneration or brain injuries. (6) Research conducted by the Research Center for Stem Cell Biology and Cell Therapy in Sweden is still underway, but current findings show that stem cells can improve synaptic circuits, optimize functional recovery, offer relief from degeneration symptoms, slow down disease progression and potentially even more.

Some of the ways that stem cell injections/grafts work in neurodegeneration treatment are: normalizing striatal dopamine release, impairing akensia (loss of voluntary movement), replacing neurons destroyed by the ischemic lesions following strokes and halting destruction of nigrostriatal dopaminergic neurons.

Immune rejection is the term used to describe damage to healthy tissue and cells in patients with autoimmune disorders and other inflammatory conditions. In people who suffer from type1 diabetes, for example, the cells of the pancreas that normally produce insulin are destroyed by the patients own immune system; in people with thyroid disorders, the thyroid gland is attacked and damaged.

Research continues to show us that certain adult stem cells are capable of differentiating and producing needed cells, such as insulin-producing cells that eventually could be used in with people diabetes. This strategy is still being researched extensively and is not yet widely available, as scientists continue to experiment with reliable strategies for generating new cells/tissues that will not be rejected or harm the patient once implanted.

Meanwhile, a promising clinical trial led by Dr. Richard Burt of Northwestern University that explores the potential benefits of stem cell therapy for multiple sclerosis is underway as of March 2018. The 110 patients participating either received a drug treatment or hematopoietic stem cell transplantation (HSCT).The clinical trial looks promising given that after one year of treatment only one relapse occurred among patients in the stem cell group compared with 39 relapses in the drug treatment. And, after about three years, the stem cell transplants had a 6 percent failure rate compared with a failure rate of 60 percent in the control (drug treatment) group.

The researchers note that stem cell therapy doesnt work for all cases of MS and its not an easy process. First patients must undergo chemotherapy to destroy their faulty immune system. Then stem cells that help make blood through a process called hematopoiesis are removed from the patients bone marrow and reinfused into the patients bloodstream. These fresh stem cells, which are not affected by MS, rebuild the patients immune system. Despite this challenging process, preliminary results demonstrate that this could be an effective treatment in the future. (7, 8)

For decades researchers and doctors primarily used two kinds of stem cells taken from animals and humans, especially when they were still embryos (not yet born). These are calledembryonic stem cells and non-embryonic (somatic or adult) stem cells. In the late 1990s, it was discovered that stem cells could be taken from human embryos and grown inside of laboratoriesfor reproductive purposes, including for in vitro fertilization.

Then in 2006 a breakthrough discovery was made that some specialized adult stem cells could be reprogrammed and used in many other ways to help repair damaged tissue. These are referred to as induced pluripotent stem cells (iPSCs) and are the type used in many of the treatments described above.There remains a lot to learn about the potential uses of stem cell therapies, and how scientists can continue to explore transforming unspecialized adult stem cells into the types of specialized cells needed.

The NIH reports that in future years some of the primary goals of stem cell therapy research are to: identify howundifferentiated stem cells become the differentiated cells that form the tissues and organs, determine how stem cells can turn humangenes on and off, learn to predictably control cell proliferation and differentiation, and investigate more uses for stem cells in serious medical conditions such as cancerand birth defects.

The hope going forward is that stem cells can also be used as a renewable source of replacement cells and tissues to treat common and serious diseases without the need for organ transplants or surgeries, including: maculardegeneration, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis, rheumatoid arthritis and cancer.

Cancer treatment is a particular important area under investigation, as early studies are showing that stem cells are safe and well-tolerated in patients with acute and chronic leukemia, lymphoma, multiple myeloma and other cancers. (9)

Stem cell treatments are offered by various doctors who practice pain-management and other techniques, including orthopedics and anesthesiologists.Depending on the type of treatment needed, its also possible to visit a neurologist, cardiologist, etc.Commonly these treatments are offered at clinics with ateam of doctors who work together to specialize in diagnosing, preventing and/or correcting a range of musculoskeletal, neurological or connective tissue disorders/injuries.

