NEW YORK, Sept. 19, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE MKT:NBS) ("NeoStem" or the "Company"), a rapidly emerging market leader in the fast growing cell therapy market, today announced that Company management has been invited to participate at BIOX, the Noble Financial Capital Markets' Life Sciences Exposition on Monday, September 24. Company management will make a webcasted company presentation and participate in a cell therapy panel.

Noble Financial Capital Markets Investor Conference - BIOX Life Sciences Exposition

For more information about the conference, please visit http://www.nobleresearch.com/BIOX.htm.

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy, capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT"), with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cell product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a substantial intellectual property estate, we believe we are well positioned to succeed.

For more information on NeoStem, please visit http://www.neostem.com.

Forward-Looking Statements for NeoStem, Inc.

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, including with respect to the Company's or its partners' successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company's ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company's actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors, including but not limited to (i) the Company's ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company's clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company's ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company's ability to protect its intellectual property, (xi) the company's ability to successfully divest its interest in Erye, and (xii) matters described under the "Risk Factors" in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company's other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.

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NeoStem to Present at Noble Capital Markets' Life Sciences Exposition on September 24

Public release date: 2-Sep-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-206-2805 University of California - Los Angeles Health Sciences

UCLA researchers have discovered a type of cell that is the "missing link" between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function.

The studies were done using human bone marrow, which contains all the stem cells that produce blood during postnatal life.

"We felt it was especially important to do these studies using human bone marrow as most research into the development of the immune system has used mouse bone marrow," said study senior author Dr. Gay Crooks, co-director of the Eli and Edythe Broad Center of Regenerative Medicine and a co-director of the Cancer and Stem Cell Biology program at UCLA's Jonsson Comprehensive Cancer Center. "The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of postnatal life."

The research team was "intrigued to find this particular bone marrow cell because it opens up a lot of new possibilities in terms of understanding how human immunity is produced from stem cells throughout life," said Crooks, a professor of pathology and pediatrics.

Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, or cancers of the blood.

The study appears Sept. 2 in the early online edition of Nature Immunology.

Before this study, researchers had a fairly good idea of how to find and study the blood stem cells of the bone marrow. The stem cells live forever, reproduce themselves and give rise to all the cells of the blood. In the process, the stem cells divide and produce intermediate stages of development called progenitors, which make various blood lineages like red blood cells or platelets. Crooks was most interested in the creation of the progenitors that form the entire immune system, which consists of many different cells called lymphocytes, each with a specialized function to fight infection.

"Like the stem cells, the progenitor cells are also very rare, so before we can study them we needed to find the needle in the haystack." said Lisa Kohn, a member of the UCLA Medical Scientist Training Program and first author in the paper.

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UCLA researchers discover missing link between stem cells and immune system

17-08-2012 13:38 The biggest thing we've noticed is her ability to track people and her vision. Her cognitive skills have improved. Before her stem cell treatment 7 months ago, she was like a 50 watt light bulb and she is like a 200 watts in comparison. She reacts more, holds her head up more and her hands are nice and open now, not fisted like before. Hand to mouth motion is much easier for her to do. Her range of motion, in general, is much better. She can now raise her hands over her head and she was never able to do that before. Her therapists have seen dramatic changes. Our family has noticed changes. The neurologist has noticed changes. We are very thankful that we were able to get this treatment for her in Panama. We couldn't imagine her not being who she is now. She is 200 times better than what she was.

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Stem Cell Therapy for Cerebral Palsy in Panama - Video

NEW YORK, Aug. 15, 2012 (GLOBE NEWSWIRE) -- Amorcyte, a company of NeoStem, Inc. (NYSE MKT:NBS) ("NeoStem" or the "Company"), a rapidly emerging market leader in the fast growing cell therapy market, today announced that it received on August 9, 2012 approval to continue its PreSERVE AMI Phase 2 clinical trial following its first interim data and safety review by the Data Safety Monitoring Board (DSMB). The PreSERVE trial is a Phase 2, randomized, placebo controlled, double-blind study expected to include 160 patients at more than 40 clinical sites. The trial's product candidate, AMR-001, is designed to prevent major adverse cardiac events following acute myocardial infarction (AMI). Patient enrollment for the PreSERVE trial began in January 2012 and the Company anticipates completing enrollment in 2013 with six months initial data readout near the end of 2013.

"We are pleased that, similar to our Phase 1 trial, the first external review of our Phase 2 trial data confirms that there are no safety signals that would preclude the trial from continuing as planned," said Andrew L. Pecora, M.D. FACP CPE, Chief Medical Officer of NeoStem. "The PreSERVE AMI study to date indicates that multiple National Study sites are capable of acquiring the necessary volume of bone marrow to create the AMR-001 product five to seven days after an AMI in a safe and practical manner, and once created the product can be delivered and administered without a safety signal."

NeoStem management believes that cell therapy is a disruptive technology in the $50 billion worldwide regenerative medicine market. Many key opinion leaders in the scientific, medical and investment communities consider AMR-001 to be best in class. Peak annual worldwide sales of AMR-001 for this indication could exceed $1 billion based upon a conservative market penetration of its qualified target patient population. AMR-001 is protected by two issued and multiple pending U.S. patents with corresponding patent coverage in selected markets around the world. The Amorcyte AMR-001 product development program also extends to congestive heart failure (CHF). The Company is preparing to launch its CHF Phase 1 clinical trials in early 2013. The worldwide CHF patient population is estimated to be four times larger than that of AMI.

