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GeneCell International, Miami’s Only and Preferred Cord Blood Laboratory, Participates in The Stem Cells USA & World …

October 1st, 2012 11:15 am

Directors of GeneCell International, a cutting-edge facility specialized in the processing and cryogenic preservation of umbilical cord blood, cord tissue, dental pulp and adipose stem cells, attends the Stem Cells USA & World Cord Blood Congress 2012.

Miami, FL (PRWEB) October 01, 2012

The event was attended by 300+ notable and prominent doctors, scientists and regulators in the field of stem and featured numerous keynote speakers. The program kicked-off with the chairmans opening remarks on the perspective on the cord blood market. Entities involved in the collection, processing, cryopreservation, transplantation and research shared their and experiences with the rapidly evolving future of cord blood stem cells and related tissues. Some presentation topics included were:

Umbilical cord blood preservation is a process by which blood is collected from the umbilical cord of a newborn baby and is stored cryogenically in a specially-designated bank. According to the National Marrow Donor Program, cord blood contains cells that can be transfused to a patient to treat various diseases, including lymphoma and leukemia. Currently, there are approximately 80 treatable diseases and the list of illnesses continue to grow. Cord blood is rich in stem cells and there is less risk for the recipients immune system to reject the cells, because certain immune cells found in the cord blood are not mature. Cord blood can be used to treat the child from whom the blood was collected as well as some first-degree relatives who are a close genetic match, such as family members. Additionally, patients can get the treatment in about three weeks - as opposed to six to eight for bone marrow from an adult donor.

A persons blood stem cell type is inherited, which means a patient is more likely to find a matched donor from within their own ethnic group, said GeneCells Director of Operations, Jose Cirino. More than half of cord blood donations and privately banked cord blood in the United States are from Caucasians while minorities remain underrepresented. By increasing the awareness of cord blood advantages among minorities, there is a potential for increased access to therapies for more people.

The shortage, or lack of availability, affects patients of African, Asian, Hispanic and Native American Indian descent. Since patients who need a transplant are more likely to find a match within their own race, Cirino adds it is important that the pool of donors reflects the overall community.

Why isn't everyone banking these cells? What transpires is that people are not informed about stem cell banking and some have never even heard of it. Most people are not aware they have stem cells in their body, they believe that stem cells only come from human embryos since that is what is mainly discussed in politics and the news. However, this is not the case. These cells are found in adults and there are no moral, ethical or political issues surrounding these cells.

The amazing thing about these cells, aside from their potential to treat a variety of different diseases, is that for the most part they can be harvested from the individual through relatively minimally invasive procedures and can be cryogenically frozen (at a temperature of -321 F) and stored for decades until a disease manifests or they are needed for cell-based therapies added GeneCells Director of Research & Laboratory Operations, Dr. Todd R. Flower.

GeneCell International specializes in the collection, transport, processing and cryogenic storage of adult stem cells from various sources including; umbilical cord blood, cord tissue, dental pulp and adipose tissue (fat) that can later be used to treat a variety of diseases. The laboratory is also involved in scientific research and development with a range of stem cells from various adult tissues. The facility is governed and inspected by the FDA as well several other regulating bodies to ensure the safety of these cellular therapies.

Alongside its commitment to educating the public on the benefits of cord blood preservation, GeneCell is committed to being on the forefront of stem cell research. GeneCell International is the only Cord Blood, Cord Tissue and Dental Pulp Processing and Cryogenic Storage Laboratory to offer this cutting-edge, regenerative medicine technology in Miami, Florida.

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Medistem and Superview Biotechnology Co. Ltd. Initiate Collaboration on Therapeutics Development Using Antibody and …

October 1st, 2012 11:15 am

SAN DIEGO--(BUSINESS WIRE)--

Medistem Inc (Pink Sheets:MEDS) announced today the initiation of a collaboration with Superview Biotechnology Co. Ltd, a subsidiary of Yinhuan Holding Co from Yixing, China. The joint work will be aimed at using proprietary stem cell lines developed by Medistem for screening of monoclonal antibodies for therapeutic activity in the area of regenerative medicine. As part of the collaboration, the two companies will evaluate various candidates jointly, as well as apply for grants and share research data.