If youre planning on visiting a doctor for pain management, look for a physician who has board certification through an organization like the American Board of Anesthesiology orAmerican Board of Pain Medicine. I recommend viewing this Physician Finder tool to locate a practitioner who performs the advanced type of stem cell applications described above.

Personally, I most suggest checking out Regenexx, one of the only organizations to run large-scale analysis of patient stem cell procedure outcome data. It has published numerous findings from tracking their own patients on their website. Much more detailed information on improvements that can be expected following PRP procedures including those for knee meniscus, arthritis, hip dysfunction, knee pain, wrist/hand injuries, ankle/foot pain and shoulder/rotator injuries can be accessed through Regenexx directly.

Once you find a qualified physician, heres a brief overview of what you can expect from stem cell therapy treatments:

Although stem cell treatment is considered to be very safe, there are also side effects that are possible. Make sure to find a qualified practitioner and let them know if your experience following a treatment does not sound like the typical one described above.Like other types of non-invasive treatments and prolotherapy techniques, some mild side effects after injections are normal. Side effects of stem cell treatments can sometimes include:

Continue reading here:

5 Stem Cell Therapy Benefits, Uses & How It Works - Dr. Axe

Stem cells are unquestionably some of the most amazing cells in the human body. These are undifferentiated cells that do not have a direct blueprint or specific destiny. They can become differentiated into specialized cells anywhere throughout the body. They are classified as 2 different types of cells, those that are from embryonic origin and those called adult stem cells.

In the developing embryo, these cells differentiate into ectoderm, endoderm, and mesoderm. These give us rise to our spine, nerves, and all our organs. Adult stem cells are primarily used to repair, replenish, and regenerate tissues.

Historically, stem cells can come from a variety of tissues. These include umbilical cord, fetal tissue, adipose, or the best source bone marrow.

Bone marrow stem cells have the highest numbers of cells when collected and tested compared to all others. This is by far the preferred method of stem cell therapy because of sheer numbers and the fact that they are coming from your own body. This is called autologous therapy.

Stem cell research worldwide goes back over 100 years, the German research is documented back to 1860. There are a wide range of studies and articles describing its dramatic benefit for chronic diseases. Many of these publications are available for you to read on my website.

In performing stem cell therapy, extremely strict guidelines must be followed in coordination with a specialized protocol. This ensures accuracy, sterility, and quality control of the procedure. This information gathered from the procedure, including various forms of documentation can be used for medical publication at a later date. Physician notes and procedure as well as a questionnaire filled out by patients periodically are part of this process. This enables the highest level of procedure and documentation possible.

Initially, patients are examined, appropriate blood or other testing is done and reviewed and schedule is made to begin procedure. Typically, stem cell therapy is done within 2 weeks of initial consultation.

On the day of procedure, stem cells are extracted from Bone Marrow, this takes 5-10 minutes then patients sit and relax while the processing is done. It is then washed and centrifuged 3 times to allow separation of cells and harvest stem cells. At the end of the procedure, microscopic analysis can estimate the number of stem cells available for injection. Injection can be done either into joint, connective tissue, muscle or for all other organs or systemic diseases, intravenously. Intranasal technique also used for MS, Parkinsons, and Alzheimers disease. Intravenous and nebulizer is used for COPD, Emphysema, and Pumonary Fibrosis.

Research shows the therapy may benefit the following:

* Legal Disclaimer: Chelation and Hyperbaric Therapy, Stem Cell Therapy, and other treatments and modalities mentioned or referred to in this web site are medical techniques that may or may not be considered mainstream. As with any medical treatment, results will vary among individuals, and there is no implication or guarantee that you will heal or achieve the same outcome as patients herein.

As with any procedure, there could be pain or other substantial risks involved. These concerns should be discussed with your health care provider prior to any treatment so that you have proper informed consent and understand that there are no guarantees to healing.

THE INFORMATION IN THIS WEBSITE IS OFFERED FOR GENERAL EDUCATIONAL PURPOSES ONLY AND DOES NOT IMPLY OR GIVE MEDICAL ADVICE. No Doctor/Patient relationship shall be deemed to have arisen simply by reading the information contained on these pages, and you should consult with your personal physician/care giver regarding your medical treatment before undergoing any sort of treatment or therapy.

