Experimental study speeds up bone healing with 2 common medications – New Atlas

A new proof-of-concept study has found a combination of two drugs, already approved by the FDA for other uses, may boost the release of stem cells from bone marrow and accelerate the healing of broken bones. Only demonstrated in animals at this stage, the researchers suggest clinical trials could progress rapidly considering the drugs have already been demonstrated as safe in humans.

"The body repairs itself all the time, says corresponding author on the study Sara Rankin. We know that when bones break they will heal, and this requires the activation of stem cells in the bone. However, when the damage is severe, there are limits to what the body can do of its own accord.

A great deal of current research is focusing on mesenchymal stem cell (MSC) therapies. MSCs are a type of adult stem cell that can grow into a variety of different cell types including muscle, fat or bone. Many current MSC treatments in development involve extracting a small number from a patient, growing them in laboratory conditions, then injecting them back into the patient.

The new research set out to investigate whether any currently approved drugs can function to mobilize the bodys natural ability in releasing MSCs, with a view on speeding up healing of bone fractures. A study published in the journal npj Regenerative Medicine, describes the testing of two already approved drugs in a rodent spinal injury model.

The two drugs tested were an immunostimulant called Plerixafor, used to stimulate the release of stem cells from bone marrow in cancer patients, and a beta-3 adrenergic agonist developed to help bladder control.

The results suggest the duo of drugs mobilize MSCs into the bloodstream and speed up the process of bone formation and healing by enhancing the binding of calcium to the injury site. Tariq Fellous, first author on the new study, suggests the next step is to investigate whether this drug combination enhances blood MSC levels in human subjects.

We first need to see if these medications release the stem cells in healthy volunteers, before we can then test them in patients with fractures, says Fellous. We have the drugs and know they are safe to use in humans - we just need the funding for the human trials.

The researchers say prior studies have identified circulating MSCs increase in volume following injuries such as burns, bone fractures, and even heart attack. The hypothesis is that the release of MSCs is a physiological process aiding general regeneration following injury, and if circulating numbers of MSCs could be pharmacologically enhanced then a variety of types of tissue regeneration could be accelerated.

It is important to note the current study only examined increases in circulating MSCs and the rate of spine injury healing compared to no drug treatment. The current research offers no indication whether the drug duo influences nerve healing or restores movement.

So, more work is certainly necessary to understand how clinically useful these results actually are. However, as the studys co-first author Andia Redpath notes, this re-purposing of existing medicines to boost stem cell activity is an easier, cheaper, and more efficient way to enhance healing compared to other, more complex and time-consuming, stem cell treatments in development.

Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the bodys own stem cells, using existing drugs, says Redpath. We already know the treatments in our study are safe, its now just a matter of exploring further if they help our bodies heal.

The new study was published in the journal npj Regenerative Medicine.

Source: Imperial College London

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Experimental study speeds up bone healing with 2 common medications - New Atlas

Transplant for Szary Syndrome is Patient’s First Step in Returning to the Dance Floor – Dana-Farber Cancer Institute

The first time Bill Cronin Googled his own cancer diagnosis in 2016, his heart sank. He had Szary syndrome, a rare and aggressive form of cutaneous T-cell lymphoma and staring back at him were countless articles predicting a negative prognosis.

However, after receiving a stem-cell transplant at Dana-Farber/Brigham and Womens Cancer Center, Cronin is returning to the life he enjoyed before cancer.

Im at a place I never thought Id get to, Cronin says.

In 2015, Cronin, then 60, started feeling incredibly itchy and developed an accompanying rash. He went to his dermatologist, who diagnosed him with eczema and told him to return in five months. The rash continued to grow, however, and at the five month mark, Cronins dermatologist encouraged him to undergo further testing at Dana-Farber.

A blood test revealed that Cronins T-cells a type ofwhite blood cells that make up part of the immune system had becomecancerous. In the case of Szary syndrome, lymphoma cells will circulatethrough the blood stream and deposit in different areas of the skin. This willgenerally lead to a full-body rash and intense itchiness.

Cronin would need a stem cell transplant to combat the disease, but before he could receive one, his care team had to get him into remission. Patients who do not achieve remission prior to transplant have a high chance of relapsing.

When they first told me everything, I was really scared, says Cronin. But I knew I was in one of the best places in the world to figure out and treat this rare disease.

Cronins pre-transplant care was spearheaded by oncologists David Fisher, MD, and Nicole LeBoeuf, MD, MPH, clinical director of Cutaneous Oncology at Dana-Farber, with his transplant conducted by Corey Cutler, MD, MPH, medical director of the Adult Stem Cell Transplantation Program at Dana-Farber. Initially, Cronins disease was incredibly resistant; for nearly three years, mainstay drugs including steroids, monoclonal antibodies, and enzyme blockers all failed to put his disease into remission.

Ultimately, it would take a new drug, mogamulizumab (a type of immunotherapy that directly kills T-cells involved with Sezary Syndrome) to get Cronins disease into remission.

In May 2019, Cronin was cleared to undergo an allogeneic transplant, a type of transplant that uses a donors stem cells, in this case, Cronins brother. Since his transplant Cronin has remained in remission.

We had to use all of our big guns to get him totransplant, but Im pleased with where we are now, says Cutler.

I know the situation can always change, but it was great tobe able to share some good news with my family and friends, adds Cronin.

Patients like Cronin serve as a reminder of how stem cell transplants have improved and continue to impact patient outcomes, Dana-Farber experts note. Initially offered to only an incredibly small patient population when first performed at Dana-Farber in the 1970s, research advancements have, and continue to, broaden who is eligible for a transplant. In 2019, Dana-Farber/Brigham and Womens Cancer Center (DF/BWCC) surpassed 10,000 total adult transplants.

This milestone indicates our success as a program and our volume has allowed us to do the research to help move the field forward rather impressively, says Joseph Antin, MD, chief emeritus of Adult Stem Cell Transplantation at DF/BWCC.

In 1996, Dana-Farber Cancer Institute and Brigham and Womens Hospital merged their then separate transplant centers. By pooling together physical and intellectual resources, the new combined program was able to more than double the number of transplants each hospital could perform individually.

We always felt collaboration was better than competition, explains Robert Soiffer, MD, vice chair of Medical Oncology for Hematological Malignancies and chief of the Division of Hematologic Malignancies, who oversaw the merger with Antin. Each side could learn from the other, and that helped to catapult us into the leadership position we have today.

The Stem Cell Transplantation Program is also bolstered by the Connell and OReilly Families Cell Manipulation Core Facility (CMCF), which was established in 1996. The state-of-the-art center, led by Jerome Ritz, MD, not only processes the stem cells for transplant; it also assists researchers in developing new cell-based therapies for patients.

Another key component to the programs success has been the creation of the Ted and Eileen Pasquarello Tissue Bank. The Pasquarello Tissue Bank receives, processes, banks, and distributes research samplesof blood, bone marrow, and other tissues. Through a database overseen by Vincent Ho, MD, the Institute is able to log, assess, and later review every patients disease, including all complications and mutations. This technology allows researchers to explore the genetic makeup of past donors and better understand why a transplant was or was not successful.

Were still learning from biological specimens we collected 20 years ago, and it will continue to impact care 20 years from now, Soiffer says.

Today, there is a continuous push to develop new and more precise therapies to complement and improve stem cell transplants. The hope is to bring new treatment options to patients like Cronin who are facing rare and difficult diseases.

Before his diagnosis, Bill, and Barbara Finney, his partner ofnearly 30 years, were avid English Country dancers. English Country dancingevolved from the court dances of Europe in the early 17th century, and Croninand Barbara have friends from all over the country who share their passion forit.

While Cronin isnt dancing just yet, as hes stillrecovering from his transplant, he says he couldnt have gotten through thiswithout his partner on the dance floor and in life.

Barbara has been amazing and has helped take care ofeverything I couldnt do, he adds. Ive been fortunate and privileged to notonly have her, but to have been able to come to Dana-Farber.

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Transplant for Szary Syndrome is Patient's First Step in Returning to the Dance Floor - Dana-Farber Cancer Institute

Animal Stem Cell Therapy Market 2020-2026: Product Types, by Applications, By Market Trends, Market Reserach Report – Keep Reading

Our latest research report entitle Global Animal Stem Cell Therapy Market provides comprehensive and deep insights into the market dynamics and growth of Global Animal Stem Cell Therapy Industry. Latest information on market risks, industry chain structure, Animal Stem Cell Therapy cost structure and opportunities are offered in this report. The entire industry is fragmented based on geographical regions, a wide range of applications and Global Animal Stem Cell Therapy Market types. The past, present and forecast market information will lead to investment feasibility by studying the crucial Global Animal Stem Cell Therapy Industry growth factors.

