Lou Gehrig's Disease (ALS): Progress and Promise in Stem Cell Research – Video

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29-10-2010 14:40 CIRM has funded the ALS Disease Team led by scientists at UC San Diego, The Salk Institute, and Life Technologies Corporation who aim to bring a human embryonic stem cell based ALS therapy to clinical trials within four years. Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a progressive motor neuron disorder. Most people with ALS die within 3 to 5 years from the onset of symptoms. For more information visit CIRM's ALS disease fact sheet (www.cirm.ca.gov/ALS_facts) and The ALS Association website (www.alsa.org)

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Lou Gehrig's Disease (ALS): Progress and Promise in Stem Cell Research - Video

Stem Cell Pioneers Converge in Portland to Discuss and Celebrate a Revolutionary New Stem Cell Entering Human Clinical …

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SAN DIEGO, CA and PORTLAND, OR--(Marketwire -02/28/12)- Medistem Inc. (Pinksheets: MEDS.PK - News) announced today its Annual "Evening with Medistem" Event will take place in Portland, Oregon on March 7th, 2012. The event is being hosted by Vladimir Zaharchook, Vice Chairman at Medistem, Inc., and will feature stem cell luminaries and pioneers working with Medistem including Dr. Amit Patel, Director of Regenerative Medicine at University of Utah and the first person to administer stem cells into patients with heart failure, Dr. Michael Murphy, Vascular Surgeon at Indiana University and Principal Investigator for Medistem's FDA clinical trial in patients with risk of amputation, and Dr. Alan Lewis, former CEO of the Juvenile Diabetes Research Foundation, advisory board member of Medistem.

In 2007 Medistem discovered an entirely new type of stem cell, the Endometrial Regenerative Cell (ERC). This cell has proven it is a "universal donor" and can be used to treat many more conditions compared to other types of stem cells. The company received FDA clearance to begin clinical trials in September of 2011 for critical limb ischemia, a condition that is associated with amputation. Medistem is also running a Phase II clinical trial for heart failure using the new stem cell. The ERC stem cell does not involve the highly controversial use of fetal tissue, can be produced very economically and administered to the patient in a very simple manner. Medistem is exploring ways to expand clinical trials of its stem cell into other diseases.

"Stem cells and regenerative medicine offer hope in clinical conditions in which hope previously did not exist," said Dr. Stanley Cohan, Head of Neurology at the St Vincent's Hospital, the largest center for treatment of multiple sclerosis in the Pacific Northwest, who will be attending the event. "We are honored in the Portland community to have this distinguished team of accomplished researchers and medical doctors convene here and discuss with us possible collaborations."

"As a long-time member of the Portland academic community, it is exciting to have companies such as Medistem to visit us and share their experiences 'from the trenches' of what it takes to push a cellular drug through the FDA," said Dr. Shoukrat Milipotiv, Associate Scientist in the Division of Reproductive & Developmental Sciences of ONPRC, Oregon Stem Cell Center and Departments of Obstetrics & Gynecology and Molecular & Medical Genetics, and co-director of the ART/ESC core at the Center. He is an internationally recognized researcher in the area of stem cells.

"The Event is an annual celebration to honor our team and collaborators for the successes of the previous year, while at the same time educate the local business and medical community on the latest research on stem cells not just at Medistem but internationally," said Thomas Ichim, Ph.D Chief Executive Officer of Medistem Inc. "2012 is particularly exciting for us due to approvals for two clinical trials, and the initiation of patient treatments within this context."

About Medistem Inc.

Medistem Inc. is a biotechnology company developing technologies related to adult stem cell extraction, manipulation, and use for treating inflammatory and degenerative diseases. The company's lead product, the endometrial regenerative cell (ERC), is a "universal donor" stem cell being developed for critical limb ischemia and heart failure.

Cautionary Statement

This press release does not constitute an offer to sell or a solicitation of an offer to buy any of our securities. This press release may contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking information. Factors which may cause actual results to differ from our forward-looking statements are discussed in our Form 10-K for the year ended December 31, 2007 as filed with the Securities and Exchange Commission.