About NeoStem, Inc.

NeoStem, Inc. ("we," "NeoStem" or the "Company") continues to develop and build on its core capabilities in cell therapy to capitalize on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. Our January 2011 acquisition of Progenitor Cell Therapy, LLC ("PCT") provides NeoStem with a foundation in both manufacturing and regulatory affairs expertise. We believe this expertise, coupled with our existing research capabilities and collaborations, will allow us to achieve our mission of becoming a premier cell therapy company. Our PCT subsidiary's manufacturing base is one of the few current Good Manufacturing Practices ("cGMP") facilities available for contracting in the burgeoning cell therapy industry. Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011, is developing a cell therapy for the treatment of cardiovascular disease. Amorcyte's lead compound, AMR-001, represents NeoStem's most clinically advanced therapeutic and Amorcyte is enrolling patients for a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. We also expect to begin a Phase 1 clinical trial in 2013 to investigate AMR-001's utility in arresting the progression of congestive heart failure and the associated comorbidities of that disease. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is engaged in collaboration with Becton-Dickinson that is exploring the earlier stage clinical development of a T-cell therapy for autoimmune conditions. In addition, our pre-clinical assets include our VSELTM Technology platform as well as our MSC (mesenchymal stem cells) product candidate for regenerative medicine.

For more information on NeoStem, please visit http://www.neostem.com.

Forward-Looking Statements for NeoStem, Inc.

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, including with respect to the Company's or its partners' successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company's ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward-looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company's actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors, including but not limited to (i) the Company's ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company's clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company's ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company's ability to protect its intellectual property; (xi) the company's ability to successfully divest its interest in Erye, and (xii) matters described under the "Risk Factors" in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company's other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.

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NeoStem Reports Data Safety Monitoring Board Recommends Continuation of PreSERVE AMI Phase 2 Trial

A GROWING number of overseas clinics touting stem cell therapy for conditions ranging from sexual disorders to HIV are targeting Australia, where such treatments are restricted.

Australian scientists have raised concerns about so-called ''stem cell tourism'', saying many of the treatments offered are unproven, untested and potentially deadly.

The Swiss firm Fetal Cell Technologies International has been advertising in Australia since last year and Emcell, based in Ukraine, started promoting its services last month.

It is estimated as many as 200 Australians have travelled overseas for the therapy. The secretary for science policy at the Australian Academy of Science, Bob Williamson, said he empathised with the desperation of seriously ill people but warned against the unproven therapies, which can cost up to $60,000.

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''The therapies are almost all untested and unproven and sometimes they have killed people,'' Professor Williamson said. The Sun-Herald's calls to Emcell's Melbourne office were not returned.

Stem Cells Australia's Megan Munsie, who is conducting a study into stem cell tourism with Monash University, said many people she interviewed were unaware of the risks of therapy overseas.

''We're not talking about rubbing something into your skin or taking a capsule, we are talking about often a very invasive procedure,'' she said.

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Fears over 'stem cell tourism'

Johns Hopkins researchers have discovered that a single protein molecule may hold the key to turning cardiac stem cells into blood vessels or muscle tissue, according to a release from the university. This finding may lead to better ways to treat heart attack patients.

Human heart tissue typically forms scars rather than healing well after an attack. However, stem cells have been shown improve the repair process by turning into the cells that make up healthy heart tissue, including heart muscle and blood vessels. The recent discovery of a master molecule that guides the destiny of these stem cells has the potential to result in even more effective treatments for heart patients, the Johns Hopkins researchers say.

In a study published in the June 5 online edition of journal Science Signaling, the Johns Hopkins team reported that tinkering with a protein molecule called p190RhoGAP shaped the development of cardiac stem cells and prodded them to become the building blocks for either blood vessels or heart muscle. The scientists said that by altering levels of this protein, they were able to affect the future of these stem cells. In biology, finding a central regulator like this is like finding a pot of gold, said Andre Levchenko, a biomedical engineering professor and member of the Johns Hopkins Institute for Cell Engineering, who supervised the research effort.

The lead author of the journal article, Kshitiz, a postdoctoral fellow who uses only his first name, said, Our findings greatly enhance our understanding of stem cell biology and suggest innovative new ways to control the behavior of cardiac stem cells before and after they are transplanted into a patient. This discovery could significantly change the way stem cell therapy is administered in heart patients.

More:
"Master Molecule" May Help Heart Treatment

MANILA, Philippines Stem cell therapy, aside from being a potential cure for a wide range of illnesses, can also make a patient look and feel younger, a stem cell therapist said.

Dr. Ricardo Quiones, a cosmetic surgeon and dermatologist, has trained to conduct stem cell therapy, which he describes as the future of medicine.

Quiones said stem cell therapy has become popular for its ability to regenerate and heal properties of adult stem cells.

As we grow old, our stem cells dramatically decline. When we were children, we had 80 million stem cells. As we reach the age of 40, our stem cells decline to 35 million, he told Mornings@ANC on Friday.

Quiones explained that the procedure is similar to turning back the clock because it can increase a persons stem cells to 100 million.

Ive done two patients from Zamboanga City. I called them up after the procedure and they told me they look younger. They have the stamina, the vigor and they have felt an increase in short-term memory, powers of attention and concentration, he said.

Quiones also said the procedure has the potential to cure diabetes, heart damage, brain damage such as Parkinsons and Alzheimers, osteoarthritis, stroke, baldness and even sports injuries.