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Global Animal Stem Cell Therapy Market Analysis By Major Players:

VETSTEM BIOPHARMAMediVet BiologicJ-ARMCelavetMagellan Stem CellsU.S. Stem CellCells Power JapanANIMAL CELL THERAPIESAnimal Care StemCell Therapy SciencesVetCell TherapeuticsAnimacelAratana Therapeutics

Global Animal Stem Cell Therapy Market Analysis By Geographical Zones:

Europe Market (Germany, France, Italy, Russia and UK)

North America Market (Canada, USA and Mexico)

Latin America Market (Middle and Africa).

Animal Stem Cell Therapy Market in Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Asia-Pacific Market (South-east Asia, China, India, Korea and Japan).

Global Animal Stem Cell Therapy Market news, plans & policies, market drivers, analysis of upstream raw material suppliers and downstream buyers of Animal Stem Cell Therapy is carried out in this report. Global Animal Stem Cell Therapy Industry forecast study enlists the market value (in USD) and volume forecast for each region, product type, and applications.

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DogsHorsesOthers

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Veterinary HospitalsResearch Organizations

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Major points from Table of Contents for Global Animal Stem Cell Therapy Industry 2020 Market Research Report include:

1 Market Overview

2 Global Animal Stem Cell Therapy Market Competition by Manufacturers

3 Global Animal Stem Cell Therapy Capacity, Production, Revenue (Value) by Region (2020-2026)

4 Global Animal Stem Cell Therapy Industry Supply (Production), Consumption, Export, Import by Region (2020-2026)

5 Global Animal Stem Cell Therapy Production, Revenue (Value), Price Trend by Type

6 Global Animal Stem Cell Therapy Market Analysis by Application

7 Global Animal Stem Cell Therapy Industry Manufacturers Profiles/Analysis

8. Animal Stem Cell Therapy Manufacturing Cost Analysis

9 Industrial Chain, Sourcing Strategy and Downstream Buyers

10 Marketing Strategy Analysis, Distributors/Traders

11 Market Effect Factors Analysis

12 Global Animal Stem Cell Therapy Market Forecast (2020-2026)

13 Research Findings and Conclusion

14 Appendix

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Animal Stem Cell Therapy Market 2020-2026: Product Types, by Applications, By Market Trends, Market Reserach Report - Keep Reading

34 years with a new heart and counting – MDJOnline.com

Whenever Harry Wuest has a doctors appointment in northern Atlantas hospital cluster dubbed Pill Hill, he makes sure to stop by the office of Dr. Douglas Doug Murphy for a quick chat.

And Murphy, unless hes tied up in the operating room, always takes a few minutes to say hello to his former patient. Remember when ... ? is how the conversation typically starts, and its always tinged with laughter, often joyful, sometimes bittersweet.

Its a reunion of two men who shaped a piece of Georgias medical history.

Almost 35 years ago, Murphy opened the chest of Wuest and sewed in a new heart, giving him a second shot at life. Wuest was the third heart transplant patient at Emory University Hospital.

Tall, lanky, with short curly hair and a quiet demeanor, Wuest is the longest-surviving heart transplant recipient in Georgia and one of the longest-surviving in the world. The 75-year-old accountant still plays golf twice a week and only recently went from working full-time to part-time. My heart is doing just fine, he says.

Murphy is now the chief of cardiothoracic surgery at Emory Saint Josephs Hospital and still in the operating room almost every day. He has moved on to become the worlds leading expert in robotically assisted heart surgery.

Harry Wuest is originally from Long Island, New York. After a stint in the Air Force, he moved to Florida to work and go to school. He wanted to become a physical education teacher. Then, in 1973, he fell ill. It started with some pain on his left side. He didnt think much of it, but when he got increasingly winded and fatigued, he went to see a doctor.

Several months and numerous specialists later, he received the diagnosis: Cardiomyopathy, a disease of the heart muscle that can make the heart become enlarged, thick and rigid, preventing it from pumping enough blood through the body.

They didnt know how I got it, says Wuest, sitting back in a brown leather armchair in the dark, wood-paneled living room of his Stone Mountain home. Maybe it was a virus. And back then, there wasnt much they could do to treat it, except bed rest.

For the next 12 years, Wuest lived life as best as he could. He got a degree in accounting from the University of Central Florida and worked for a real estate developer. There were good days, but there were more bad days. He was often too weak to do anything, and his heart was getting bigger and bigger.

Emorys first transplant surgeon

The first successful human-to-human heart transplant was performed in Cape Town, South Africa, in 1967 a medical breakthrough that catapulted the surgeon, Dr. Christiaan Barnard, onto the cover of Life magazine and to overnight celebrity status.

This highly publicized event was followed by a brief surge in the procedure around the world, but overall, heart transplants had a rocky start. Most patients died shortly after the surgery, mainly due to organ rejection. Back then, immunosuppressive drugs, which can counteract rejection, were still in their infancy. Many hospitals stopped doing heart transplants in the 1970s.

That changed with the discovery of a highly effective immunosuppressive agent. Cyclosporine got FDA approval in 1983 and altered the world of organ transplants.

It was shortly thereafter when Emory University Hospital decided to launch a heart transplant program, but none of the senior surgeons wanted to do it. Even with the new drug, it was a risky surgery, and mortality was still high.

Its an all-or-nothing operation, Murphy says, as he sits down in his small office overlooking the grayish hospital compound. Hes wearing light blue scrubs from an early morning surgery. At 70, he still has boyish looks, with a lean build and an air of laid-back confidence. If you have a number of bad outcomes initially, it can be detrimental to your career as a surgeon, he says.

But Murphy didnt really have a choice. He remembers that during a meeting of Emorys cardiac surgeons in 1984, he was paged to check on a patient. When he returned, the physicians congratulated him on being appointed the head of the new heart transplant program. He was the youngest in the group and had been recruited from Harvards Massachusetts General Hospital just three years before.

Yeah, thats how I became Emorys first transplant surgeon, says Murphy.

He flew to California to shadow his colleagues at Stanford University Hospital, where most heart transplants were performed at the time. Back home at Emory, he put together a team and rigorously rehearsed the operation. The first transplant patient arrived in April 1985. The surgery was successful, as was the second operation less than a month later.

Around the same time, Harry Wuest wound up in a hospital in Orlando. He needed a transplant, but none of the medical centers in Florida offered the procedure. One of his doctors recommended Emory, and Wuest agreed. I knew I was dying. I could feel it. He was flown to Atlanta by air ambulance and spent several weeks in Emorys cardiac care unit until the evening of May 23, when Murphy walked into his room and said, Weve got a heart.

I could finally breathe again

The heart, as the patient later learned, came from a 19-year-old sophomore at Georgia Tech who had been killed in a car crash.

Organ transplants are a meticulously choreographed endeavor, where timing, coordination and logistics are key. While Murphy and his eight-member team were preparing for the surgery, Wuest was getting ready to say farewell to his family his wife and three teenage sons, and to thank the staff in the cardiac ward.

I was afraid, he recalls, especially of the anesthesia. It scared the heck out of me. He pauses during the reminiscence, choking briefly. I didnt know if I was going to wake up again.

The surgery took six hours. Transplants usually happen at night because the procurement team, the surgeons who retrieve different organs from the donor, only start working when regularly scheduled patients are out of the operating room.

Despite the cultural mystique surrounding the heart as the seat of life, Murphy says that during a transplant surgery, its not like the big spirit comes down to the operating room. Its very technical. As the team follows a precise routine, emotions are kept outside the door. We dont have time for that. Emotions come later.

Waking up from the anesthesia, Wuests first coherent memory was of Murphy entering the room and saying to a nurse, Lets turn on the TV, so Harry can watch some sports.

Wuest spent the next nine days in the ICU, and three more weeks in the hospital ward. In the beginning, he could barely stand up or walk, because he had been bedridden weeks before the surgery and had lost a lot of muscle. But his strength came back quickly. I could finally breathe again, he says. Before the surgery, he felt like he was sucking in air through a tiny straw. I cannot tell you what an amazing feeling that was to suddenly breathe so easily.

Joane Goodroe was the head nurse at Emorys cardiovascular post-op floor back then. When she first met Wuest before the surgery, she recalls him lying in bed and being very, very sick. When she and the other nurses finally saw him stand up and move around, he was a whole different person.

In the early days of Emorys heart transplant program, physicians, nurses and patients were a particularly close-knit group, remembers Goodroe, whos been a nurse for 42 years and now runs a health care consulting firm. There were a lot of firsts for all of us, and we all learned from each other, she said.

Wuest developed friendships with four other early transplant patients at Emory, and he has outlived them all.

When he left the hospital, equipped with a new heart and a fresh hunger for life, Wuest made some radical changes. He decided not to return to Florida but stay in Atlanta. Thats where he felt he got the best care, and where he had found a personal support network. And he got a divorce. Four months after the operation, he went back to working full-time: first in temporary jobs and eventually for a property management company.