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Stem Cell Pioneers Converge in Portland to Discuss and Celebrate a Revolutionary New Stem Cell Entering Human Clinical ...

Stem Cells: applications for understanding brain function and disease – Video

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22-11-2010 14:24 The existence of stem cells in the adult brain and their possible functions will be discussed as well as how human embryonic Stem Cells and induced Pluripotent Stem Cells can be used to model human disease. Fred H. Gage, Ph.D. (NAS/IOM) is the head of the Laboratory of Genetics at the Salk Institute for Biological Studies, the Vi and John Adler Chair for Research on Age-Related Neurodegenerative Diseases and an Adjunct Professor in the Department of Neurosciences, UCSD, La Jolla.

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Stem Cells: applications for understanding brain function and disease - Video

Stem Cell Research Today: Larry Goldstein – CIRM Science Writer's Seminar – Video

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17-11-2011 09:48 (Part 1 of 9) Larry Goldstein, MD, spoke at the Scientific Writer's Seminar, a workshop presented on September 17, 2008 at CIRM headquarters in San Francisco. Goldstein presented an overview of the basic principles and concepts of stem cell biology and stem cell clinical trial development. He has a CIRM grant to use human embryonic stem cells to understand and to develop new therapies for Alzheimer's disease.

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Stem Cell Research Today: Larry Goldstein - CIRM Science Writer's Seminar - Video

Embryonic Blood Vessels that Make Blood Stem Cells can also Become Beating Heart Muscle Cells

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Newswise UCLA stem cell researchers have found for the first time a surprising and unexpected plasticity in the embryonic endothelium, the place where blood stem cells are made in early development.

Scientists found that the lack of one transcription factor, a type of gene that controls cell fate by regulating other genes, allows the precursors that normally generate blood stem and progenitor cells in blood forming tissues to become something very unexpected - beating cardiomyocytes, or heart muscle cells.

The finding is important because it suggests that the endothelium can serve as a source of heart muscle cells. The finding may provide new understanding of how to make cardiac stem cells for use in regenerative medicine, said study senior author Dr. Hanna Mikkola, an associate professor of molecular, cell and developmental biology in Life Sciences and a researcher with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

It was absolutely unbelievable. These findings went beyond anything that we could have imagined, Mikkola said. The microenvironment in the embryonic vasculature that normally gives rise to blood cells can generate cardiac cells when only one factor, Scl, is removed, essentially converting a hematopoietic organ into a cardiogenic organ.

The two-year study is published Aug. 3, 2012 in the peer-reviewed journal Cell.

The findings were so surprising, in fact, that Mikkola and her team did not want to believe the results until all subsequent assays proved the finding to be true, said Amelie Montel-Hagen, study co-first author and a post-doctoral fellow.

To make sure we had not switched the samples between blood forming tissues and the heart we ran the experiments again and repeatedly got the same results, Montel-Hagen said. It turns out Scl acts as a conductor in the orchestra, telling the other genes in the endothelium who should be playing and who shouldnt be playing.

The team used microarray technology to determine which genes were playing in embryonic endothelium to generate blood stem and progenitor cells and found that in the absence of Scl, the genes required for making cardiomyocytes were activated instead, said study co-first author Ben Van Handel, a post-doctoral fellow.

The lone difference was that Scl was missing in the process that resulted in the fate switch between blood and heart.

Scl has a known role as a master regulator of blood development and when we removed it from the equation, no blood cells were made, Van Handel said. That the removal of Scl resulted in fully functional cardiomyocytes in blood forming tissues was unprecedented.

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Embryonic Blood Vessels that Make Blood Stem Cells can also Become Beating Heart Muscle Cells

BioLife Cell Bank and Intrexon Establish Worldwide Exclusive Collaboration for Spinal Muscular Atrophy (SMA)

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DALLAS and GERMANTOWN, Md., Aug. 2, 2012 (GLOBE NEWSWIRE) -- BioLife Cell Bank, Inc., the leader in cryogenic storage of adipose (fat) tissue and adult mesenchymal stem and regenerative cells, and Intrexon Corporation, a synthetic biology company that utilizes its proprietary products to provide control over cellular function, announced today the formation of a global exclusive research collaboration. Under the collaboration, BioLife, with its extensive physician network and stem cell experience coupled with Intrexon's innovative technology, will strive to produce new treatments for Spinal Muscular Atrophy (SMA).