To date, the majority of stem cell companies are focusing on the stem cell itself being a product. By collaborating with Superview Biotechnology, we aim to assess the feasibility of developing antibodies that can modulate the activity of stem cells that already exist in the body, said Thomas Ichim, CEO of Medistem. This approach not only provides methods of activating stem cells but also allows for the development of stem cell adjuvant therapies that could be used to resurrect stem cell candidates that failed in clinical trials.

Superview Biotechnology has developed proprietary methods of rapidly generating monoclonal antibodies to esoteric protein targets. Medistem has a history of success in the area of stem cells, being the only company to take a stem cell product from discovery to FDA clearance in the short span of 4 years.

One of the significant driving forces behind our company is to develop innovative targets for our monoclonal antibodies. Although monoclonal antibodies have generated sales of billions of dollars in areas ranging from rheumatoid arthritis, to cancer, to preventing blindness, we feel that the potential of this therapeutic tool is only beginning to be recognized, said Jiong Wu, CEO of Superview Biotechnology. Our opinion is that the barriers to entry for monoclonal antibody-based therapies modulating endogenous stem cells is lower than stem cell based therapies. We are eager to work with the Medistem team at exploring this hypothesis.

A joint grant is expected to be filed with the National Natural Science Foundation of China to support part of the proposed collaboration by end of October, 2012.

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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Celprogen Obtained US Patent (US8,236,297B2) Method of Treating Lactose Intolerance Utilizing Genetically Engineered …

October 1st, 2012 2:20 am

LOS ANGELES--(BUSINESS WIRE)--

Celprogen Inc., a leader in the Stem Cell Research and Therapeutics industry for the development of stem cell technologies for regenerative medicine, today announced that they obtained a Patent for Treating Lactose Intolerance Utilizing Genetically Engineered Bacteria US8,236,297B2. Acquired lactase deficiency is the most common disorder of complex carbohydrate absorption throughout the world, affecting 75% of world population. In the United States 15% of Caucasians, over 50% of Hispanics and over 80% of African-Americans suffer from lactose intolerance.

The present invention relates to genetically engineered bacteria that are able to colonize the mammalian intestine and actively produce mammalian lactase. This lactose-digesting enzyme is stable and active under the conditions normally found in the mammalian small intestine. Experimental subjects colonized with the genetically engineered bacteria show improved ability to digest lactose in dairy foods.

About Celprogen Inc.

Celprogen Inc. is a global Stem Cell Research & Therapeutics company which is developing a proprietary portfolio of unique therapeutics products and life science research tools that includes genetic engineering technologies, stem cell technologies for regenerative medicine, as well as bio-engineering products for tissue & organ transplants. Headquartered in San Pedro, California, Celprogen is committed to the research, development, and manufacture of quality Stem Cell, Cancer Stem Cell and Primary Cell Culture products to serve our global community. Additional information about Celprogen is available at http://www.celprogen.com.

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Stem Cell Therapy—Breakthrough in Health Paradigm

October 1st, 2012 2:20 am

By Sharmistha Banerjee - September 25, 2012 | Tickers: NBS, OSIR, PSTI | 0 Comments

Sharmistha is a member of The Motley Fool Blog Network -- entries represent the personal opinions of our bloggers and are not formally edited.

Far-reaching accomplishments in the biotechnology sector meet its most ambitious expectations, stem cell therapy. The birth of this new industry has boosted the enthusiasm and energy of investors and has brought unprecedented capability and optimistic predictions. New developments in regenerative medicine are bringing about exciting, novel approaches to create therapies for hard to treat diseases. The biotechnology industry has been soaring in 2012 as companies both large and small have shown impressive growth.