After having been sick for 12 years, I was just so excited to be able to work for eight hours a day, he recalls. That was a big, big deal for me.

At 50, he went back to school to get his CPA license. He also found new love.

Martha was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.

Harry Wuest and his wife, Martha. She was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.

Having worked in the transplant office, I saw the good and the bad, Martha Wuest says. A petite woman with short, perfectly groomed silver hair, she sits up very straight on the couch, her small hands folded in her lap. Not every transplant patient did as well as Harry. And I had a lot of fear in the beginning. Now he may well outlive her, she says with a smile and a wink.

Wuests surgeon, meanwhile, went on to fight his own battles. Two and a half years into the program, Murphy was still the only transplant surgeon at Emory and on call to operate whenever a heart became available. Frustrated and exhausted, he quit his position at Emory and signed up with Saint Josephs (which at the time was not part of the Emory system) and started a heart transplant program there.

At St. Josephs, Murphy continued transplanting hearts until 2005. In total, he did more than 200 such surgeries.

Being a heart transplant surgeon is a grueling profession, he says, and very much a younger surgeons subspecialty.

He then shifted his focus and became a pioneer in robotically assisted heart surgery. He has done more than 3,000 operations with the robot, mostly mitral valve repairs and replacements more than any other cardiac surgeon in the world.

Heart transplants "remain the gold standard"

Since Murphy sewed a new heart into Wuest 35 years ago, there has been major progress in the field of heart transplants, but it has been uneven.

There is improved medication to prevent rejection of the donor heart, as well as new methods of preserving and transporting donor hearts.

Yet patients requiring late-stage heart failure therapy, including transplantation, still exceed the number of donor hearts available. In 2019, 3,551 hearts were transplanted in the United States, according to the national Organ Procurement and Transplantation Network. But 700,000 people suffer from advanced heart failure, says the American Heart Association.

New technologies and continued research are providing hope to many of these patients. There has been significant progress in the development of partial artificial hearts, known as Left Ventricular Assist Devices, or LVADs. They can be used as bridge devices, to keep patients alive until donor hearts are available, or as destination therapy, maintaining patients for the remainder of their lives.

Also, total artificial hearts have come a long way since the first artificial pump was implanted in a patient in 1969. The technology is promising, says Dr. Mani Daneshmand, the director of Emorys Heart & Lung Transplantation Program. But its not perfect.

Long-term research continues into xenotransplantation, which involves transplanting animal cells, tissues and organs into human recipients.

Regenerative stem cell therapy is an experimental concept where stem cell injections stimulate the heart to replace the rigid scar tissue with tissue that resumes contraction, allowing for the damaged heart to heal itself after a heart attack or other cardiac disease. Certain stem cell therapies have shown to reverse the damage to the heart by 30 to 50 percent, says Dr. Joshua Hare, a heart transplant surgeon and the director of the Interdisciplinary Stem Cell Institute at the University of Miamis Miller School of Medicine.

All of these ideas have potential, says Daneshmand. But none of them are ready to replace a human donor heart. A heart transplant remains the gold standard, because you cant accommodate the same success with a machine right now, he says.

Efforts around expanding the donor pool are really the best way to address this problem, while we wait for technology to catch up, he adds.

Besides Emory, other health care systems in Georgia that currently have a heart transplant program are Piedmont Healthcare, Childrens Healthcare of Atlanta and Augusta University Health.

Organ rejection remains a major issue, and long-term survival rates have not improved dramatically over the past 35 years. The 10-year survival is currently around 55 percent of patients, which makes long-term survivors like Harry Wuest rare in the world of heart transplants.

The United Network of Organ Sharing, or UNOS, which allocates donor hearts in the United States, doesnt have comprehensive data prior to 1987. An informal survey of the 20 highest-volume hospitals for heart transplants in the 1980s found only a scattering of long-term survivors.

In for the long haul

Being one of the longest-living heart transplant recipients is something that Wuest sees as a responsibility to other transplant patients, but also to the donors family, which hes never met. If you as a transplant recipient reject that heart, thats like a second loss for that family.

Part of this responsibility is living a full and active life. Both he and Martha have three children from their previous marriages and combined they have 15 grandchildren. Most of their families live in Florida, so they travel back and forth frequently. Wuest still works as a CPA during tax season, and he does advocacy for the Georgia Transplant Foundation. In addition to golf, he enjoys lifting weights and riding his bike.

Hes had some health scares over the years. In 2013, he was diagnosed with stage 1 kidney cancer, which is in remission. Also, he crossed paths with his former surgeon, and not just socially. In 2014, Murphy replaced a damaged tricuspid valve in Wuests new heart. That operation went well, too.

Murphy says there are several reasons why Wuest has survived so long. Obviously, his new heart was a very good match. But a patient can have the best heart and the best care and the best medicines and still die a few months or years after the transplantation, the surgeon says. Attitude plays a key role.

Wuest was psychologically stable and never suffered from depression or anxiety, Murphy says. Hes a numbers guy. He knew the transplant was his only chance, and he was set to pursue it.

Wuest attributes his longevity to a good strong heart from his donor; good genetics; great doctors and nurses; and a life that he loves. Im just happy to be here, he says.

Quoting his former surgeon and friend, he adds: Doug always said, Having a transplant is like running a marathon. And Im in for the long haul.

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34 years with a new heart and counting - MDJOnline.com

Lineage Cell Therapeutics to Present New Data From OpRegen and Vision Restoration Programs at the Association for Research in Vision and Ophthalmology…

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, announced today that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (dry AMD), have been accepted for presentation at the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting, which will be held May 3rd through May 7th, 2020 at the Baltimore Convention Center in Baltimore, MD. The abstract presentation, entitled, Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results, will be presented as part of the Gene Therapy and Stem cells Session on May 3rd, 2020 from 3:00PM to 4:45PM EDT by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute and University of Cincinnati School of Medicine; Clinical Governance Board, Cincinnati Eye Institute (presentation number 865). The presentation will provide updated data from patient cohorts 1 through 4 of the clinical study and will include data on the first patients dosed with both a new subretinal delivery system as well as with a new Thaw-and-Inject (TAI) formulation of OpRegen.

We continue to be encouraged by positive data with OpRegen for the treatment of dry AMD, stated Brian M. Culley, CEO of Lineage. The five patients treated as part of cohort 4, which more closely match our intended patient population, have all demonstrated an increase in the number of letters they can read on an Early Treatment Diabetic Retinopathy Scale (ETDRS), having gained between 10 25 letters. Importantly, the first patient treated using both a new subretinal delivery system and our TAI formulation of OpRegen demonstrated notable improvements in vision, having gained 25 readable letters (or 5 lines) 6 months following administration of OpRegen RPE cells, as assessed by the ETDRS. This represents an improvement in visual acuity from a baseline of 20/250 to 20/100 in the treated eye. These visual acuity measurements are meaningful and can translate into quality of life enhancements to things like reading, driving, or avoiding accidents. With the opening of two leading ophthalmology research centers as clinical sites for our study, we are focused on rapid enrollment so that our clinical update at ARVO can be as mature and informative as possible. Our objective is to combine the best cells, the best production process and the best delivery system, which we believe will position us as the front-runner in the race to address the unmet opportunity in the potential billion-dollar dry AMD market.

In addition, Lineage will present new preclinical results from its Vision Restoration Program, a proprietary program based on the ability to generate 3-dimensional human retinal tissue derived from pluripotent cells. Lineages 3-dimensional retinal tissue technology may address the unmet need of implementing a retinal tissue restoration strategy to address a wide range of severe retinal degenerative conditions including retinitis pigmentosa and advanced forms of AMD. In 2017 and 2019, the Small Business Innovation Research program of the National Institutes of Health awarded Lineage grants of close to $2.3 million to further develop this innovative, next generation vision restoration program.

- The poster presentation, entitled, Transplantation of organoid-derived human retinal tissue in to the subretinal space of CrxRdy/+ cats), will be presented as part of the Animal models for visual disease and restoration Session on May 4th, 2020 4:00PM to 5:45PM EDT in Session Number 291 by Igor Nasonkin, Ph.D., Principal Investigator, Director of Research & Development at Lineage (Poster board Number: 2253 - B0162).

- The poster presentation, entitled, Intraocular biocompatibility of Hystem hydrogel for delivery of pharmaceutical agents and cells, will be presented as part of the Stem cells and organoids: Technical advances Session on May 5th, 2020 between 8:45AM to 10:30AM EDT in Session Number 332 by our collaborator Randolph D. Glickman, Ph.D., Professor of Ophthalmology, UT Health San Antonio (Poster board Number: # A0247).