"With the tremendous potential of this collaboration, we are more than excited for the SMA community," said John A. Carbona, Chief Executive Officer of BioLife. "Intrexon's impressive suite of technologies will give researchers access to unprecedented resources including new techniques and processes which could rapidly propel us toward the development of new treatments and products to help treat children with SMA."

Carbona continued, "I am happy to help realize the dream of our founders, Dr. David G. Genecov and John D. Harkey Jr., to make a positive and sustainable impact in health care. We're elated by our new collaboration with Intrexon and will undertake immediately the establishment of relationships with the world's leading scientists in SMA research such as: Dr. Arthur Burgess of Ohio State, Dr. Brian Kaspar of Nationwide Children's Hospital, Dr. Charlotte Sumner of Johns Hopkins, Dr. Chris Lorson at the University of Missouri, and Dr. Kathy Swaboda at the University of Utah. BioLife, through its ongoing relationships with Intrexon and these dedicated individuals, hopes for great strides toward lessening the impact of this terrible disease, if not curing it completely--which, of course, is our ultimate goal."

Thomas D. Reed, Ph.D., Founder and Chief Science Officer of Intrexon, said, "Intrexon's mission is to invent, acquire, and integrate the diverse technology platforms required to modulate cellular behavior through genome re-engineering. We are dedicated to building the molecular toolbox and scientific expertise needed to empower clinicians to treat previously intractable diseases. SMA is a devastating genetic disorder that requires a gene rescue paradigm. Intrexon looks forward to working with BioLife and their growing network of clinical specialists to define, explore, and develop several different cell therapy approaches for treating SMA."

Under the collaboration, Intrexon, acting through its Human Therapeutics Division, will be applying its technologies to the discovery of autologous, genetically-modified stem cell therapeutics. BioLife will be supplying the collaboration with stem cells and clinical expertise. BioLife also will be responsible for conducting preclinical and clinical development of candidate SMA therapeutic products that may be advanced out of the collaboration, as well as for aspects of manufacturing and regulatory approval.

About Spinal Muscular Atrophy

Spinal Muscular Atrophy (SMA) is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons. SMA is caused by a genetic defect in the SMN1 gene which codes SMN, a protein necessary for survival of motor neurons. SMA kills more infants than any other genetic disease in today's world.

About BioLife

As part of their core business, BioLife Cell Bank, Inc. offers individuals a way to safely store their adipose (fat) tissue and/or their adipose-derived stem and regenerative cells--giving patients and physicians easy, multi-use access to cells and tissue for future cosmetic, reconstructive, and regenerative therapies. Tissue is extracted via liposuction and sent to BioLife in a collection kit (validated to E.T.L. standards). Tissue is processed using proprietary technology and Cytori Therapeutics' (CYTX) products. Tissue is cryogenically preserved, and may be stored indefinitely. BioLife is registered with the FDA as a processing bank and complies with FDA regulations and guidance including current Good Tissue Practice (cGTP). BioLife is based in Dallas, Texas, at Forest Park Medical Center. For more information: http://www.biolifecellbank.com.

About Intrexon Corporation

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BioLife Cell Bank and Intrexon Establish Worldwide Exclusive Collaboration for Spinal Muscular Atrophy (SMA)

Stem cell 'makeovers' provide a way to get rid of wrinkles

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MIAMI (WTVJ/NBC) - It is widely known that stem cells can be used in life-saving treatments for deadly diseases.

Now they are being used in the fight against wrinkles.

Donna Pritchit recently had a "stem cell" makeover.

The 64-year-old headed into the operating room wanting to turn back the hands of time without it being totally obvious.