3-hour procedure

Quiones said any patient, except those diagnosed with cancer, can undergo the procedure, which he said will only last for about 3 to 4 hours.

After receiving clearance from a physician and passing medical and laboratory tests, anesthesia will be administered to a patient before stem cells are harvested.

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Stem cell therapy 'turns back clock'

ScienceDaily (July 3, 2012) searchers from the University of Maryland School of Maryland report promising results from using adult stem cells from bone marrow in mice to help create tissue cells of other organs, such as the heart, brain and pancreas -- a scientific step they hope may lead to potential new ways to replace cells lost in diseases such as diabetes, Parkinson's or Alzheimer's.

The research in collaboration with the University of Paris Descartes is published online in the June 29, 2012 edition of Comptes Rendus Biologies, a publication of the French Academy of Sciences.

"Finding stem cells capable of restoring function to different damaged organs would be the Holy Grail of tissue engineering," says lead author David Trisler, PhD, assistant professor of neurology at the University of Maryland School of Medicine.

He adds, "This research takes us another step in that process by identifying the potential of these adult bone marrow cells, or a subset of them known as CD34+ bone marrow cells, to be 'multipotent,' meaning they could transform and function as the normal cells in several different organs."

University of Maryland researchers previously developed a special culturing system to collect a select sample of these adult stem cells in bone marrow, which normally makes red and white blood cells and immune cells. In this project, the team followed a widely recognized study model, used to prove the multipotency of embryonic stem cells, to prove that these bone marrow stem cells could make more than just blood cells. The investigators also found that the CD34+ cells had a limited lifespan and did not produce teratomas, tumors that sometimes form with the use of embryonic stem cells and adult stem cells cultivated from other methods that require some genetic manipulation.

"When taken at an early stage, we found that the CD34+ cells exhibited similar multipotent capabilities as embryonic stem cells, which have been shown to be the most flexible and versatile. Because these CD34+ cells already exist in normal bone marrow, they offer a vast source for potential cell replacement therapy, particularly because they come from a person's own body, eliminating the need to suppress the immune system, which is sometimes required when using adults stem cells derived from other sources," explains Paul Fishman, MD, PhD, professor of neurology at the University of Maryland School of Medicine.

The researchers say that proving the potential of these adult bone marrow stem cells opens new possibilities for scientific exploration, but that more research will be needed to see how this science can be translated to humans.

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Adult stem cells from bone marrow: Cell replacement/tissue repair potential in adult bone marrow stem cells in animal ...

FOR IMMEDIATE RELEASE: July 3, 2012

UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE INVESTIGATORS FIND CELL REPLACEMENT/ TISSUE REPAIR POTENTIAL IN ADULT BONE MARROW STEM CELLS IN ANIMAL MODEL

Scientists Looking for Potential Avenue to Grow Cells of Different Organs

Newswise Baltimore, MD July 3, 2012. Researchers from the University of Maryland School of Maryland report promising results from using adult stem cells from bone marrow in mice to help create tissue cells of other organs, such as the heart, brain and pancreas - a scientific step they hope may lead to potential new ways to replace cells lost in diseases such as diabetes, Parkinsons or Alzheimers. The research in collaboration with the University of Paris Descartes is published online in the June 29, 2012 edition of Comptes Rendus Biologies, a publication of the French Academy of Sciences.

Finding stem cells capable of restoring function to different damaged organs would be the Holy Grail of tissue engineering, says lead author David Trisler, PhD, assistant professor of neurology at the University of Maryland School of Medicine.

He adds, This research takes us another step in that process by identifying the potential of these adult bone marrow cells, or a subset of them known as CD34+ bone marrow cells, to be multipotent, meaning they could transform and function as the normal cells in several different organs.

University of Maryland researchers previously developed a special culturing system to collect a select sample of these adult stem cells in bone marrow, which normally makes red and white blood cells and immune cells. In this project, the team followed a widely recognized study model, used to prove the multipotency of embryonic stem cells, to prove that these bone marrow stem cells could make more than just blood cells. The investigators also found that the CD34+ cells had a limited lifespan and did not produce teratomas, tumors that sometimes form with the use of embryonic stem cells and adult stem cells cultivated from other methods that require some genetic manipulation.

When taken at an early stage, we found that the CD34+ cells exhibited similar multipotent capabilities as embryonic stem cells, which have been shown to be the most flexible and versatile. Because these CD34+ cells already exist in normal bone marrow, they offer a vast source for potential cell replacement therapy, particularly because they come from a persons own body, eliminating the need to suppress the immune system, which is sometimes required when using adults stem cells derived from other sources, explains Paul Fishman, MD, PhD, professor of neurology at the University of Maryland School of Medicine.

The researchers say that proving the potential of these adult bone marrow stem cells opens new possibilities for scientific exploration, but that more research will be needed to see how this science can be translated to humans.

The results of this international collaboration show the important role that University of Maryland School of Medicine researchers play in advancing scientific understanding, investigating new avenues for the development of potentially life-changing treatments, says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs at the University of Maryland and the John Z. and Akiko K. Bowers Distinguished Professor and dean of the University of Maryland School of Medicine.

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Study Results: Adult Stem Cells From Bone Marrow

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

Osiris Therapeutics, Inc. (OSIR), announced today interim one-year results from its groundbreaking clinical trial evaluating Prochymal (remestemcel-L) for the treatment of patients experiencing first-time acute myocardial infarction. The trial is the largest study of allogeneic or "off-the-shelf" stem cells ever conducted in heart attack patients. A total of 220 patients were given a single infusion of either Prochymal or placebo through a standard intravenous line within seven days of an acute heart attack.