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the potential applications in Lineages Vision Restoration Program. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Lineage Cell Therapeutics to Present New Data From OpRegen and Vision Restoration Programs at the Association for Research in Vision and Ophthalmology...

6 Healthcare Stocks That Could Make You Filthy Rich – The Motley Fool

Picking home-run stocks -- or stocks capable of generating exponential returns on capital -- is no easy matter. That being said, there has arguably never been a more favorable environment for risk-tolerant investors on the hunt for unusual growth opportunities.

The fact of the matter is that the highly diversehealthcaresector has been churning out jaw-dropping returns on capital for the better part of the last decade.Companies like Axsome Therapeutics, for instance, have made a shocking amount of money for their early shareholders in an exceedingly short period of time. With this theme in mind, here is a look at six healthcare stocks that could make their early-bird investors filthy rich by the end of the current decade.

Image source: Getty Images.

Adaptimmune Therapeutics (NASDAQ:ADAP), a clinical-stage cancer immunotherapy company, has already gapped up by a staggering 235% so far this year. Investors have been taking notice of this small-cap biotech stock for two reasons. First off, Adaptimmune recently shored up its long-term financial health through a much-needed capital raise. Secondly, the biotech inked a lucrative partnering deal with the Japanese pharmaceutical giantAstellas to co-developnew stem-cell derived allogeneic T-cell therapies for people with cancer.

The main attraction here is the growing potential for Adaptimmune'saffinity-enhanced T-cell therapies to become a huge leap forward in the relatively new field of anti-cancer cell therapy. So, if that line holds, this small-cap biotech could rocket to large-cap status within just a matter of months. Adaptimmune's first major test will come with the next clinical update for its synovial sarcoma treatmentADP-A2M4. A positive readout should trigger more high-dollar licensing deals or perhaps even a buyout at a sky-high premium.

After a blistering 2019, Amarin (NASDAQ:AMRN), a mid-cap cardiovascular care company, has been a huge let down for investors during the early innings of 2020. The big-ticket item is the company's omega-3 treatment Vascepa, which was recently awarded a broader label as an add-on to statin therapy in patients with stubbornly high triglyceride levels and who are at risk of a heart attack, stroke, and certain types of heart issues requiring hospitalization.

Long story short, some investors bought the stock on the hope and belief that a high-dollar buyout was imminent in the wake of Vascepa's successful label expansion. Vascepa, after all, has a shot at one day posting multi-billion-dollar-a-year sales. Unfortunately, a buyout has yet to materialize.

But that doesn't mean that investors should throw in the towel. In the not-so-distant future, most industry insiders do expect Vascepa to at least reach blockbuster status (greater than $1 billion in annual sales). So, whether or not a suitor ever emerges, Amarin's equity should turn out to be a big winner for patient shareholders.

Canadian pot titan Aphria (NYSE:APHA) has also been a big disappointment for investors in 2020. Through no fault of its own, Aphria's shares have shed more than 17.4% of their value during the first six weeks of the new year. The key issue weighing down the pot company's stock is the dismal near-term outlook for the legal cannabis space at large. Canada's legal pot market has suffered from structural and regulatory setbacks, as well as the slow pace of legalization in the international arena. Nonetheless, Aphria could turn out to be a hidden gem.

Unlike most of its cannabis peers, Aphria has been able to attractdeep-pocketed investors this year, it has been turning a profit on a consistent basis, and it also has a fairly healthy cash runway. So, even though 2020 will probably be another tough year for pot stocks in general, Aphria's low-ball valuation and various competitive advantages over the broader field arguably make it one of the few compelling buys in this space right now. In fact, it wouldn't be surprising if Aphria morphed into a blue-chip company by the end of the decade.

The U.K. based drugmaker Bicycle Therapeuticsplc (NASDAQ:BCYC) has all the hallmarks of a hidden gem. The company's proprietary bicyclic peptide technology, or Bicycle for short, has the potential to push the field of immuno-oncology to new heights within a few short years. That being said, Bicycle is a highly speculative bet at this early stage of the game.

Point blank, the company's novel anti-cancer platform has yet to be validated via a large dataset in human subjects. Bicycle's highly differentiated immuno-oncology pipeline, though, is just the type of drug development platform that tends to attract blue-chip partners. As a result, investors comfortable with heavy doses of risk may want to get in on the ground floor of this unique immunotherapy company. After all, Bicycle's stock will surely catch fire at some point if its bicyclic peptide technology continues to hit the mark in the clinic.

Novavax (NASDAQ:NVAX) is a late-stage vaccine developer. Although the biotech's shares have been getting a boost from the COVID-19 threat of late, the real reason to consider this small-cap stock is the company's experimental flu vaccine known as NanoFlu. NanoFlu has the potential to reach blockbuster status perhaps within its first full year on the market due to the dire need for more effective flu vaccines. The company is expected to release the vaccine's pivotal stage results by the end of the first quarter.

What's the risk? Novavax has repeatedly rolled out promising early-to-mid-stage results for its vaccine development platform. However, the company has a poor track record when it comes to late-stage trials, which is likely the underlying reason its current valuation stands at a tiny fraction of NanoFlu's commercial opportunity right now.

In effect, the market doesn't have much confidence that Novavax will hit pay dirt with NanoFlu. So, if the company does prove its critics wrong this time around, its shares could quite possibly quadruple in value in the blink of an eye. Again, though, the high degree of risk with this name shouldn't be ignored. This clinical-stage biotech stock is basically an all-or-nothing value proposition at this critical juncture in its lifecycle.

Puma Biotechnology (NASDAQ:PBYI), a small-cap cancer specialist, has been blazing the comeback trail in 2020. The drugmaker's shares have gained a healthy 40% so far this year due to the growing optimism surrounding the commercial prospects of its much-maligned breast cancer treatmentNerlynx.Nerlynx's real-world commercial opportunity has basically been shrouded in mystery since its launch. The core issue is that the drug tends to produce severe bouts ofdiarrhea, which has led to uncomfortably high discontinuation rates among patients. The drug's sales, in turn, have consistently missed Wall Street's consensus estimates by a country mile ever since its commercial debut.

The market's extremely pessimistic take on this biotech stock, though, appears to be overdone at this point. Puma's shares are now trading at a dirt cheap price-to-sales ratio of 1.71. Most cancer specialists are currently garnering premiums that are four to five times higher. Puma's stock may ultimately fail to launch due to Nerlynx's problematic side effect profile.But there's also a decent chance that the biotech's clearance-rack valuation might attract one or more suitors before year's end. This grossly undervalued biotech could thus turn out to be the comeback story of the year.

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6 Healthcare Stocks That Could Make You Filthy Rich - The Motley Fool

Red Cross and American Cancer Society Partnering to Help Cancer Patients – WLTZ 38 NBC

Patients fighting cancer need more blood than patients fighting any other disease, using nearly one-quarter of the nations blood supply. Thats why this February, the American Red Cross and the American Cancer Society have teamed up to encourage people across the country to Give Blood to Give Time, ensuring loved ones have the strength and support to battle cancer.

According to the American Cancer Society, 1 in 3 people in the U.S. will be diagnosed with cancer in their lifetime. An estimated 28,570 new cases of cancer will be diagnosed in Alabama this year. Many of these people will likely have a need for blood.

A loved ones cancer diagnosis often makes families and friends feel helpless. Thats why the Give Blood to Give Time partnership with the American Cancer Society is so important, said Dr. Pampee Young, chief medical officer, American Red Cross. When someone donates blood or platelets or makes a financial gift, they are helping to give patients and their families time, resources and the hope they need to fight back.

To schedule a blood or platelet donation appointment or make a financial gift, visit GiveBloodToGiveTime.org.

Some types of chemotherapy can damage bone marrow, reducing red blood cell and platelet production. Other times, the cancer itself or surgical procedures cause the problem. Blood products are often needed. In fact, five units of blood are needed every minute to help someone going through cancer treatment. Yet only 3% of people in the United States give blood. More people are needed to donate regularly to help meet the need.

The need for blood in cancer treatments is an important and untold story, said Gary Reedy, chief executive officer of the American Cancer Society. The American Cancer Society is excited to be working with the Red Cross on Give Blood to Give Time. Through this partnership, we want people to know there are multiple ways they can help and make a meaningful difference in the lives of patients and their families.

Individuals can honor their loved ones by making a blood donation appointment or financial contribution at GiveBloodToGiveTime.org.

Who blood donations help

In April 2015, Stephenie Perry was diagnosed with Hodgkin lymphoma exactly 10 days before her commencement ceremony from graduate school. One week after commencement, treatments began. During chemotherapy and two stem cell transplants, she needed blood and platelet transfusions.

I dont even know how many units of blood Ive received, she said. But I know all of that blood came from selfless individuals who made the decision to make an appointment and donate.