"I don't want someone to stop and go by and say Oh, she had a facelift.' I want to have someone say Donna went on vacation she must be having a great life,'" she said before the $5,000 procedure began.

Dr. Sharon McQuillan at the Ageless Institute in Aventura, FL marked the areas where she would take fat out of Pritchit's belly - and place it back into her face.

The retired teacher also hoped it would be her last step in getting rid of embarrassing acne scars.

The outpatient procedure began with traditional liposuction, and then McQuillan and her team processed that fat and concentrated the stem cells so they could be injected into Pritchit's wrinkles and in places where she has lost fullness.

"Stem cells in general are the cells in your body that regenerate tissue and heal tissue, and they make the skin look beautiful and younger," McQuillan explained.

While there are not many long-term studies on the procedure, McQuillan said the results are permanent.

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Stem cell 'makeovers' provide a way to get rid of wrinkles

Medical Megatrends – Stem Cells – Part II of III

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Imagine a man with a recent severe heart attack who has the muscle repaired with stem cells or a child with a severe bladder defect repaired with stem cells grown on a biodegradable scaffold. Sounds like science fiction but these are actual clinical studies in progress today. Stem cell therapies promise to be one of those scientific breakthroughs that will have an enormous impact on health care in the future. Stem cells will bring us closer to the goal of personalized medicine, just as genomics is doing. The course of a disease will change once we have the technology to develop and then insert stem cells into the human body to actually create a tissue. For example, a person with a heart attack will not go on to live the rest of his or her life with damaged heart muscle and resultant heart failure. Instead, stem cells will repopulate the heart muscle and make it whole again. Similarly, a person with Parkinsons disease will recover full faculties thanks to the ability of stem cells to regenerate the damaged area of the brain. The person with type I diabetes will be free of the disease because of the formation of new pancreatic islet cells. The athlete will play again because new cartilage will be created for the worn knee. This is the promise of regenerative medicine. I have written the above as though each will definitely happen, a promise that will be kept. They probably will, but it may be a long time before the science of stem cells is sufficiently developed that these types of incredible results will be commonplace. Adult stem cells are being used today for treatment of a few diseases and there are studies ongoing and planned for many additional possibilities. Lets consider a few of them. Each of our tissues has a population of cells that can divide as needed to keep the organ or tissue functional as cells die or are injured. We see this with our skin as it constantly lays down new cells which make their way to the surface as the dead cells on the surface are rubbed off in the shower. We also see it when we cut ourselves and yet in a few days the wound is completely healed that was stem cells at work. It appears that essentially every organ has its own pool of such cells. There are cells in the bone marrow that can become stem cells for many different tissues. These cells circulate in the blood and can be called to assist a tissue or organ to rebuild itself after injury or damage. So for example, if a surgeon takes one half of a fathers liver for transplantation into his son, we know that the fathers liver will grow back to normal size within about 6 to 8 weeks. Some of the stem cells will have been those already in the liver but some will have come from the blood stream to assist. Of course, the liver is the exception to the rule that if a portion of an organ is removed by trauma or surgery, it will not grow back. Cut off your finger and stem cells will help it to heal but not to grow back to its original state. Adult stem cells are the ones used for treating leukemia, myeloma and other cancers and for correcting certain childhood immune deficiencies. Most often is the use of allogeneic hematopoietic stem cell transplantation, meaning the use of stem cells obtained from a closely matched individual. An identical twin is ideal but few have such a potential donor. Only 25% of siblings will likely match completely. This leaves the use of the National Marrow Donor Registry to find as close a match as possible from unrelated individuals. The Registry has markedly improved the chances for a close match and thus for successful transplantation outcomes. Many parents are now having umbilical cord blood saved and frozen to have available in the unlikely event that their child requires a transplant many years later. Although these cells are identical they usually are not sufficient in numbers to lead to engraftment and often the white blood cells (neutrophils) recover only very slowly leaving a prolonged period of infection risk. Perhaps a technique will be found to get the umbilical stem cells to multiply in the laboratory so that a larger number would be available. Adult stem cells are being used in studies of myocardial infarction and heart failure. Current guidelines of immediate angioplasty and stent insertion as appropriate help protect the heart from permanent damage after an infarct. Still, about 400,000 new cases of heart failure are developing in the USA each year. Long term survival is limited once overt failure develops. Could the damaged heart muscle be fixed? The concept is to use stem cells to repopulate the muscle fibers and to have those cells divide over and over and differentiate into new muscle fibers or perhaps also the small vessels that carry blood to the muscle cells. So far there are some exciting animal studies and even some trials in patients that are encouraging enough to warrant further evaluations. For example, one study uses adult mesenchymal stem cells derived from the bone marrow and infused intravenously within 7 days after a heart attack. 42 centers are collaborating in this double blind, randomized trail in conjunction with Osiris Therapeutics. 220 patients will receive either the stem cells or a placebo and then be monitored with various imaging and functional studies. So, stay tuned. Another common albeit less lethal problem is loss of bladder control leading to incontinence. There are studies in progress to determine if stem cells placed into the bladders sphincter muscle will help it regain control. The adult stem cells are obtained from a leg muscle biopsy. Stem cells are isolated and allowed to grow in tissue culture. These are then injected into the weakened bladder sphincter muscle. Once again, these are studies just beginning but with intriguing early results. Here is another bladder repair concept. When the bladder muscle is weak or largely missing in children it may be possible to literally rebuild the bladder by tissue engineering. A biopsy of the bladder yields cells that can be grown in the laboratory to large numbers. They can then be placed on a biodegradable scaffold and grown further. In time they seem to create a new bladder muscle wall complete with blood vessels. This layer of cells can be implanted in the bladder of children with a defect. Once more I need to note that it is still early days in these studies but they do raise exciting possibilities. The message here is that adult stem cells are being used today for life threatening and life impairing diseases with excellent success and are being studied in other diseases with exciting prospects for the future.