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Stem Cell Therapy / drcalapai.net

Adipose Derived Stem Cells:

Adipose derived stem cells are obtained from a sample of human abdominal fat tissue. The cells themselves are not fat. They are encased in and surrounded by fat tissue.Once the fat sample has been obtained, a special enzyme is used to melt-away the fat, and leave only something called SVF (short for Stromal Vascular Fraction), which contains stem cells, accessory cells and growth factors.

This SVF is washed and purified in the lab, and is then isolated for infusion or injection.

This method provides a much larger number of stem cells than bone marrow or peripheral blood, making it more efficient and highly effective in a variety of conditions, especially those that require creation of new blood vessels, and repairing tissues damaged due to lack of oxygenation.

Whartons Jelly Derived Stem cells:

Unlike Fat, Bone marrow or other tissues that can be harvested for stem cell isolation, Whartons Jelly does not contain SVF. It is a gelatinous substance found in the umbilical chord, which separates maternal and fetal tissues, acting as a kind of buffer so that these tissues dont come into direct contact with each other. This particular characteristic is the reason they became known as universal donor cells, since they are able to interact with any tissue, in any host, without causing any form of immune response.

Unlike cells obtained with SVF, stem cells in Whartons jelly are not found grouped with other cells types or blood products. They can be obtained in much larger numbers, and are already completely isolated, which means that you get stem cells exclusively.

The fact that they are obtained from umbilical chords, donated by pre-screened donors after their pregnancy has come to full-term, means that they are much easier to harvest, in much larger numbers, and without the need for a specific, invasive and painful procedure. It also means that because of the incredibly large number of cells that can be obtained, they can be cryo-preserved and stored at pre-determined dose sizes in individual containers for specific uses.

The rest is here:

Stem Cell Therapy in Mexico - Certified Treatment Clinics

Cardiac-Pulmonary Conditions

Led by Melvin M. Propis, M.D., South Florida Stem Cell Center is one of theleading Stem Cell Regenerative Therapy Clinics in South Florida. Dr. Propis is a seasoned M.D. and surgeon who has had solid success rates.

Stem Cell Regenerative Therapy is a breakthrough in medical science that treats and prevents conditions and diseases using stem cells. This is accomplished by harvesting cells and then concentrating those cells in a lab before precisely re-injecting them. This greatly increases your bodys own natural repair cells and promotes healing.

South Florida Stem Cell Center is made up of research scientists and experts in Stem Cell Therapy.Our passion and belief is that our treatments truly helpthose that are suffering and need our help.

Maribella MKnee Injury

I injured both of my knees. After confirming that the cartilage was still in the joint, Dr. Propis injected my knees with a mixture of stem cells and PRP 4 months ago. Today I walk comfortably, No pain in those joints. I have noticed significant improvement in my balance and no longer need a walker or narcotics for pain.

Mia HCrohn's Disease

I have had Crohns disease for most of my short life which has led me to miss out on many teenage activities. After seeing other patients improve from having stem cells injected, I (and my mother) decided to try it. It was a wonderful thing to gradually be able to discontinue giving myself Humara shots routinely. I can actually have an active social life without worrying and even married the love of my life last year. Thanking my doctor, mom, God, and the many people who believe in stem cells for my happy ending!

George BDiabetes

I flew to the US in hopes of getting help for my diabetes. Having tried medicine & diets with no results, I was ready to try stem cells. After 1 treatment (and a six month period) I am off all meds and not considered diabetic anymore. To me, life changing! Especially after a relatively simple procedure. Thank you to the office of Dr. Propis and staff.

We Specialize In Treating:

Immunological Conditions

A chronic inflammatory bowel disease that affects the lining of the digestive tract.

Widespread muscle pain and tenderness.

A chronic inflammatory disorder affecting many joints, including those in the hands and feet.

An inflammatory disease caused when the immune system attacks its own tissues.

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Neurological Conditions

A congenital disorder of movement, muscle tone, or posture.

Damage to the brain from interruption of its blood supply.

A progressive disease that destroys memory and other important mental functions.

A disease in which the immune system eats away at the protective covering of nerves.

A disorder of the central nervous system that affects movement, often including tremors.