Cardiac MRI assessments were conducted for six months following infarct to evaluate cardiac remodeling. Patients receiving Prochymal had significantly less cardiac hypertrophy, as measured by cardiac MRI, compared to patients receiving placebo (p<0.05). Patients treated with Prochymal also experienced significantly less stress-induced ventricular arrhythmia (p<0.05). Cardiac hypertrophy and ventricular arrhythmia are indicators of pathological remodeling following heart injury and provide insight into the mechanism by which mesenchymal stem cells attenuate heart injury following a myocardial infarction.

The mechanistic data is complemented by clinical data showing treatment with Prochymal resulted in a statistically significant reduction in heart failure. In the study, seven patients who were treated with placebo have progressed to heart failure requiring treatment with intravenous diuretics, compared to none of the Prochymal patients (p=0.01). Furthermore, patients receiving placebo tended to require re-hospitalization for cardiac issues sooner than the patients receiving Prochymal (median 27.5 days vs. 85.5 days).

This study is the largest of its kind and provides key insights into the mechanism of action of mesenchymal stem cells in the setting of acute myocardial infarction, said Lode Debrabandere, Ph.D., Senior Vice President of Therapeutics at Osiris. These important mechanistic observations are consistent with data obtained from our preclinical models and from the first placebo-controlled human trial with Prochymal published in the Journal of the American College of Cardiology. Given the quality of the data and highly encouraging results observed thus far, we are extending the trial's duration to capture a better understanding of the long-term clinical benefits of MSCs."

The trial also demonstrated that treatment with Prochymal was safe. There were no infusional toxicities observed in patients receiving Prochymal. Serious adverse events occurred with equal frequency in both treatment groups (31.8%). To date, there have been 5 deaths in the trial, 2 in the Prochymal group and 3 in the placebo group.

For interventional cardiologists, keeping our myocardial infarction patients from progressing to heart failure is central to our mission, said Mark Vesely, M.D., Principal Investigator on the Study and Assistant Professor of Medicine (Interventional Cardiology) at the University of Maryland School of Medicine. It is remarkable and very encouraging to see significant changes in clinically meaningful parameters this early in the study. We look forward to the additional data that will be gathered as the study progresses, which will help us to better understand both the magnitude and durability of the benefit to treatment.

Prochymal, the worlds first and only stem cell drug approved by an internationally recognized regulatory authority, is used for the treatment of graft vs. host disease (GvHD). GvHD is a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected. 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 (EAP).

About the Trial

This Phase 2, multi-center, randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of Prochymal (ex-vivo cultured adult human mesenchymal stem cells) intravenous infusion following acute myocardial infarction. A total of 220 patients were randomized (1:1) at 33 centers in the United States and Canada and received a single intravenous infusion of Prochymal or placebo within 7 days following first acute myocardial infarction. In addition to screening and baseline visits prior to the infusion, initially follow-up evaluations were scheduled to be conducted through 2 years. Given the encouraging results observed at the one year time-point, the trial is being extended to include 5 years of follow-up. Both male and female subjects between 21 and 85 years of age were enrolled. Patients had to have a left ventricular ejection fraction (LVEF) between 20% and 45% as determined by quantitative echocardiography or cardiac MRI at least 24 hours after successful reperfusion of the culprit vessel. In addition, troponin levels must have been greater than 4 times the upper limit of normal during the first 72 hours of hospitalization for the MI.

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Prochymal Significantly Reduces Hypertrophy, Arrhythmia and Progression to Heart Failure in Patients Suffering a Heart ...

The Irish Times - Monday, July 2, 2012

LORNA SIGGINS

WORLD leaders in stem-cell technology are due to exchange knowledge of potential treatments at a conference opening in NUI Galway today.

Researchers from NUIG, University College Cork and NUI Maynooth will participate in the event, which has been billed as the first major conference on stem-cell therapy in Ireland.

Prof Anthony Hollander of the University of Bristol, England who was one of a team which successful created and then transplanted the first tissue-engineered trachea or windpipe is among a number of international speakers presenting findings.

The gathering will focus on the realities of stem-cell treatment, Prof Frank Barry, director of NUIGs National Centre for Biomedical Engineering Science has said.

The therapy is complex and controversial, and sometimes exaggerated claims are made, he said.

The researchers are specialists in Mesenchymal, or adult, stem cells, and will be concentrating on what is likely in the future, he added.

The list of conditions which could be treated successfully by stem cells is small, but growing, Prof Barry said.

Leukaemia and other diseases of the blood appear to respond best.

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Stem-cell research leaders to meet in NUIG

Editor's Choice Main Category: Muscular Dystrophy / ALS Also Included In: Transplants / Organ Donations Article Date: 29 Jun 2012 - 11:00 PDT

Current ratings for: Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice

A new study published in Science Translational Medicine reveals that researchers have, for the first time, managed to turn fibroblast cells, i.e. common cells within connective tissue, from muscular dystrophy patients into stem cells and subsequently changed these cells into muscle precursor cells. After modifying the muscle precursor cells genetically, the researchers transplanted them into mice.

In future, this new technique could be used in order to treat patients with the rare condition of limb-girdle muscular dystrophy, which primarily affects the shoulders and hips, and maybe other types of muscular dystrophies. The method was initially developed in Milan at the San Raffaele Scientific Institute and was completed at UCL.