Stephen C Schimpff, MD is an internist, professor of medicine and public policy, former CEO of the University of Maryland Medical Center and is chair of the advisory committee for Sanovas, Inc. and senior advisor to Sage Growth Partners. He is the author of The Future of Medicine Megatrends in Healthcare and The Future of Health Care Delivery- Why It Must Change and How It Will Affect You from which this post is partially adapted. Updates are available at http://medicalmegatrends.blogspot.com

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Medical Megatrends – Stem Cells – Part II of III

Stemcells, Inc. Reports Second Quarter 2012 Financial Results and Provides Business Update

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NEWARK, Calif., Aug. 2, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM), a leading stem cell company developing and commercializing novel cell-based therapeutics and tools for use in stem cell-based research and drug discovery, today reported financial results for the second quarter ended June 30, 2012 and provided a business update.

"We continue to be encouraged by our progress in developing cell-based therapeutics for a broad array of disorders affecting the central nervous system," said Martin McGlynn, President and CEO of StemCells, Inc. "We have already reported top line results from our Phase I PMD trial and I am pleased to report that the manuscript with the complete PMD trial data is under peer review for publication by a top tier journal.

Our other clinical development efforts also continue to advance. We recently reported interim safety data from our chronic spinal cord injury trial, showing that our cells and the procedure have been well tolerated. We have also initiated a Phase I/II trial in dry AMD and look forward to enrolling our first patients in this study soon. Lastly, we recently reported preclinical data showing that our cells restored memory in two animal models relevant to Alzheimer's disease without having to reduce beta-amyloid or tau burden that are the pathological hallmarks of the disease. Results of this kind underscore the potential of our HuCNS-SC cells to potentially address a number of devastating CNS disorders.

Financially, we aim to do more with less and continue to carefully manage our burn rate. Last week's decision by CIRM to award us a $20 million disease team award is exciting and welcome. This award will not only provide additional resources, but is also a vote of confidence in our technology, our program and our people. Moving forward, we will continue to generate clinical data from our HuCNS-SC program in a thoughtful, cost effective manner, which is, we believe, the best pathway to grow shareholder value."