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Degenerative Conditions

Damage to any part of the spinal cord or nerves at the end of the spinal canal.

A chronic condition that affects the way the body processes blood sugar (glucose).

Kidney Failure (Renal Failure)

A condition in which the kidneys lose the ability to remove waste and balance fluids.

A type of arthritis that occurs when flexible tissue at the ends of bones wears down.

Occurs when a man can't get or keep an erection firm enough for sexual intercourse.

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South Florida Stem Cell Center | Regenerative Therapy Clinic

There is exciting and innovative research and progress occurring related to the potential of many types of stem cells for slowing MS disease activity and for repairing damage to the nervous system. In light of the urgent need for more effective treatments for MS, particularly for those with more progressive forms of the disease, we believe that the potential of all types of cell therapies must be explored.

Stem cell therapy is any treatment that uses or targets stem cells, which are the types of cells that differntiate into many different specialized cells in our bodies. Stem cells are found in both embryos and adults.

Many types of stem cells are being explored for their potential benefits for treating multiple sclerosis. Only when the results of these and subsequent clinical trials are available will it be possible to determine what the optimal cells, delivery methods, safety and actual effectiveness of these current experimental therapies might be for people with MS.

Although cell based therapy has generated a great deal of interest and holds promise, the field is in its infancy and much more research is needed before cell based therapies become a MS treatment option.

Continue reading here:

Stem Cells in MS : National Multiple Sclerosis Society

Infusio uses various forms of stem cell-based therapies. One of the most advanced forms of regenerative medicine is called Stromal Vascular Fraction (SVF). This complex combination of mesenchymal and blood stem cells, exosomes, and transcription factors plays a vital role in combating chronic degenerative diseases like multiple sclerosis, diabetes, COPD, neurological disorders, and autoimmune diseases.

The process involves the removal of approximately 50ccs of a patients own fat tissue. Impurities are removed and the highly concentrated serum is then prepared and re-introduced intravenously causing a systemic response.

The stem cells and supportive cells contained in SVF are able to regulate the immune response, counteract inflammation, and even repair organ and nerve damage. SVF can also be administered via injection to target specific joint damage and rebuild cartilage in a particular area. This one-two punch of tissue regeneration and immune recalibration is one of the most powerful treatments Infusio offers.

As a follow up therapy, we often use PBMC an auto-cyto immunotherapy (ACT) which utilizes specific immune cells that are extracted from your blood and reintroduced to strengthen the immune response in the ongoing recovery.

The most recent development in Infusios cell therapy research is exosome therapy. This form of cell therapy uses stem cell messengers which are part of the so-called stem cell secrotomes. Exosomes are derived from placental mesenchymal stem cells the youngest form of stem cell lines available. They carry important anti-inflammatory proteins and so called genetic blueprints which they can insert into cells and thereby cause a regenerative effect. This advanced therapy may be recommended for patients that are severely immune deficient and want to avoid the liposuction procedure.

Regardless what form of cell therapy you receive, Infusio ensures an ideal foundation for healing by applying the Five Steps to Health and optimizing your body in advance, for the most effective impact of your cell therapy.

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Cell Therapy | Infusio.org

Stem cell therapy is an exciting area of research that holds tremendous potential for helping chronic pain patients reduce their pain. Rapid advances in this field of medicine are buoying doctors and patients hopes that the exciting therapy could change lives. Advances are being made nearly every day, and as scientists further understand the nature of these cells, even more uses for them are becoming known. If youve ever wondered about how stem cell therapy for pain could help you, these 15 questions delve into how this therapy works and who it could be used for.

Science has a strong interest in stem cells because of their renewing properties and the ability of these cells to develop into any type of tissue in the body of the organism. Researchers believe that they have the potential for immeasurable clinical uses in health. Research is advancing many stem cell based therapies for people who suffer from diseases such as:

Mayo Clinic explains how this therapy works:

Stem cell therapy, also known as regenerative medicine, promotes the reparative response of diseased, dysfunctional or injured tissue using stem cells or their derivativesResearchers grow stem cells in a lab. These stem cells are manipulated to specialize into specific types of cells, such as heart muscle cells, blood cells or nerve cells. The specialized cells can then be implanted into a person.