The cell therapy space has seen relatively small companies making strides in the right direction with increased government support. Osiris Therapeutics (NASDAQ: OSIR) a leading stem cell company is currently the only company with an approved cell therapy. The approval is more of a first step in a long walk for Osiris. Reuters reported that shares of Osiris Therapeutics rose 15% on May 30, 2012, after U.S. health regulators said the stem cell technology company's wound treatment was eligible for reimbursement when used in hospitals in out-patient settings or in ambulances. The company carries over a $300 million market capitalization and trades at $9.50 per share, primarily on the strength of a recent Canadian approval for its stem cell drug for graft-versus-host disease. Osiris Therapeutics has a 1-year low of $4.12 and a 1-year high of $14.46. The company has a market cap of $311.3 million and a price-to-earnings ratio of 90.98. Investors are impressed and optimistic with Osiris progress in cell-based therapies. They currently have a $9.75 target price on the stock. Despite having to negotiate a more challenging regulation process the company has continued to show investors strong gains in 2012.With a current ratio of 8.51 and debt equity of (0.00%) the company boasts of a financially secure position in the market.

Pluristem Therapeutics (NASDAQ: PSTI) a small firm with a market cap of less than $180 million has been concentrating on its placenta-based cell therapies, is considered one of the more advanced in the cell therapy arena, and unlike OSIR, its lead candidates treat diseases that could potentially return significant revenue. The upside for PSTI is lower costs, quicker healing time, ease of administration, and most importantly, it can grow vessels and provide the possibility of a cure, which has led to optimism surrounding the stock. Shares of Pluristem Therapeutics are up over 3.98% and most likely headed higher in the days ahead. It has traded higher by 85% during the last three months and is now valued at $200 million. Pluristem may actually beat OSIR in the race to become the first U.S. approved cell therapy with its bone marrow therapy, in which it has recently applied for approval. Pluristem is a company that I think is showing great promise. From the stock's action in the last several months, it is clear investors recognize that Pluristem's unique platform technology has the potential for tremendous value in a lucrative range of medical markets both the very large and the very small. The company wins both ways. Its clinical segment is creating candidates with large revenue potential, with analysts projecting peak sales of $700 million for AMR-001, which treats patients following acute myocardial infarction. The company is reasonably well funded with around $42 million in cash and cash equivalents.

NeoStem (NYSEMKT: NBS) is by far the leader in regards to the manufacturing business, and no other company comes close. In addition, its stock has returned the most over in the last three months, with a 100% gain. NeoStem stocks looks promising as a biotechnology investment. First, the company is focusing on several promising areas of new stem cell treatment development. Second, its contract manufacturing business brings in revenues to offset some of its drug development expenditures. Third, the contract manufacturing business could earn substantial royalties if any of the products on which it works with customers proves to be a commercial success. NeoStem's manufacturing segment which is also known as PCT, is well positioned to return larger gains over the next 24 months with several late stage candidates under development. a $110 million company that has increased in value by 70% during the last three months, In addition to the PCT business, NeoStem's most promising therapy is aimed at preventing major cardiac problems following acute myocardial infarction (AMI), an area that is potentially a multibillion-dollar business. NeoStem's therapy is meeting endpoints never before reached,

The three companies discussed above are showing much potential for growth and each present a significant upward shift in the current stock prices while contributing greatly to the advances of cell therapy.

Osiris is the closest to generating substantial revenue by already having two approvals, and is currently testing its therapy on other diseases, thereby leaving open the possibility of future gains. Pluristem has candidates to treat diseases in potentially large markets, and is expanding with its manufacturing facility. Although Stem Cells is in the early phases of development, it still has a very innovating therapy that, if proven effective, could advance the space even further. NeoStem possesses all the benefits of an innovating technology, a diversified pipeline, and is a candidate with significant revenue potential.