Second Quarter and Recent Business Highlights

Second Quarter Financial Results

Revenue from product sales increased 14% to $211,000 in the second quarter of 2012 compared to the same period of 2011 as our SC Proven media and reagents business continued to see increased unit volume. Total revenue in the second quarter of 2012 was $249,000, compared to $234,000 in the same period of 2011.

Our operating expenses decreased 24% to $5,535,000 in the second quarter of 2012 compared to the same quarter of 2011. Research and development expenses were 26% lower, and selling, general and administrative expenses were 16% lower, compared to the second quarter of 2011. The significant reduction in operating expenses was primarily attributable to continuing tight cost controls and a number of measures taken last year to reduce infrastructure and overhead costs, including a reduction in workforce implemented in May 2011.

Other income in the second quarter of 2012 was $6,184,000, compared to $3,055,000 in the second quarter of 2011. This increase was primarily due to a decrease in the estimated fair value of warrant liability. Our outstanding warrants are classified as a liability, with subsequent changes in the estimated fair value recorded as income or loss.

Loss from operations in the second quarter of 2012 was $5,350,000, a 25% decrease compared to the same period in 2011. Net income for the quarter was $834,000, or $0.03 per share, compared with a net loss of $4,035,000, or $(0.29) per share, for the second quarter of 2011.

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Osiris Bolsters its Stem Cell Intellectual Property Estate

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COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics, Inc. (OSIR), announced today the expansion of its intellectual property protection around Prochymal (remestemcel-L). The United States Patent and Trademark Office recently granted Osiris two patents that cover multiple mechanisms of action related to cardiac tissue repair. Additionally, Osiris has enhanced its mesenchymal stem cell (MSC) patent estate with the issuance of patents across Europe and Australia covering stem cells expressing all therapeutically useful levels of cell surface receptors for TNF-alpha, a receptor essential to the cell's ability to counteract inflammation. These patents further support Osiris' considerable intellectual property position, which includes 48 issued U.S. patents around the production, composition, testing and use of the mesenchymal stem cell from both allogeneic and autologous sources.

"These recent additions to Osiris patent estate, combined with the existing broad coverage of our pioneering MSC platform technology, reinforce our industry leading IP portfolio and bolster our dominant position regarding the manufacture and use of mesenchymal stem cells for the treatment of a broad range of diseases, said Chris Alder, Chief Intellectual Property Counsel of Osiris. We have invested significant time and resources building our intellectual property estate, and with the commercialization of Prochymal, we are preparing to take the necessary action to enforce our considerable rights.

Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program. With Prochymal (remestemcel-L) entering commerce, Osiris has initiated the process of identifying entities that may be infringing upon its intellectual property rights and will take appropriate action as necessary.

About Prochymal (remestemcel-L)

Prochymal is the worlds first approved drug with a stem cell as its active ingredient. Developed by Osiris Therapeutics, Prochymal is an intravenous formulation of MSCs, which are derived from the bone marrow of healthy adult donors between the ages of 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor. Prochymal is truly an off-the-shelf stem cell product that is stored frozen at the point-of-care and infused through a simple intravenous line without the need to type or immunosuppress the recipient. Prochymal is approved in Canada and New Zealand for the management of acute graft-versus-host disease (GvHD) in children and is available for adults and children in eight countries including the United States, under an Expanded Access Program. Prochymal is currently in a Phase 3 trial for refractory Crohns disease and is also being evaluated in clinical trials for the treatment of myocardial infarction (heart attack) and type 1 diabetes.

About Osiris Therapeutics

Osiris Therapeutics, Inc. is the leading stem cell company, having developed the worlds first approved stem cell drug, Prochymal. The company is focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets. In Biosurgery, Osiris currently markets Grafix for burns and chronic wounds, and Ovation for orthopedic applications. Osiris is a fully integrated company with capabilities in research, development, manufacturing and distribution of stem cell products. Osiris has developed an extensive intellectual property portfolio to protect the company's technology, including 48 U.S. and 144 foreign patents.