Muscular dystrophy is a genetic disorder, which typically affects skeletal muscles. The condition leads to severely impaired mobility and can, in severe cases result in respiratory and cardiac dysfunction. At present, there is no effective treatment for the condition. A number of new potential therapies, including cell therapy, are entering clinical trials.

The scientists of this study concentrated their research on genetically modifying mesoangioblasts, i.e. a self-renewing cell that originates from the dorsal aorta and differentiates into most mesodermal tissues, which demonstrated its potential for treating muscular dystrophy in earlier studies.

Given that the muscles of patients with muscular dystrophy are depleted of mesonangioblasts, the researchers were unable to obtain sufficient numbers of these cells from patients with limb-girdle muscular dystrophy, and therefore "reprogrammed" adult cells from these patients into stem cells, which enabled them to prompt them to differentiate into mesoangioblast-like cells.The team then genetically corrected these 'progenitor' cells by using a viral vector, and injected them into mice with muscular dystrophy so that the cells targeted damaged muscle fibers.

In a mice study, the same process demonstrated that dystrophic mice were able to run on a treadmill for longer a longer time than dystrophic mice that did not receive the cells.

Research leader, Dr Francesco Saverio Tedesco, from UCL Cell & Developmental Biology, who led the study, explained:

Professor Giulio Cossu, also an author at UCL, concluded:

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Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice

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

Contact: Tom Vasich tmvasich@uci.edu 949-824-6455 University of California - Irvine

Irvine, Calif., June 28, 2012 An international consortium of Huntington's disease experts, including several from the Sue & Bill Gross Stem Cell Research Center at UC Irvine, has generated a human model of the deadly inherited disorder directly from the skin cells of affected patients.

The re-created neurons, which live in a petri dish, will help researchers better understand what disables and kills brain cells in people with HD and let them gauge the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

UCI scientists were part of a consortium that in 1993 identified the autosomal dominant gene mutation responsible for HD, but there is still no cure, and no treatments are available to even slow its onset or progression. The research, published online today in the journal Cell Stem Cell, is the work of the Huntington's Disease iPSC Consortium. Participants examined several other cell lines and control cell lines to ensure that their results were consistent and reproducible in different labs.

"Our discovery will enable us for the first time to test therapies on human Huntington's disease neurons," said Leslie Thompson, UCI professor of psychiatry & human behavior and neurobiology & behavior, one of the world's leading HD experts and a senior author of the study. "This has been a remarkable time in HD research, with the advent of stem cell technologies that have allowed these scientific advancements. Also, having a team of scientists working together as a consortium has benefited the research tremendously and accelerated its pace."

Leslie Lock, a UCI assistant professor of developmental & cell biology and biological chemistry whose lab helped develop the induced pluripotent stem cells (iPSC), added: "It's exciting to be carrying out work that provides hope for HD patients and their families."

Thompson said that UCI scientists will use the new model to study the specific gene expression changes in human brain cells that trigger the onset of HD, helping them understand how these changes happen and how to correct them.

Huntington's disease afflicts about 30,000 people in the U.S. typically striking in midlife and another 75,000 carry 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 individuals with HD progressively lose their ability to walk, talk and reason. It invariably culminates in death. While rare, HD is the most common inherited neurodegenerative disease.

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Human model of Huntington's disease created from skin's stem cells

Newswise LOS ANGELES (EMBARGOED UNTIL NOON EDT ON JUNE 28, 2012) Cedars-Sinai scientists have joined with expert colleagues around the globe in using stem cells to develop a laboratory model for Huntingtons disease, allowing researchers for the first time to test directly on human cells potential treatments for this fatal, inherited disorder.

As explained in a paper published June 28 on the Cell Stem Cell website and scheduled for print in the journals Aug. 3 issue, scientists at Cedars-Sinais Regenerative Medicine Institute and the University of Wisconsin took skin cells from patients with Huntingtons disease and reprogrammed them into powerful stem cells; these were then made into the nervous system cells affected by the disease. Seven laboratories around the world collaborated to demonstrate the cells had hallmarks of Huntingtons.

This Huntingtons disease in a dish will enable us for the first time to test therapies on human Huntingtons disease neurons, said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a senior author of the study. In addition to increasing our understanding of this disorder and offering a new pathway to identifying treatments, this study is remarkable because of the extensive interactions between a large group of scientists focused on developing this model. Its a new way of doing trailblazing science.

The Huntingtons Disease iPSC Consortium united some of the worlds top scientists working on this disease. Cedars-Sinai researchers took skin cells from a several Huntingtons patients, including a six-year-old with a severe juvenile form of the disease. They genetically reprogrammed these tissues into induced pluripotent stem cells, which can be made into any type of cell in the body. The cells lines were banked by scientists at Cedars-Sinai and scrutinized by all consortium members for differences that may have led to the disease. These cell lines are now an important resource for Huntingtons researchers and have been made available via a National Institutes of Health-funded repository at Coriell Institute for Medical Research in New Jersey.

Huntingtons, known to the public, for example, as the cause of folksinger Woody Guthries death, typically strikes patients in midlife. It causes jerky, twitching motions, loss of muscle control, psychiatric disorders and dementia; the disease ultimately is fatal. In rare, severe cases, the disorder appears in childhood.

Researchers believe that Huntingtons results from a mutation in the huntintin gene, leading to production of an abnormal protein and ultimately cell death in specific areas of the brain that control movement and cognition. There is no cure for Huntingtons, nor therapies to slow its progression.