At this point, it appears that the entire space is moving forward and has lifted observers' expectations by making rapid progress. It makes sense that these three stocks would trade with such considerable gains, as investors can now identify the benefits of cell therapies. And as more approvals occur, it could be a space that trades considerably higher regardless of the market's indecisiveness. With the sector growing and maturing, investing in biotech stocks seems a promising choice in future.

SharmisthaB has no positions in the stocks mentioned above. The Motley Fool has no positions in the stocks mentioned above. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.If you have questions about this post or the Fools blog network, click here for information.

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Reverse aging? Scientists find way to make old muscles young again

September 30th, 2012 6:15 pm

It is a dream for everyone as they grow older to turn back the clock and live in a younger body once again. While many have developed ways to make the body look younger cosmetically, there have been very few effective methods to combat the aging process within the body until now.

For the first time ever, researchers have identified a crucial protein responsible for the decline of muscle repair and agility as the body ages. Upon this discovery, the scientists were able to effectively halt muscle decline in mice, giving hope to similar treatments for humans in the future.

According to the studys authors, loss of muscle strength and repair is one of the major concerns facing elderly citizens.

A great advantage of medicine is that people are not dying as early as they used to, but the body hasnt figured out how to maintain its muscle repair, Andrew Brack, of the Massachusetts General Hospital Center for Regenerative Medicine and corresponding study author, told FoxNews.com. The average loss of muscle mass for the 80-year-old male is 40 percent. Elderly people will fall over and break bones, they go to the hospital where they lose more muscle strength, and then dont recover.

Brack noted that muscle strength is also one of the main factors that keeps elderly individuals out of the hospital and allows them to be productive members of the workforce. In order to combat this muscle decline, Brack and Albert Basson, who met at Kings College London, teamed up to see if they could put the process in reverse.

The key revolves around stem cells found within muscles. During exercise or injury, these stem cells become activated and work fervently by dividing and multiplying into new muscle fibers that help to repair the muscle. When they are no longer need, they retreat into a reservoir within the muscle and lay dormant until they are needed again.

The problem with aging muscles is that these fixer stem cells dont remain dormant when theyre not needed. Instead, they become activated more and more and unnecessarily divide and multiply causing them to die at a faster rate. Since muscles only have a finite amount of these stem cells, the quicker the cells die, the less effective muscles become at repairing themselves.

Wondering exactly why the stem cells became more activated with age, Brack and Basson screened older muscles, finding higher levels of a protein called FGF2 a protein that stimulates cell division. The scientists figured these levels could explain the unnecessary cell activation.

As your muscle gets old, you start making more of this FGF2 protein, Basson, senior lecturer at Kings College London Dental Institute, told FoxNews.com. When theres more, the FGF2 starts waking up these stem cells and they start dividing. The stem cells have a limited number of times they can divide before they die or differentiate into other cells.

Basson figured that if they were able to boost a gene called SPRY2, which inhibits FGF2, then the stem cells would lay dormant until they were absolutely needed. To test this theory, the researchers administered a common drug containing SPRY2 to suppress FGF2 levels in elderly mice. Sure enough, the drugs halted the decline of muscle stem cells in the mice.

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San Francisco Dentist Follows Stem Cell Research Regarding Craniofacial Tissue Renewal

September 30th, 2012 6:15 pm

SAN FRANCISCO, Sept. 18, 2012 /PRNewswire/ -- Case results published in the journal Cell Transplantation revealed that human craniofacial tissues regenerate faster using stem cells than with traditional bone regeneration therapy.

San Francisco dentist Dr. Greg Larson awaits the benefits this research has in store for restorative dentistry treatments.

The University of Michigan School of Dentistry, the Michigan Center for Oral Health Research and Aastrom Biosciences, Inc. in Ann Arbor, Mich., performed research in a dual effort. Clinical trials included 24 participants needing jawbone reconstruction following tooth extraction. Researchers prescribed some participants bone regeneration therapy, while others received ixmyelocel-T cells, developed by Aastrom Biosciences.