Stem cell therapy is actually a type of treatment within the larger umbrella of regenerative medicine. Our longer post on regenerative medicine discusses the types of treatments available, including stem cell therapy and platelet-rich plasma injections.

Stem cells are essentially blank canvases that can transform into any type of cell in the human body. Specialized cells like bone cells, liver cells, and heart cells begin as stem cells. The process of the cells transforming from blank slates into specialized cells is called differentiation.

Stem cell therapy is the process of injecting these cells into damaged areas of the body, such as arthritic knees or shoulders. The stem cells then differentiate into damaged tissue, helping to regenerate the entire area.

There are two different types of natural stem cells and one that is genetically reprogrammed within the laboratory. Embryonic stem cells are the most immature and are found within the early stages of a growing embryo, usually after it has been left to develop five to six days. After the egg and sperm have united, the fertilized egg divides and creates stem cells that differentiate into the specialized cells the body requires to function. Many techniques using stem cells for pain therapies no longer rely on these types of stem cells.

The second type of stem cell, found naturally in organisms, is adult stem cells. These are present in developed tissue, such as muscle, skin, bone, brain, and blood. Also called tissue stem cells, they can self-renew and generate one specialized cell type. Under normal circumstances these tissue stem cells, or adult stem cells, will generate the type of cells that make up the organ in which they reside. These cells are used by the body to divide and repair injured areas or regenerate into specialized cells to replace the ones that are dead or damaged.

The third type of stem cells, which are genetically reprogrammed in the laboratory, are induced pluripotent stem cells. After years of stem cell research and development, it was discovered that artificially triggering certain genetic components would prompt different cells to become pluripotent stem cells, which were similar in nature to embryonic stem cells. This helps avoid ethical concerns associated with using human embryonic tissue for research and regenerative medicine.

The following video from an Arizona pain doctor goes into detail about how stem cell therapy for pain works.

There are twomajor types of stem cells: those harvested from adults and those harvested from embryonic tissues.

However, there are other ways to retrieve stem cells that reduce the use of embryonic stem cells, including:

As the Euro Stem Cell organization reports, some of these types of stem cells are more effective than others.

One source of stem cells is human embryos. These cells are called pluripotent stem cells, and theyre very useful to researchers because they can be multiplied indefinitely in the laboratory. Although these cells are often cultivated from embryos that are just a few days old, they can also be taken from fetal tissue thats older than eight weeks, according to the National Institutes of Health (NIH).

The majority of therapeutic stem cells come from adults. Even though embryos are the richest source of stem cells, humans of all ages have stem cells. Stem cells give adults the ability to replace damaged tissue, heal wounds, and grow hair.The patients own adult stem cells are extracted, purified, concentrated, and then injected into the damaged tissue. This process is usually non-surgical and the individual has very little recovery time. Most patients report only some soreness around the site of the injection. Sometimes there is also slight bruising. There have been no reports of serious side effects from treatments using stem cell therapy.

Newer research has given scientists the ability to reprogram specialized adult cells so they essentially return to their original stem cell state. These reprogrammed cells are known as induced pluripotent stem cells. Although this ability exists, scientists arent sure how or if these artificially created stem cells behave differently than other types.

Despite these unknowns, the reprogrammed stem cells are already being used in the development of medicines and helping scientists learn more about specific types of diseases, according to NIH.

Stem cells have many uses, and the full spectrum of their application isnt yet known. One way stem cells are helping researchers is by illuminating the inner workings of various diseases. Stem cells offer scientists the ability to model human disease progression in a laboratory setting.

This is exciting because many studies rely on animals with similar, but not exact, biology to humans. The more scientists can understand about human-specific disease progression, the greater insight they have regarding potential treatments.

For example, one of the earliest uses for stem cells were bone marrow transplants, used to help patients with leukemia or sickle cell anemia heal. This treatment has been used for more than 40 years. In addition, stem cell therapy may be used to treat:

The application of most interest to chronicpatients is likely the emerging field of regenerative medicine, which is the science of helping tissues regenerate. This field examines the potential of stem cells to repair damaged tissue and heal areas of the body bone and potentially organs, too affected by arthritis, diabetes, spinal cord injuries, nerve damage, Parkinsons disease, and more.