The consortium showed Huntingtons cell deficits or how they differ from normal cells, including that they were less likely to survive cultivation in the petri dish. Scientists tried depriving them of a growth factor present around normal cells, or stressing them, and found that Huntingtons neurons died even faster.

It was great that these characteristics were seen not only in our laboratory, but by all of the consortium members using different techniques, said Virginia Mattis, a post-doctoral scientist at the Cedars-Sinai Regenerative Medicine Institute and one of the lead authors of the study. It was very reassuring and significantly strengthens the value of this study.

This new model will provide the foundation for a new round of experiments by the consortium funded by a new grant from the NIH and the California Institute for Regenerative Medicine.

The Cedars-Sinais Regenerative Medicine Institute has made a major commitment to projects like this Huntingtons study in which stem cell research helps to advance understanding of human disease and open new and innovative methods to identify treatments and cures. The institute has developed an induced pluripotent stem cell core facility and recruited faculty to work in this emerging area of regenerative medicine research.

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Cedars-Sinai Researchers, with Stem Cells and Global Colleagues, Develop Huntington's Research Tool

COLORADO SPRINGS, Colo.--(BUSINESS WIRE)--

HemoGenix announced today that FDA CBER has given HemoGenix its first Master File Number for an in vitro blood stem cell potency, quality and release assay (HALO-96 PQR) (1)for cellular therapy products(2)used for stem cell transplantation purposes. HALO-96 PQR is the first commercially available stem cell potency assay for cellular therapy products. It incorporates the most sensitive readout available to measure changes in the cells energy source (ATP) as a function of the potential for stem cells to proliferate. Potency and quality of stem cell therapeutic products are required to be measured prior to use to help predict the engraftment of the cells in the patient. At the present time, tests such as cell number, viability and a stem cell marker called CD34 are routinely used. However, none of these tests specifically measure stem cells and none determine the stem cell biological activity required for a potency assay. The only cell functionality test presently used in this field, especially for umbilical cord blood transplantation, is the colony-forming unit (CFU) assay, which is subjective, non-validated and has been used since the early 1970s. HALO-96 PQR changes this paradigm. It is particularly needed in the umbilical cord blood stem cell transplantation field by providing an application-specific test incorporating all of the compliance characteristics required not only by regulatory agencies(3) and standards organizations, but also the cord blood community(4).

Stem cell potency is one of the most important parameters necessary for any therapeutic product, especially stem cells. Without it, the dose cannot be defined and the transplantation physician has no indication as to whether the product will engraft in the patient. The number of cord blood units collected and stored and the number of cord blood stem cell transplantations have increased exponentially over the last 12 years. During this time, significant advancements have been made in pre- and post stem cell transplantation procedures. Yet the tests used during the preparation and processing of the cells have remained unchanged and do not even measure the biological functionality of the stem cells being transplanted. Indeed, the standards organizations responsible for applying regulatory guidance to the community have so far failed to allow any new and alternative assays to be used during cord blood processing. HALO-96 PQR is the first test that actually quantitatively characterizes and defines the stem cells in cord blood, mobilized peripheral blood or bone marrow as high quality and potent active ingredients for release prior to transplantation. Presently, approximately 20% engraftment failure is encountered in cord blood transplantation. HALO-96 PQR could help reduce the risk of engraftment failure by providing valuable and time-sensitive information on the stem cells prior to use. HALO-96 PQR complies with the guidelines not only with the cord blood community, but also with regulatory agencies thereby providing a benefit to both the stem cell transplantation center and the patient, said Ivan Rich, Founder and CEO of HemoGenix (www.hemogenix.com).

About HemoGenix, Inc.

HemoGenix is a privately held Contract Research Service and Assay Development Laboratory based in Colorado Springs, Colorado. Specializing in predictive in vitro stem cell toxicity testing, HemoGenix provides its services to small, medium and many of the largest biopharmaceutical companies. HemoGenix has developed several assays for stem cell therapy and regenerative medicine applications. These and other patented and proprietary assays are manufactured and produced in Colorado Springs and sold worldwide. HemoGenix has been responsible for changing the paradigm and bringing in vitro stem cell hemotoxicity testing into the 21st century. With HALO-96 PQR the company is now also changing the paradigm to become a leader in stem cell therapy assays. To this end, HemoGenix is a member of the Alliance for Regenerative Medicine and working with other companies to decrease risk and improve safety for the patient.

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Newswise If you break a bone, you know you'll end up in a cast for weeks. But what if the time it took to heal a break could be cut in half? Or cut to just a tenth of the time it takes now? Qian Wang, a chemistry professor at the University of South Carolina, has made tantalizing progress toward that goal.

Wang, Andrew Lee and co-workers just reported in Molecular Pharmaceutics that surfaces coated with bionanoparticles could greatly accelerate the early phases of bone growth. Their coatings, based in part on genetically modified Tobacco mosaic virus, reduced the amount of time it took to convert stem cells into bone nodules from two weeks to just two days.

The key to hastening bone healing or growth is to coax a perfectly natural process to pick up the pace.

"If you break a rib, or a finger, the healing is automatic," said Wang. "You need to get the bones aligned to be sure it works as well as possible, but then nature takes over."

Healing is indeed very natural. The human body continuously generates and circulates cells that are undifferentiated; that is, they can be converted into the components of a range of tissues, such as skin or muscle or bone, depending on what the body needs.