Osiris, Prochymal, Grafix and Ovation are registered trademarks of Osiris Therapeutics, Inc. More information can be found on the company's website, http://www.Osiris.com. (OSIRG)

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Osiris Bolsters its Stem Cell Intellectual Property Estate

Gladstone scientists use stem cell technology to tackle Huntington's disease

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Public release date: 28-Jun-2012 [ | E-mail | Share ]

Contact: Diane Schrick diane.schrick@gladstone.ucsf.edu 415-734-2538 Gladstone Institutes

SAN FRANCISCO, CAJune 28, 2012Scientists at the Gladstone Institutes and an international team of researchers have generated a human model of Huntington's diseasedirectly from the skin cells of patients with the disease.

For years, scientists have studied Huntington's disease primarily in post-mortem brain tissue or laboratory animals modified to mimic the disease. Today, in Cell Stem Cell, the international team shows how they developed a human model of Huntington's disease, which causes a diverse range of neurological impairments. The new model should help scientists better understand the development of Huntington'sand provide better ways to identify and screen potential therapeutics for this devastating disease.

This new model comes at a time of concentrated federal efforts to accelerate solutions for diseasesincluding a number of debilitating conditions that touch only small percentages of the population. Last year, the National Institutes of Health consolidated its efforts to attack rare diseases under the new National Center for Translational Sciences.

Huntington's is such a rare disease, although it is the most common inherited neurodegenerative disorder. It afflicts approximately 30,000 people in the United Stateswith another 75,000 people carrying the gene that will eventually lead to it. Caused by a mutation in the gene for a protein called huntingtin, the disease damages brain cells so that people with Huntington's progressively lose their ability to walk, talk, think and reason.

"An advantage of this human model is that we now have the ability to identify changes in brain cells over timeduring the degeneration process and at specific stages of brain-cell development," said Gladstone Senior Investigator Steve Finkbeiner, MD, PhD. "We hope this model will help us more readily uncover relevant factors that contribute to Huntington's disease and especially to find successful therapeutic approaches."

In this research, Dr. Finkbeiner and others took advantage of advanced "reprogramming" techniques pioneered by Gladstone Senior Investigator Shinya Yamanaka, MD, PhD. They reprogrammed skin cells from Huntington's disease patients into stem cells known as induced pluripotent stem cells, or iPS cellswhich can become virtually any cell type in the body. The researchers then instructed the iPS cells to develop into neurons, a key type of brain cell. Importantly, each cell line contained a complete set of the genes from each Huntington's disease patient. Because each patient has a different pattern of disease onset and duration, this model may replicate Huntington's more faithfully than animal models do. The model is likely to prove more useful in understanding the disease's progression.

"The iPS cells will provide insights into Huntington's disease, helping us to develop new therapies and test drug candidates," said Dr. Finkbeiner, who is also a professor of neurology and physiology at the University of California, San Francisco, with which Gladstone is affiliated. "We hope that drugs developed with this new human model will have greater success in clinical trials. The track record of animal models for predicting therapies that will work in people has been poor, making drug discovery for neurodegenerative diseases very costlyand therefore less attractive to drug companies. We hope to change that."

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Medistem Receives Notice of Patent Allowance Covering Fat Stem Cell Therapy of Autoimmune Diseases

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SAN DIEGO CA--(Marketwire -06/29/12)- Medistem Inc. (MEDS) announced today notice of allowance from the United States Patent and Trademark Office (USPTO) for a patent covering the use of fat stem cells, and cells associated with fat stem cells for treatment of diseases related to a dysfunctional immune system. Such diseases include multiple sclerosis, Type 1 diabetes, rheumatoid arthritis and lupus. The allowed patent, entitled "Stem Cell Mediated Treg Activation/Expansion for Therapeutic Immune Modulation" has the earliest priority date of December 2006.

"We have previously published that giving multiple sclerosis patients cells extracted from their own fat tissue, which contains stem cells, appears to confer clinical benefit in a pilot study," said Thomas Ichim, CEO of Medistem. "The current patent that has been allowed, in the broadest interpretation of the claims, gives us exclusive rights to the use of specific types of fat stem cell therapy for autoimmune diseases such as multiple sclerosis."