Last February, Perry received the good news that her cancer is in remission. The next day, her then-boyfriend, Justin Perry, proposed to her just in time for Valentines Day. They talked about marriage while she battled cancer, but they wanted to wait until she was in remission. Justin Perry said he didnt want to go another day without asking her to marry him. They were married in September.

Her red blood cell counts still get low at times. When that happens, she goes in for another blood transfusion.

I think its safe to say that my successful battle with cancer depended upon complete strangers and their donated blood. For this, I am grateful, Stephenie Perry said. Sometimes I hear stories from friends about people who are scared of needles or afraid to donate blood. I wish I could stand face-to-face with those people and tell them there is nothing scary about saving a life a life like mine.

Courtesy: American Red Cross

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Red Cross and American Cancer Society Partnering to Help Cancer Patients - WLTZ 38 NBC

Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD – Yahoo Finance

Patient Analyses and Safety Data Continue to Underscore Positive Impact of KD025 in cGVHD

Pre-NDA Meeting with FDA Planned for March 2020; Topline Results of Primary Analysis to be Announced in Q2 2020

NEW YORK, NY / ACCESSWIRE / February 23, 2020 / Kadmon Holdings, Inc. (KDMN) today announced expanded results from the previously reported interim analysis of ROCKstar (KD025-213), its ongoing pivotal trial of KD025 in chronic graft-versus-host disease (cGVHD). The data were presented today in the oral latebreaker session at the 2020 Transplantation & Cellular Therapy (TCT) Meetings.

As announced in November 2019, KD025 met the primary endpoint of Overall Response Rate (ORR) at the study's planned interim analysis, two months after completion of enrollment. KD025 showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once daily (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (95% CI: 54%, 78%; p<0.0001). In the expanded KD025-213 dataset presented today, ORRs were consistent with the previously reported interim analysis across key subgroups, including in patients with four or more organs affected by cGVHD (n=69; 64%), patients who had prior treatment with ibrutinib (n=45; 62%) and patients who had prior treatment with ruxolitinib (n=37; 62%). Three patients achieved a Complete Response. Responses were observed in all affected organ systems, including in organs with fibrotic disease. KD025 has been well tolerated: adverse events were consistent overall with those expected to be observed in cGVHD patients receiving corticosteroids, and no apparent increased risk of infection was observed. Additional secondary endpoints, including duration of response, corticosteroid dose reductions, Failure-Free Survival, Overall Survival and Lee Symptom Scale reductions continue to mature and will be available later in 2020.

"KD025 has been well tolerated and has already demonstrated high response rates in patients with severe and complex cGVHD after a median of five months of follow-up," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and a KD025-213 study investigator and Steering Committee member.

"We are extremely pleased with the interim outcomes of this pivotal trial of KD025 in cGVHD, which track closely our findings from our earlier Phase 2 study. KD025 achieved robust response rates across all subgroups of this difficult-to-treat patient population, who had a median of four prior lines of therapy, and 73% of whom had no response to their last line of treatment," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We plan to meet with the FDA for a pre-NDA meeting in March 2020 and to announce topline results from the primary analysis of this trial in Q2 2020."

At the TCT Meetings, Kadmon also presented long-term follow-up data from KD025-208, its ongoing Phase 2 study of KD025 in cGVHD (Abstract #15205). These data were recently presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in December 2019.

About the ROCKstar (KD025-213) Trial

KD025-213 is an ongoing open-label trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg once daily or KD025 200 mg twice daily, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%.

While the ORR endpoint was met at the interim analysis, which was conducted as scheduled two months after completion of enrollment, topline data from the primary analysis of the KD025-213 study, six months after completion of enrollment, will be reported in Q2 2020. Full data from the primary analysis will be submitted for presentation at an upcoming scientific meeting.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates immune response as well as fibrotic pathways. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). KD025 was granted Breakthrough Therapy Designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy.

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About cGVHD

cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.

About Kadmon

Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Our clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.

Forward Looking Statements

This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates; (iv) the timing or likelihood of regulatory filings and approvals; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe, China, Japan and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel (xx) the potential benefits from any of our product candidates being granted orphan drug or breakthrough designation; (xxi) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; and/or (xxii) other risks and uncertainties. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the U.S. Securities and Exchange Commission (the "SEC"), including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and subsequent Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

Contact Information

Ellen Cavaleri, Investor Relations646.490.2989ellen.cavaleri@kadmon.com

SOURCE: Kadmon Holdings, Inc.

View source version on accesswire.com: https://www.accesswire.com/577466/Kadmon-Announces-Expanded-Results-of-Interim-Analysis-of-Pivotal-Trial-of-KD025-in-cGVHD

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Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD - Yahoo Finance

bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress – Yahoo Finance

- First conditional approval of ZYNTEGLOTM (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent -thalassemia who do not have 0/0 genotype in Europe achieved in 2019; Germany launch underway

- Announced positive top-line data from pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma

- Presented clinical data across studies of LentiGlobin gene therapy for -thalassemia (betibeglogene autotemcel) and LentiGlobin gene therapy for sickle cell disease (SCD) and bb21217 in multiple myeloma at American Society of Hematology (ASH) Annual Meeting

- Ended quarter with $1.24 billion in cash, cash equivalents and marketable securities

bluebird bio, Inc. (NASDAQ: BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2019.

"2019 was truly a transformative year for bluebird, with our first commercial product now launched in Europe and exciting progress across our first four clinical programs and pipeline," said Nick Leschly, chief bluebird. "Notably, our data in SCD continues to build, and at the ASH annual meeting in December we presented data that showed a 99% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in HGB-206 Group C patients with history of VOCs and ACS who had at least six months follow-up. In -thalassemia, the consistency with which patients who do not have a 0/0 genotype in our Northstar-2 (HGB-207) study are achieving transfusion independence is very encouraging and were starting to see indications that we may be able to see similar outcomes with many patients with 0/0 genotypes as well in our Northstar-3 (HGB-212 study). These data put us in a strong position as we progress our European launch, currently underway in Germany. At the end of 2019, we also announced positive top-line data from the pivotal KarMMa study of ide-cel. We and our partners at BMS look forward to submitting these data to the FDA in the first half of this year. Amidst all of our progress in 2019, our birds demonstrated time and again their dedication to patients and ability to meet and learn from the many challenges we have faced along the way. I look forward to facing the challenges of 2020 with this amazing flock."

Recent Highlights:

TRANSFUSION-DEPENDENT -THALASSEMIA

SICKLE CELL DISEASE (SCD)

MULTIPLE MYELOMA

COMPANY

Upcoming Anticipated Milestones:

Fourth Quarter and Full Year 2019 Financial Results

LentiGlobin for -thalassemia Safety

Non-serious adverse events (AEs) observed during the HGB-204, HGB-207 and HGB-212 clinical studies that were attributed to LentiGlobin for -thalassemia were hot flush, dyspnoea, abdominal pain, pain in extremities, thrombocytopenia, leukopenia, neutropenia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

With more than five years of follow-up to date, there have been no new unexpected safety events, no deaths, no graft failure and no cases of vector-mediated replication competent lentivirus or clonal dominance. In addition, there have been no new reports of veno-occlusive liver disease (VOD) as of the data cutoff presented at ASH.

About LentiGlobin for -Thalassemia (betibeglogene autotemcel)

The European Commission granted conditional marketing authorization for LentiGlobin for -thalassemia, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

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LentiGlobin for -thalassemia adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

bluebird bio has initiated its rolling BLA submission of LentiGlobin for -thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for -thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for -thalassemia. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the plans for regulatory submissions and commercialization for ZYNTEGLO and the companys product candidates, including anticipated regulatory milestones, the execution of the companys commercial launch plans, planned clinical studies, as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or future clinical trials; the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product, in the adoption of value-based payment models, or in obtaining sufficient coverage or reimbursement for our products; the risk that our collaborations, including the collaborations with Bristol-Myers Squibb and Forty Seven, will not continue or will not be successful; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

bluebird bio, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except per share data)(unaudited)

For the three months endedDecember 31,

For the year endedDecember 31,

2019

2018

2019

2018

Revenue:

Collaboration revenue

$ 7,159

$ 18,382

$ 36,469

$ 52,353

License and royalty revenue

2,838

861

8,205

2,226

Total revenues

9,997

19,243

44,674

54,579

Operating expenses:

Research and development

161,821

119,722

582,413

448,589

Selling, general and administrative

76,202

53,508

271,362

174,129

Cost of license and royalty revenue

1,073

818

2,978

885

Change in fair value of contingent consideration

1,435

2,156

2,747

2,999

Total operating expenses

240,531

176,204

859,500

626,602

Loss from operations

(230,534)

(156,961)

(814,826)

(572,023)

Interest income, net

6,855

6,209

34,761

14,624

Other (expense) income, net

535

1,916

(10,088)

1,961

Link:

bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress - Yahoo Finance

Michael Schumacher reportedly underwent breakthrough treatment; Here are details of the secret procedure – EconoTimes

Michael Schumacher turned 51 in January and as he became a year older, his fans are hoping that he is doing fine. Apparently, fans did not stop supporting him even if he not visible since his sustained massive brain injury due to a freak accident while he was skiing in December 2013.