The conversion of these cells called stem cells is set into motion by external cues. In bone healing, the body senses the break at the cellular level and begins converting stem cells into new bone cells at the location of the break, bonding the fracture back into a single unit. The process is very slow, which is helpful in allowing a fracture to be properly set, but after that point the wait is at least an inconvenience, and in some cases highly detrimental.

"With a broken femur, a leg, you can be really incapacitated for a long time," said Wang. "In cases like that, they sometimes inject a protein-based drug, BMP-2, which is very effective in speeding up the healing process. Unfortunately, it's very expensive and can also have some side effects."

In a search for alternatives four years ago, Wang and colleagues uncovered some unexpected accelerants of bone growth: plant viruses. They originally meant for these viruses, which are harmless to humans, to work as controls. They coated glass surfaces with uniform coverings of the Turnip yellow mosaic virus and Tobacco mosaic virus, originally intending to use them as starting points for examining other potential variations.

But they were surprised to find that the coatings alone could reduce the amount of time to grow bone nodules from stem cells. Since then, Wang and co-workers have refined their approach to better define just what it is that accelerates bone growth.

Over the course of the past four years, they've demonstrated that it's a combination of the chemistry as well as the topography of the surface that determines how long it takes a stem cell to form bone nodules. The stem cells are nestled into a nanotopgraphy defined by the plant virus, and within that nanotopography the cells make contact with the variety of chemical groups on the viral surface.

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Speeding Up Bone Growth by Manipulating Stem Cells

Public release date: 22-Jun-2012 [ | E-mail | Share ]

Contact: William Gilroy gilroy.6@nd.edu 574-631-4127 University of Notre Dame

Alumnus Michael Gallagher and his wife, Elizabeth, have made a $5 million gift to establish the Elizabeth and Michael Gallagher Family Professorships in Adult Stem Cell Research at the University of Notre Dame.

Their gift, which will fund three new endowed professorships in adult and all forms of non-embryonic stem cell research, will strengthen Notre Dame's leadership in the field of stem cell research and enhance the University's effective dialogue between the biomedical research community and the Catholic Church on matters related to the use and application of stem cells and regenerative medicine.

"As a Catholic university, Notre Dame carries a mantle of responsibility to use our scholarship and resources to help alleviate human suffering, and, in this area of research in particular, to do so with deep respect for the sanctity of all human life," said Rev. John I. Jenkins, C.S.C., the University's president. "These new professorships will enable us to effectively build upon an already strong foundation in this critically important field. We are tremendously grateful to the Gallaghers for making this possible with their transformative gift."

Despite years of research, there are no known cures for a large number of degenerative diseases, such as Type 1 diabetes, Parkinson's disease, cardiovascular disease, macular degeneration and spinal cord injuries. Stem cell research has the potential to contribute to the discovery of new and successful treatments for these and other diseases because it holds the unique promise of regenerating damaged cells and tissues, fully restoring tissues and organs to their normal function.

Although this vital area of research could accelerate the ability to alleviate much human suffering, it has generated extensive ethical debate with the use of embryonic versus non-embryonic stem cells. The Catholic Church affirms the dignity of all human life at every stage and vigorously opposes the destruction of human embryos for the harvesting of stem cells. At the same time, the Church strongly endorses the use of adult and non-embryonic stem cell research as a potential therapy for individuals suffering from these debilitating diseases. Research has demonstrated that adult stem cells, including all forms of non-embryonic stem cells, such as induced pluripotent stem cells and umbilical cord stem cells, can be harvested and programmed to achieve pluripotency the same characteristic that enables embryonic stem cells to differentiate into any type of cell.

An urgent need exists to increase the number of faculty experts performing adult stem cell research at Notre Dame. Doing so will expand upon the strong foundation the College of Science holds in these areas and will help create an environment for excellence in which faculty and students can learn, grow, collaborate and ultimately affect human health.

"We are overwhelmed with gratitude at the generous gift from Mike and Liz Gallagher," said Gregory P. Crawford, dean of the College of Science. "The impact of this gift is truly beyond measure. It will play a crucial role in attracting three more of the best faculty in the field of adult stem cell research to Notre Dame. Furthermore, this gift will equip our existing talented group of adult stem cell researchers at Notre Dame to take the next great leap toward ultimately forming a premier center in adult stem cell research."

Michael Gallagher is a 1991 graduate of Notre Dame, and his wife, Elizabeth, is a 1992 graduate of Saint Mary's College. They have two sons, Brock and Jack, and currently live near Denver.

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Notre Dame establishes professorships in adult stem cell research

FOR THE PAST decade, two things have consumed large chunks of Malvern native Tom Kramer's time.

The first is his training regimen. Kramer, 46, is a practicing triathlete who will compete Saturday morning in the eighth annual Philadelphia Insurance Triathlon in Fairmount Park.

The second is the search for a bone-marrow match for his wife Pam, also a triathlete, who was diagnosed with a rare form of leukemia in 2000 and eventually willl need a bone marrow transplant.

At some point, Kramer made a creative decision to have those cumbersome obligations intersect. Desperate to spread the word about the importance of registering as a bone-marrow donor he estimates only 9 million people are registered Kramer embarked on a four-event quest over the span of 8 months to raise awareness.

"It was just me in the beginning," he said. "All I had was a banner and some testing kits."

Kramer completed a marathon, two Ironman half-triathlons and a full Ironman triathlon. Eventually his effort gained steam, finally culminating last year when the Kramers incorporated their hard work into the non-profit Racing to Register.