Exciting research has also uncovered the potential for stem cells to expand the number of lungs available for transplant. A portion of lungs available for transplant arent used because they become damaged. However, research from the American Physiological Society has found stem cells could help repair the organs and prepare them to save lives.

Other recent researchstories include:

For many of the studies underway, time is needed to fully examine the benefits and potential dangers of this treatment. Another obstacle is obtaining specific types of adult stem cells. Theyre difficult to grow in the laboratory, making it hard to produce the large numbers available for research.

Another potential issue with donor stem cells is the possibility of rejection. The immune system of the recipient could reject the cells, essentially making it difficult for the treatment to work as intended and causing ancillary problems.

Finally, since this is such a new treatment area, some government agencies are calling for more oversight of its use. Others are pushing back, claiming that stem cell therapy provides a new area of treatment for patients who have exhausted all other options.

That being said, even though there are complications and roadblocks to its use, the benefits of stem cell therapy could be huge. As the American Academy of Anti-Aging Medicine notes:

[A]n analysis of the potential benefits of stem cells based therapies indicates that 128 million people in the United States alone may benefit with the largest impact on patients with Cardiovascular disorders (5.5 million), autoimmune disorders (35 million) and diabetes (16 million US patients and more than 217 million worldwide).

Californias Stem Cell Agency gives a great overview of this process, noting:

In order to be approved by the FDA for use in human trials, stem cells must be grown in good manufacturing practice (GMP) conditions. Under GMP standards, a cell line has to be manufactured so that each group of cells is grown in an identical, repeatable, sterile environment. This ensures that each batch of cells has the same properties, and each person getting a stem cell therapy gets an equivalent treatment. Although the FDA hasnt yet issued guidelines for how pluripotent stem cells need to meet GMP standards, achieving this level of consistency could mean knowing the exact identity and quantity of every component involved in growing the cells.

Stem cell therapy is being studied for a number of chronic pain conditions, especiallypain in the:

Stem cell therapy for pain could help reduce the inflammation that results in chronic pain, or it could help to heal regenerative conditions that lead to pain, such as arthritis.

Using stem cell therapy for knee pain is one of the leading areas of research. Stem cell therapy for knees can be provided as stem cell injections or as blood platelet treatments from the body itself (another form of regenerative medicine).These two treatments may help relieve pain associated with:

The leading researchers on stem cell therapy for knee pain claim that it can help patients avoid surgery, with its associated costs and risks.

Since stem cell therapy promises to treat a number of conditions related to degenerative conditions, like arthritis and tendonitis, stem cell therapy may present a great treat option for hip pain related to these causes.

The National Multiple Sclerosis Society is leading the efforts in research, but currently reports the following:

At present, there are no approved stem cell therapies for MS. Larger, longer-term, controlled studies are needed to determine the safety and effectiveness of using stem cells to treat MS. When the results of these and subsequent clinical trials are available, it should be possible to determine what the optimal cells, delivery methods, safety and actual effectiveness of these current experimental therapies might be for different people with MS.

Potentially. One of our sister clinics, Arizona Pain, is participating in a study evaluating the potential of stem cells to reduce back pain related to degenerative disc disease. This progressive condition sometimes results from injury, but other times has no clear cause.

The study is exciting because it involves stem cells harvested from the bone marrow of healthy, young adults, and therefore itdoesnt come with the ethical concerns of embryonic stem cells. So far, the results have been very positive, and a significant number of people who received stem cells for their back pain have experienced reduced discomfort and improved quality of life.

This study is currently in Phase III, which is the phase immediately preceding potential FDA approval. This means it could soon be available to many more patients and potentially covered by insurance, although each insurance companys coverage policy varies.

Absolutely, and the research into this area is very promising. In fact, scientists have recently uncovered the specific type of stem cell most likely to reduce arthritis pain. Theyre special cells that are specifically able to rebuild tissue, bone, and cartilage, potentially offering much relief to osteoarthritis patients.