A few weeks after the accident, Schumachers family refused to release information about the F1 champs health recovery. The family completely shut down the media so fans were deprived of information as to what is going on and any development in Schumis health. The secrecy went on for years and up to now, they remained secretive when it comes to the motorsport legends condition.

Secret treatment

It has been over six years since Micheal Schumacher had an accident and since then, some new treatments were developed. It was reported that the former sportsman tried out one of the latest procedures available today and it is called the stem cell treatment.

The Telegraph reported that Schumi was brought to Paris and admitted at the Georges-Pompidou hospital in September. The procedure was supposedly carried out by cardiovascular surgeon Philippe Menasch and it was said that one of the medical staff attested that Schumacher is conscious after the stem-cell procedure, a treatment dubbed as the pioneer in cell surgery."

It was said that Michael Schumacher stayed at the hospital for three days and on the last day, he was transported back to his home in Switzerland via ambulance. The question is - did he improved after the treatment?

How is he today?

As per The Daily Mail, even the surgeon who performed the procedure on Michael Schumacher said that he doesnt perform miracles. It appears he is saying that even though the treatment is considered a breakthrough in the medical field today, it may not work for everyone. With the racers condition, he may have developed some kind of complications that could have made his health situation worst and this treatment may have not affected Schumi as it should.

It is believed that Michael Schumacher is still paralyzed and being nursed on his bed. His movements are still limited and If there are improvements, perhaps, he could talk a bit and move some parts of his body like his head.

Continued here:

Michael Schumacher reportedly underwent breakthrough treatment; Here are details of the secret procedure - EconoTimes

Crohn’s Disease Treatment Market Development, Top Trends and Future Scope with Upcoming Opportunities – Instant Tech News

Crohns Disease is a type of inflammatory bowel disease (IBD) causing inflammation and damage to the digestive tract usually affecting the end of small intestine and beginning of colon, but inflammation may occur anywhere along the digestive tract. The disease may be hereditary or grow due to the malfunctioning of the immune system. It includes some risk factors, such as genetics, combined with an overactive immune system & the environmental changes, and certain bacteria, like, mycobacterium, etc.

Get Sample Copy Of The [emailprotected]https://www.trendsmarketresearch.com/report/sample/3967

Demand Scenario

The global Crohns disease treatment market was USD 9,674 million in 2018 and is estimated to reach USD 12,834 million by 2025 at a CAGR of 4.12% during the forecast period

Growth by Region

North America accounted for the largest market share in 2018 due to the largest contribution from the growing US market. Growth in Europe is driven by the higher per capita income and strong healthcare penetration in the region. Asia Pacific is likely to create significant growth opportunities for the market players owing to develop infrastructure, R&D activities related to biologics and increase in prevalence of Crohns disease in the region. Emerging countries such as India and China are expected to show the fastest growth owing to increase in R&D investment and healthcare expenditure in these countries.

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Drivers and Restraints

The major driver for Crohns disease treatment market is increase in number of people being infected by the disease. Moreover, increase in demand for biologics and high unmet needs within the anti-TNF refractory patient group that presents untapped market opportunities. Moreover, strong pipeline and novel mechanisms to treat Crohns disease are boosting the growth of the market. However, patent expiry of the important drugs, high cost of treatment, high R&D investment for development of biological drugs and unknown aetiology of the Crohns disease are the factors hindering the market growth.

Industry Trends and Updates

In March 27, 2018, Researchers at the University of Alberta (Canada), published the study of the potential biomarkers they have developed to help reduce the suffering of Crohns disease patients. These scientists used high performance liquid chromatography with mass spectrometry and created a profile of potential biomarkers. In April 03, 2018, TiGenix NV (Belgium), a global cell therapy company and Takeda Pharmaceutical (Japan), the largest global pharmaceutical company, together announced approval from the EC (European Commission) for the treatment Alofisel (darvadstrocel) that they have developed to treat the complex perianal fistulae in adult patients with non-active(mildly active) luminal Crohns disease. This is the initial allogeneic stem cell therapy (originating from donor stem cells) to receive central marketing authorisation (MA) approval in Europe.

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Crohn's Disease Treatment Market Development, Top Trends and Future Scope with Upcoming Opportunities - Instant Tech News

Study tests whether stem cells heal arthritis in large dogs – Los Angeles Times

About a year ago, Cheryl Timmons was worried her dog Baxter would soon need to retire from being a therapy dog due to arthritis in his hips.

The 99-pound German shepherds physical health was wearing down after years of bringing joy to childrens hospitals, senior homes and courtrooms, where he served as the first and only service dog providing comfort to child trafficking victims in Orange County.

Timmons, who rescued Baxter from the streets of San Bernardino, worried that she may even have to put him down.

To combat the worsening arthritis, Timmons took him to therapy sessions. A GoFundMe campaign to help pay for the therapy reached a goal of $4,500.

But the arthritis was still taking hold, affecting how Baxter functioned during long workdays.

Then in late August, he was given stem cell injections as part of a new study at the Anaheim Hills Pet Clinic. The effort, headed by San Diego-based Animal Cell Therapies, is testing whether stem cells can help alleviate arthritis in dogs weighing 70 pounds or more.

Baxter, now 11 years old, has been feeling better since he received his injection.

His arthritis is greatly improved, Timmons said. I swear by the stem cell treatment. It has made such a huge difference.

Everybody in the court would notice that he wasnt having a good day. Now hes looking great again. Hes running through the courtroom. He is one happy boy.

Baxter was one of about 10 dogs that was tested at the Anaheim clinic. Animal Cell Therapies is conducting the testing at a dozen clinics throughout the country.

There are about 35 dogs currently enrolled in the study. Researchers are hoping to test between 60 and 80 dogs.

Kathy Petrucci, chief executive of Animal Cell Therapies, said its too early to tell whether the treatment is successful in treating arthritis in large dogs, but the early results are promising.

The company conducted a similar study a year ago, which showed benefits for arthritis in dogs under 70 pounds. However, the results were mixed for bigger dogs.

Petrucci said they increased the dosage for the current study.

We dont know every single mechanism that is involved ... it helps decrease inflammation in the joints, Petrucci said of the treatment. We think that the cells secrete a lot of positive beneficial growth factors that help decrease inflammation, help make the environment a better, more friendly place for more normal cells to come in and help repair the joints.

Whatever the cause, Timmons just hopes the treatment allows Baxter to keep doing what he does best.

With the stem cells, he acts like hes invincible, Timmons said, laughing. I really hope he is.

To enroll in the study, visit dogstemcellstudy.com.

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Study tests whether stem cells heal arthritis in large dogs - Los Angeles Times

Canine Stem Cell Therapy Market Regulations and Competitive Landscape Outlook to 2029 – Instant Tech News

Canine Stem Cell Therapy Market Research Study

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The report evaluates the current state of the Canine Stem Cell Therapy Market in terms of volume (X units), consumption, value (Mn/Bn), production and more. In addition, the study tracks the latest proceedings within the various market segments, end use industries, geographies, and regulatory landscape.

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Stem Cell Therapy Market 2020 Company Profiles, Trends by Types and Application, Operating Business Segments 2026 – News Parents

New Jersey, United States, This report looks at the global market Stem Cell Therapy Status and future trends, focusing on the global market major enterprises, while the current and future trends in North America, Europe, China, Asia Pacific and South America and other regions.

Global Stem Cell TherapyMarketwas valued at USD 86.62 million in 2016 and is projected to reach USD 221.03million by 2025, growing at a CAGR of 10.97% from 2017 to 2025.

This article focuses on the market size, market share, market positioning, product type and development planning of enterprises with important roles in the global Market.

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The main companies operating in the Stem Cell Therapy market are listed in the report.

In the segmentation section of the report, the authors have elaborately presented key driving factors for different segments of the global Stem Cell Therapy industry. The report offers a Complete research study on product type and application segments of the global Stem Cell Therapy industry. The segmental analysis provided in the report is expected to help players and investors to identify lucrative growth pockets of the global Stem Cell Therapy industry.

The report offers a complete company profiling of leading players competing in the global Stem Cell Therapy industry with high focus on share, gross margin, net profit, sales, product portfolio, new applications, recent developments, and several other factors. It also throws light on the vendor landscape to help players become aware of future competitive changes in the global Stem Cell Therapy industry.