Subsequent to the filing of the patent application, Medistem together with collaborators at the Lawson Health Sciences Research Institute, Canada, reported data that fat tissue contains high numbers of T regulatory cells, a type of immune cell that is capable of controlling autoimmunity.

This finding was independently confirmed by Dr. Diane Mathis' laboratory at Harvard University, who published a paper in the prestigious journal, Nature Medicine, in which detailed experimental evidence was provided supporting the initial finding that adipose tissue contains high numbers of T regulatory cells. A video describing the paper can be accessed at http://www.youtube.com/watch?v=rEJfGu29Rg8.

The current patent discloses the use of T regulatory cells from fat, combinations with stem cells, and use of fat-derived mononuclear cells. Given that there are currently several groups utilizing this technology in the USA in treating patients, Medistem believes revenue can be generated through enforcement of patent rights.

"Our corporate philosophy has been to remain highly focused on our ongoing clinical stage programs using Medistem's universal donor stem cell, the Endometrial Regenerative Cell (ERC), in the treatment of critical limb ischemia and congestive heart failure," said Dr. Vladimir Bogin, Chairman and President of Medistem. "However, due to the ease of implementation of our fat stem cell technology, combined with the major burden that autoimmune diseases have on our health care system, we are highly incentivized to explore partnering, co-development and licensing opportunities."

Autoimmune conditions occur as a result of the body's immune system "turning on itself" and attacking its own organs or cells. Current treatments for autoimmune conditions are based on "globally" suppressing the immune system by administration of immunosuppressive drugs. This is associated with an increased predisposition to infections and significant side effects. The utilization of stem cells and T regulatory cells offers the potential to selectively suppress pathological immunity while preserving the ability of the body to fight bacteria and viruses. According to the NIH there are approximately 23 million victims of autoimmune conditions.

Links to Documents:

Link to peer-reviewed publication: http://www.translational-medicine.com/content/pdf/1479-5876-7-29.pdf

Link: http://www.marketwire.com/press-release/medistem-files-patent-application-on-therapeutic-cell-population-found-in-fat-tissue-frankfurt-s2u-812298.htm

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Medistem Receives Notice of Patent Allowance Covering Fat Stem Cell Therapy of Autoimmune Diseases

UMass stem cell lab to close

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The laboratory grew and stored human stem cells, which are capable of becoming any cell in the body, and made them available to scientists nationwide for use in experiments to study diseases such as diabetes and spinal cord injuries. When it is dismantled, several thousand vials of stem cellswill be sent back to the research centers where they originated, and the equipment will be given to other UMass labs.

Susan Windham-Bannister, president of the Massachusetts Life Sciences Center, a quasi-public agency that oversees the $1 billion life sciences initiative, defended the decision to initially fund the stem cell bank. She said there are many examples of technology that in hindsight are unnecessary, but at the time it was conceived, when the investment was made, it was absolutely state of the art. The center, she said, was one of them.

Originally, the bank was seen as a repository for embryonic stem cell lines that were being created but were not eligible for federal funding under Bush-era restrictions. The field has evolved significantly since then, with President Obamas loosening of restrictions on federal funding and the development of new technologies for making stem cells.

Still, stem cell banks are seen as useful by some. The California Institute for Regenerative Medicine, for example, is preparing to invest $10 million in its own stem cell banking initiative, and another $20 million to underwrite the creation of stem cells from patients with specific diseases.

Massachusetts Senate minority leader Bruce Tarr, Republican of Gloucester, said he was concerned that lawmakers had not been told the bank would close.

Given the fact that this is a resource that was created by an act of the Legislature, I would hope anyone seeking to change its status would consult with the Legislature, he said. The notion has always been we have been working hard to make Massachusetts a leader in stem cell research, and I dont know how ceasing the operations of the stem cell bank advances that goal.

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UMass stem cell lab to close