Using endurance sports as a platform, Racing to Register aims to enlarge the pool of potential donors for blood cancer patients in need of lifesaving bone marrow or stem cells.

"We think that the endurance part the reason we chose that platform is that you have to have a lot of endurance to go through that kind of treatment," Kramer said. "There is that marriage there if we can put ourselves through this, you can register."

Athletes that who join Team RTR complete the donor registration process and, in return, the program facilitates their endurance training through coaching, discounted gear and more.

While his wife's illness is what got him started, Kramer says the event has grown into something much bigger. With more than 2,100 registrants, RTR has produced four potential matches.

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Husband competes to raise awareness about bone-marrow registration

NEWARK, Calif., June 21, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced initiation of a Phase I/II clinical trial of the Company's proprietary HuCNS-SC(R) product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD) referred to as Geographic Atrophy. There are no approved treatments for dry AMD.

The trial is being conducted at the Retina Foundation of the Southwest's (RFSW) Anderson Vision Research Center in Dallas, Texas, one of the leading independent vision research centers in the United States. David G. Birch, Ph.D., Chief Scientific and Executive Officer of the RFSW and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, is the principal investigator of the study.

"Dry AMD is the most common form of macular degeneration, and has a very debilitating effect on quality of life," said Dr. Birch. "Transplanting neural stem cells to protect photoreceptors in patients diagnosed with AMD is an innovative, but logical, approach, well supported by the Company's recently published preclinical data. We are very excited to be conducting this trial at RFSW."

A summary of the Company's preclinical data was featured in the February 2012 issue of the international peer-reviewed European Journal of Neuroscience (available online at http://onlinelibrary.wiley.com/doi/10.1111/j.1460-9568.2011.07970.x/abstract). The data demonstrated that HuCNS-SC cells protect host photoreceptors and preserve vision in the Royal College of Surgeons (RCS) rat, a well-established animal model of retinal disease which has been used extensively to evaluate potential cell therapies. Transplantation of HuCNS-SC cells significantly protects photoreceptors from degeneration. Moreover, the number of cone photoreceptors, which are responsible for central vision, remained constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed in the study. In humans, degeneration of the cone photoreceptors accounts for the unique pattern of vision loss in dry AMD.

"Unlike others in the field, our clinical strategy is to preserve visual function before it is lost," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Our published preclinical data provides a strong rationale for this approach in dry AMD and we hope to replicate these results in this clinical trial. We are very pleased to be working with Dr. Birch and the Retina Foundation of the Southwest, who have the expertise and referral base to undertake this important study. We anticipate that we will be able to accrue the requisite number of patients for this trial in relatively short order."

About Age-Related Macular Degeneration

Age-related macular degeneration refers to a loss of photoreceptors (rods and cones) from the macula, the central part of the retina. AMD is a degenerative retinal disease that typically strikes adults in their 50s or early 60s, and progresses painlessly, gradually destroying central vision. According to the RFSW website, there are approximately 1.75 million Americans age 40 years and older with some form of age-related macular degeneration, and the disease continues to be the number one cause of irreversible vision loss among senior citizens in the US with more than seven million at risk of developing AMD.

About the Trial

The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina in the most affected eye. Patients' vision will be evaluated using both conventional and advanced state-of-the-art methods of ophthalmological assessment. Evaluations will be performed at predetermined intervals over a one-year period to assess safety and signs of visual benefit. Patients will then be followed for an additional four years in a separate observational study. Patients interested in participating in the clinical trial should contact the site at (214) 363 3911.

About HuCNS-SC Cells

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StemCells, Inc. Initiates Phase I/II Clinical Trial in Dry Age-Related Macular Degeneration

Public release date: 19-Jun-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES (June 19, 2012) Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

The study, published in the June 19 online issue of PLoS ONE, extends the institute's work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

"With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute.

Svendsen and his team have investigated this disease for some time now. In 2009, Nature published a study by Svendsen and his colleagues detailing how skin cells taken from a patient with the disorder were used to generate neurons of the same genetic makeup and characteristics of those affected in the disorder; this created a "disease-in-a-dish" that could serve as a model for discovering new drugs.

As the disease is unique to humans, previous methods to employ this approach had been unreliable in predicting how it occurs in humans. In the research published in PLoS ONE, to the team reproduced this model with skin cells from multiple patients, taking them back in time to a pluripotent stem cell state (iPS cells), and then driving them forward to study the diseased patient-specific motor neurons.

Children born with this disorder have a genetic mutation that doesn't allow their motor neurons to manufacture a critical protein necessary for them to survive. The study found these cells die through apoptosis the same form of cell death that occurs when the body eliminates old, unnecessary as well as unhealthy cells. As motor neuron cell death progresses, children with the disease experience increasing paralysis and eventually death. There is no effective treatment now for this disease. An estimated one in 35 to one in 60 people are carriers and about in 100,000 newborns have the condition.

"Now we are taking these motor neurons (from multiple children with the disease and in their pluripotent state) and screening compounds that can rescue these cells and create the protein necessary for them to survive," said Dhruv Sareen, director of Cedars-Sinai's Induced Pluripotent Stem Cell Core Facility and a primary author on the study. "This study is an important stepping stone to guide us toward the right kinds of compounds that we hope will be effective in the model and then be reproduced in clinical trials."

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Cedars-Sinai researchers, with stem cells, advance understanding of spinal muscular atrophy