Global Stem Cell Therapy Market: Regional Analysis

This part of the report includes detailed information of the market in different regions. Each region offers different scope to the market as each region has different government policy and other factors. The regions included in the report are North America, South America, Europe, Asia Pacific, and the Middle East. Information about different region helps the reader to understand global market better.

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What does the report hold?

Industry size and forecast: Industry analysts have provided historical, current and estimated forecasts of industry size in terms of value and volume.

Future opportunities: This section of the report provides Stem Cell Therapy participants with information on the future prospects that the Stem Cell Therapy industry is expected to offer.

Industry trends and developments: Here the authors of the report talked about the most important trends and developments in the Stem Cell Therapy market and its estimated effects on overall growth

Industry segmentation study: this part of the report provides a detailed breakdown of the main segments of the industry for Stem Cell Therapy , including product type, application and industry.

Regional analysis: Stem Cell Therapy providers receive important information about high-growth regions and their respective countries so that they can invest in profitable regions

Competitive landscape: This unit of the report highlights the competitive scenario of Stem Cell Therapy by focusing on the key strategies of providers to consolidate their presence in the Stem Cell Therapy business.

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TAGS: Stem Cell Therapy Market Size, Stem Cell Therapy Market Growth, Stem Cell Therapy Market Forecast, Stem Cell Therapy Market Analysis, Stem Cell Therapy Market Trends, Stem Cell Therapy Market

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Stem Cell Therapy Market 2020 Company Profiles, Trends by Types and Application, Operating Business Segments 2026 - News Parents

DDP and Austin Aries Reportedly Pitching Stem Cell Venture to AEW – 411mania.com

Diamond Dallas Page and Austin Aries had business at last nights AEW according to a new report, but not the kind you might immediately think of. As noted last night, DDP and Aries were seen entering the area at last nights episode in Atlanta.

According to BodySlam.net, the two were there because theyre part of a stem cell treatment venture. They were there to pitch the idea to AEW in hopes that the company would buy into the venture. Stem Cell therapy has become increasingly popular among wrestlers to deal with injury issues over the past several years, and Aries is one of several people (along with Ken Shamrock, Rob Van Dam and others) to make regular mention of the BioXcellerator stem cell therapy clinic.

DDP has been a semi-regular on AEW, making several appearances on Dynamite over the past few months.

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DDP and Austin Aries Reportedly Pitching Stem Cell Venture to AEW - 411mania.com

High-throughput screening reveals new compound to maintain lung health – Drug Target Review

Researchers have screened 20,000 molecules to discover a potent compound with low toxicity that restores the balance of healthy stem cells in the lungs of mice.

A molecule identified by researchers through screening helps maintain a healthy balance of cells in airway and lung tissue. According to the scientists, if the compound, so far only studied in isolated human and mouse cells, has the same effect in people, it may lead to new drugs to treat or prevent lung cancer.

We think this could help us develop a new therapy that promotes airway health, said Dr Brigitte Gomperts, lead author of the study and professor of paediatrics and pulmonary medicine at the University of California LA (UCLA), US, where the stiudy was conducted. This could not only inform the treatment of lung cancer, but help prevent its progression in the first place.

The human respiratory system is regularly replenished with healthy cells. That process is driven by airway basal stem cells, which divide to produce both more stem cells and the mucociliary cells that line the airways and lungs.

Mucociliary cells include:

In healthy lungs, airway basal stem cells stay balanced between producing mucociliary cells and self-renewing to maintain a population of stem cells.In pre-cancerous cells in the lungs, basal stem cells divide more often than usual, generating a large number of stem cells but too few mucociliary cells. The resulting imbalance of cells in the airway leaves it unable to properly clear debris and creates a greater risk that the pre-cancerous cells will develop into a tumour.

In the new study, the researchers analysed airway cells from equal numbers of biopsies of healthy people, patients with pre-malignant lung cancer lesions and patients with squamous lung cancer. They discovered that one group of molecules collectively called the Wnt/beta-catenin signaling pathway was present at different levels in the basal stem cells of the patient samples versus the cells from healthy people.

Normal airway tissue, left and lung cancer tissue, right, with an overabundance of basal stem cells (green). The activated form of beta-catenin (red) in the lung cancer can be targeted by the Wnt Inhibitor Compound 1 (credit: Broad Stem Cell Research Center).

Altering the levels of these molecules in healthy airway cells from mice, the team found the balance between stem cells and mucociliary cells shifted, mimicking the imbalance seen in lung pre-cancers.

When you activate the Wnt/beta-catenin signaling pathway, these stem cells just divide and divide, said Gomperts.

Finally, the researchers screened more than 20,000 chemical compounds using high-throughput methods. They investigated their ability to reverse this effect in human cells, lowering levels of Wnt and restoring the balance of stem cells and mature airway cells.

One compound, named as Wnt Inhibitor Compound 1 (WIC1), was particularly effective at limiting the proliferation of basal stem cells and restoring the balance of the stem cells and mucociliary cells to regular levels. The researchers also noted that the compound was less toxic to airway cells than previously discovered molecules that block Wnt/beta-catenin signalling.

The identification of this new drug is a nice tool to tease apart the biology of the Wnt/beta-catenin signalling pathway and its effects on lung health, said Cody Aros, the first author of the new paper and a UCLA graduate student. Its also very exciting that it may act in a new way than other existing Wnt/beta-catenin signalling pathway inhibitors and has such low toxicity.

As WIC1 was identified through a random drug screen, the researchers do not yet know exactly how it works, but they are planning future studies on its mechanism and safety.

The study was published in Cell Reports.

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High-throughput screening reveals new compound to maintain lung health - Drug Target Review

Locking in and preserving your healthy stem cells has never been easier and more accessible as Acorn Biolabs partners with Coverdale Clinics. – Yahoo…

Appointments for stem cell & DNA collection now available.

TORONTO , Feb. 20, 2020 /CNW/ - With the rapid emergence of regenerative medicine therapies and genetic analysis testing reaching mainstream medicine, consumers are demanding increased opportunities to prepare for their future healthcare needs, including banking a viable source of cells to preserve their current health.

Acorn Biolabs (CNW Group/Acorn Biolabs)

To meet this growing demand, Acorn Biolabs, the leading non-invasive stem cell collection, cryopreservation and analysis company, today announced its partnership with Coverdale Clinics Inc., a premium network of specialty care clinics in Canada . Together, Acorn and Coverdale will help make stem cell collection more accessible and affordable in the West Greater Toronto Area .

Through their partnership, Coverdale Clinics' Oakville location be offering Acorn's non-invasive stem cell collection services. The simple process involves plucking a few hair follicles from a client's head, which are then analyzed and cryopreserved for future use.

Acorn's innovative solution for live cell collection enables the collection of stem cells without the need for surgery or other painful and invasive procedures, making stem cell collection for life-long storage significantly more affordable and accessible for everyone.

"Our partnership with Acorn Biolabs opens up a great opportunity for us to expand Coverdale Clinics service offering into the emerging regenerative medicine and geneticsmarket. We're pleased to be able to leverage our specialty clinic in Oakville to offer a service that improves access to exciting and novel health care technologies," said Chris Dalseg , BioScript Solutions' Vice President of Strategic Growth and Marketing. "We have always been at the forefront of providing innovative health care services to Canadians, and adding stem cell collection services from Acorn exemplifies our ongoing commitment."

Once stem cells are collected, Acorn uses its proprietary capabilities to keep cells alive during transportation before going into long term cryogenic storage. The process turns collected hair follicles into a highly valuable and accessible resource for further regenerative medicine and genetics. Not only are these stem cells securely stored for future use, but the company's scientists will also be able to extract critical genetic information through DNA tests, for clients that want it, that will unlock valuable data about a person's health over their lifetime.

"We are excited to bring Acorn's preventative, personalized healthcare services to more people through this partnership with Coverdale Clinics. The cells collected are a life-long resource for these clients, not only in regenerative medicine, but also for advanced analytics, helping to identify diseases even before the first symptom," said Dr. Drew Taylor , Co-founder and CEO at Acorn Biolabs Inc. "The partnership is an important extension for Acorn to serve health-minded individuals in more geographies, freezing the clock on their stem cells so they can use them later in life, when they will need them most."

Clients can book their non-invasive stem cell collection appointment in Oakville, Ontario through Acorn's website today at http://www.acorn.me

About Acorn Biolabs, Inc.

Story continues

Acorn helps you live a longer, healthier tomorrow by freezing the clock on your cells today. Founded in 2017 by Steven ten Holder, Patrick Pumputis and Dr. Drew Taylor and borne out of years of research, Acorn is a healthcare technology company based at Johnson & Johnson INNOVATION JLABS in Toronto . Acorn is focused on giving everyone the best chance to experience more healthy years with its easy, affordable and non-invasive stem cell collection, analysis and cryopreservation service. For more information, visit acorn.me.