What other questions do you have regarding stem cell therapy for pain? If youre ready to learn more about using stem cell therapy to treatyour pain, click the button below to find a pain specialist in your area.

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15 Stem Cell Therapy For Pain Questions, Answered

An ideal injectable therapy would combine safe long term anti-inflammatory pain management and long term regenerative healing. The Center for Nonsurgical & Regenerative Orthopedics now offers the most sophisticated and powerful Stem Cell product on the market. ReNu is the only stem cell injection that combines the anti-inflammatory capacity of amniotic tissue with the regenerative power of fetal stem cells.

ReNu is a revolutionary noninvasive treatment in which human amniotic tissue and stem cells are injected into injured tissue to provide relief from pain and stimulate the body's natural healing process. This amniotic tissue contains fetal mesenchymal stem cells that help to regenerate damaged tissue such as cartilage, tendons, and ligaments. The growth factors in the amniotic tissue provide anti-inflammatory, anti-adhesive, and anti-microbial properties to the injured area and can significantly help reduce scarring for quicker healing. Amniotic tissue is also immune privileged, which means there is virtually no chance that your body will reject the treatment.

ReNu stem cells come from the amniotic membrane and fluid of donated placentas following normal childbirth. With the mother's consent, the amnion is removed and harvested from the placenta of pre-screened and tested donors. It is then sterilely processed and undergoes further rigorous testing to assure safety and efficacy. To be clear, while some people may have ethical issues with embryonic stem cell therapy, almost everyone agrees that the use of amniotic stem cell therapy raises no ethical or moral questions.

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ReNu Stem Cell Therapy - Advanced Orthopedics - Nonsurgical ...

Many doctors and athletes use stem cell therapy to treat sports injuries, such as Achilles tendinopathy or damaged knee ligaments.

Some physicians use stem cells to repair damaged knee cartilage.Read Knee Cartilage Repair, Regeneration, and Replacement

While increasing in popularity, stem cell therapy is not considered standard practice by sports medicine doctors and not covered by most insurance companies. Patients considering paying out-of-pocket are advised to learn about stem cells potentialand limitationsfor treating sports injuries.

See Treating Acute Sports and Exercise Injuries in the First 24 to 72 Hours

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Doctors use stem cells to treat a wide variety of sports injuries, including damage to:

These injuries may be due to a one-time trauma or chronic overuse.

Stem cells can be applied to an injured area via:

See Regenerative Medicine for Sports Injuries

When administering injections, many physicians use ultrasound or other medical imaging to ensure cells are delivered precisely to the site of damaged tissue.

See Types of Regenerative Medicine for Sports Injuries

Stem cells are different than skin cells, muscle cells, liver cells, or any other human cells. What makes stem cells special is that they can:

Many physicians who use stem cell therapy hypothesize that, when placed into a certain environment, stem cells can transform to meet a certain need. For example, stem cells that are placed near damaged Achilles tendon are hypothesized to develop into healthy Achilles tendon cells.

In almost all cases, the stem cells used in sports medicine come from the patient. Clinical use of fetal or embryonic stem cells is banned in United States.

The process of collecting stem cells is often called harvesting. Physicians usually harvest stem cells from the patients fat, blood, or bone marrow.

Before a bone marrow aspiration, a patient is given a local anesthetic and may also be given a sedative.

Mesenchymal stem cellsAll three types of stem cells listed aboveadipose (fat), peripheral blood, and bone marrowbelong to a category of stem cells called mesenchymal stem cells. These stem cells, sometimes called adult stem cells, can be obtained from the patients own body and are being increasingly used for treating sports injuries.

See What Are Stem Cells? on Arthritis-health.com to learn more.

Right now, there are no formal medical guidelines regarding who can receive stem cell therapy for sports injuries. Whether or not to use stem cells for treatment is up to patients and their doctors. A lack of standardization further complicates the nature of treatment at one clinic versus another.

See Who Is a Candidate for Platelet-Rich Plasma Therapy?

Some doctors have certain criteria for recommending stem cell therapy. For example, they only recommend it to patients who are healthy and younger (e.g. under 50). Other doctors make recommendations on a case-by-case basis.

Read more:

Stem Cell Therapy for Sports Injuries