About Coverdale Clinics

Coverdale Clinics is a premium network of specialty care clinics, providing patients with a safe, comfortable environment to receive specialty medications by infusion or injection. With more than 100 clinics nationwide, our nurses take a personalized approach to patient care that includes education and counselling to better support medication adherence.

About BioScript Solutions

BioScript Solutions is committed to helping patients with chronic illnesses achieve the best possible health outcomes. With our total care approach, we simplify access to complex, specialty drug therapies and deliver full-service specialty care solutions at every stage of the patient's treatment journey. Through our specialty pharmaceutical distribution, pharmacies, patient support programs and clinical services, BioScript has the capability to manage the needs of manufacturers, payors, prescribers and health care practitioners across Canada today, and tomorrow. To learn more, please visit bioscript.ca.

SOURCE Acorn Biolabs

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Locking in and preserving your healthy stem cells has never been easier and more accessible as Acorn Biolabs partners with Coverdale Clinics. - Yahoo...

Dutch startup Neuroplast raises 4M for its stem cell-based technology to treat patients with Spinal Cord Injury – Silicon Canals

Neuroplast is a company based in Maastricht (the Netherlands) developing autologous stem cell therapies for patients suffering from neurodegenerative diseases such as spinal cord injury (SCI), amyotrophic lateral sclerosis (ALS) and traumatic brain injury.

Recently, the company has raised 4 million from Dutch-based Brightlands Venture Partners and LIOF and from an existing shareholder and informal investor Lumana Invest BV.

CEO Johannes de Munter said:

The financing and support of the investors will enable us to perform multicenter clinical trials in the Netherlands, Denmark, Germany, and Spain and bring the product to market.

This Dutch startup will use the fund to perform a phase II/III clinical trial with the aim of obtaining conditional market approval for the treatment of patients suffering from Spinal Cord Injury.

Founded by physician Hans de Munter and neurologist Erik Wolters in 2014, Neuroplast has expanded with Juliette van den Dolder, who was appointed as COO and management team member.

In the case of SCI, isolating, manufacturing, and reinserting patients own cells, very promising preclinical outcomes have resulted in an Orphan Drug Designation from European regulatory authorities, allowing a fast-track procedure for the clinical trials. These trials are expected to start in March 2020.

Marcel Kloosterman Director at Brightlands Venture Partners:

Neuroplast combines breakthrough science with a solid management team. In a sizable market characterised by major unmet need, successful treatment of (accident caused) paralysed patients would make life so much easier for them and their families while lowering the burden and costs for the society.

Yearly, 24,500 people in Europe and the USA are diagnosed with Spinal Cord Injury, usually caused by accident. Its worth mentioning that for Europe and the US, the medical cost associated with Spinal Cord Injury is over 13 bn per year.

CEO Johannes de Munter adds:

Neuroplast is becoming an ATMP player in the region and wants to contribute to our beautiful eco-system.

Main image credits:Neuroplast

Stay tuned toSilicon Canalsfor more European technology news

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Dutch startup Neuroplast raises 4M for its stem cell-based technology to treat patients with Spinal Cord Injury - Silicon Canals

UCLA researchers identify compound that could improve lung disease treatment – Daily Bruin

A UCLA study identified a chemical compound that may improve lung health, potentially suggesting new approaches to preventing and treating lung diseases.

According to the study published Tuesday in Cell Reports, the UCLA team discovered a compound now named Wnt Inhibitor Compound 1, or WIC1, that successfully improved the health of isolated cancerous human and mouse airway cells.

The compound targets a group of molecules, called the Wnt/-catenin signaling pathway, that is more activated in the lungs of people with precancerous lesions or lung cancer than in the lungs of healthy people. Elevated activity in this pathway has also been linked to lung cancer in other studies.

The researchers tested around 20,000 compounds on their abilities to block this pathway in the process of identifying WIC1.

The researchers also found that the compound WIC1 was far less toxic than other known inhibitors of the Wnt/-catenin signaling pathway. The compound could therefore be used to develop safer drugs to prevent and treat lung diseases linked to the pathway.

Led by Brigitte Gomperts, a UCLA professor of pediatrics and of pulmonary medicine, and Cody Aros, a molecular biology graduate student, the team is planning to further investigate the safety of WIC1 and the mechanism by which it blocks the pathway.

The study was supported in part by the National Institutes of Health, the National Cancer Institute and the Broad Stem Cell Research Center Training Program.

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UCLA researchers identify compound that could improve lung disease treatment - Daily Bruin

On the Trail of Cancer Stem Cells – Technology Networks

Two research teams from the Max Delbrck Center for Molecular Medicine and their collaborators have produced a detailed cell atlas of an entire salivary gland tumor in a mouse model, mapping individual cells throughout the tumor and its surrounding tissue. The "single cell" approach, recently described inNature Communications, has provided key insights about cellular composition changes through the earliest stages of cancer development.

A solid tumor is not, as many might assume, a lump of cells that are all the same. Rather it is mix of many different cell types, including a variety of stromal and immune cells besides the actual tumor cells.

"Conventional methods in molecular biology often consider a sample as a whole, which fails to recognize the complexity within it," said Dr. Samantha Praktiknjo, senior scientist and first author from MDC's Systems Biology of Gene Regulatory Elements Lab headed by Professor Nikolaus Rajewsky at the Berlin Institute for Medical Systems Biology (BIMSB). Developing a detailed understanding of the different cells within a tumor and how they interact could help identify more effective treatment strategies.Strength in numbers

The team used single-cell RNA sequencing technologies developed in theRajewsky laband novel epitope profiling to produce the cell atlas, and identified the cells that were specific to the tumor by leveraging the reproducibility and the large sample size of their data.

The latter was possible by using a mouse model, developed in MDC's Signal Transduction in Development and Cancer Lab headed by Professor Walter Birchmeier, which harbors designed mutations that induce a salivary gland squamous cell carcinoma. This system provides a consistent supply of genetically similar tumors to sequence from the earliest stages of development, which is nearly impossible with human patients.

"In a patient, the tumor is already developed and you cannot go back and rewind time and look at how it started," said Dr. Benedikt Obermayer, a co-first author now at the Berlin Institute of Health (BIH). "Here, we have a model that is so controlled, we can watch it happen." And Dr. Qionghua Zhu, the third first author and a former postdoc at theBirchmeier Lab, added: "To fight cancer effectively, we need to find the driver mutations. This method gives us clues about the evolution trajectories of a tumor."

Sequencing technologies have advanced so that it is now possible to quickly and affordably sequence the RNA inside single cells, one at a time, as well as the proteins on the surfaces of cells in the tissues. While other methods grind up the tissue and identify what genes and molecules are present in the mix, the single cell approach precisely identifies how many of each type of cell is present, and which genes and molecules are associated with which cell.

For this study, the researchers sequenced more than 26,000 individual salivary gland cells from mice with tumors and healthy mice. They used computational models to analyze the huge amount of data and identify each individual cell and sort them into groups - such as stromal cells, immune cells, saliva producing cells, cancer cells - based on the hundreds of genes expressed and molecules present.A surprise

The single cell approach revealed something that surprised the researchers: "When I saw the data, I thought, where is the tumor?" Obermayer said. The population of cancer stem cells in the tumor was extremely small - less than one percent of all profiled cells in the tissue. Due to their low abundance, investigation of these cells still heavily depends on assumptions about surface markers and is often performed in cell culture-based systems. Here, the authors were able to identify the cancer stem cells directly from the solid tumor samples with their single cell approach.

Furthermore, the team was able to predict the progression of the different cell types through different stages of tumor development. Their model suggests that the cancer stem cells emerge from cancerous basal cells, then develop into another subtype before ultimately becoming a population of cells similar to luminal cells, a cell type present in normal, healthy salivary glands.

This progression supports the idea that when something goes awry in the basal cells of this solid tumor model, they are triggered to turn into cancer stem cells, which can then become a different type of cell. "What I found fascinating was clearly seeing the order of signals and events, transitioning from the progenitor to the progeny populations of the cancer stem cells," Praktiknjo said.Next steps

Further research is required to verify that individual cells are transforming through these stages, and explore the cellular and molecular interactions driving tumor growth. The team anticipates the approach they've demonstrated here can be applied to other cancer types as well.

"To me the main conceptual insight is that we can apply ideas from single-cell based developmental biology to reconstruct molecular progression of tumorigenesis ," said Professor Nikolaus Rajewsky, who heads MDC's Systems Biology of Gene Regulatory Elements Lab and is the scientific director of the BIMSB.

Reference:Praktiknjo, et al. (2020) Tracing tumorigenesis in a solid tumor model at single-cell resolution, Nature Communications, DOI: 10.1038/s41467-020-14777-0.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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On the Trail of Cancer Stem Cells - Technology Networks