Aastrom researchers used the bone marrow of participants to create a variety of cell types, including stem cells that would later be implanted into their mouths and jaws.

"Patients who received tissue repair cells achieved greater bone density and quicker bone renewal and required less bone grafting during implant procedures when compared to those who underwent traditional guided bone regeneration therapy," according to the U-M press release.

U-M School of Dentistry Assistant Professor Darnell Kaigler says the regenerated bone will provide dentists with a stronger foundation when using dental implants. By replacing missing teeth with dental implants, dentists like Larson can improve a patient's ability to eat, speak and smile normally.

Kaigler, also the principal investigator on the project, added that another benefit of this treatment method is that patients can renew tissues using their own cells, as opposed to exposing their bodies to foreign cells.

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Scientists Discover How To Halt Aging Muscles

September 30th, 2012 6:15 pm

Editor's Choice Main Category: Stem Cell Research Also Included In: Seniors / Aging Article Date: 27 Sep 2012 - 12:00 PDT

Current ratings for: Scientists Discover How To Halt Aging Muscles

4.27 (11 votes)

This groundbreaking study, published in Nature, explains why muscle mass decreases with age, an important factor in weakness, causing lack of mobility and falls in elders.

Previous research has told us that stem cells can play a crucial role in stimulating muscle regeneration. This particular study looked at stem cells found in muscles that are responsible for repairing injuries and why the muscles' capacity of regeneration deteriorates with age.

An inactive supply of stem cells is present inside every muscle, ready to be put into action by exercise or injury to fix any damage. When these stem cells are needed, they can divide into hundreds of new muscle threads and repair the injured muscle. At the end of the repairing process, a few of these cells refill the supply of dormant stem cells, enabling the muscle to carry on repairing itself continuously.

Researchers used elderly mice to conduct this study and found the number of inactive stem cells in the extra pool decreases with age, explaining the decline in the muscle's ability to regenerate and repair as the body gets older. When these muscles were examined, the scientists found high levels of FGF2, a protein that has the capacity to trigger the division of cells. While stimulating inactive cells to split and repair muscle is a normal and vital process, they found FGF2 could awaken the dormant stem cells even when they were not needed. Continuous activation of the dormant stem cells meant depletion of the surplus, leaving the muscles without necessary stem cells when repair was really needed.

After this finding, the team attempted to obstruct FGF2 in old muscles in order to prevent the stem cell surplus from being activated unnecessarily. By giving the mice a common FGF2 inhibitor drug, they were able to slow down the decline in the number of muscle stem cells.

Dr Albert Basson, Senior Lecturer from the Department of Craniofacial Development and Stem Cell Biology at the King's College London Dental Institute, said:

Written by Kelly Fitzgerald Copyright: Medical News Today Not to be reproduced without permission of Medical News Today

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GeneCell International an International Leader in Cord Blood and Dental Pulp Stem Cells Expands to India

September 30th, 2012 6:15 pm

Miami, FL (PRWEB) September 19, 2012

GeneCell International, an international leader in the processing and preservation of umbilical cord blood stem cells, announced today the expansion of its services into the country of India.

The company, with its corporate headquarters located in Miami, Florida, privately collects, processes and stores stem cells from umbilical cord blood, cord tissue, dental pulp and adipose tissue that can later be used to treat a variety of diseases. GeneCell International has deep roots in Latin America and more than a decade of experience in helping parents make informed decisions that can lead to potentially life-saving possibilities. The company also plans to collaborate with the medical community to further educate the expecting parents on the benefits stem cells offer.

The expansion into India provides easy and accessible resources for parents looking to preserve viable adult stem cells from both umbilical cord blood and dental pulp, said GeneCells Director of Operations, Jose Cirino. GeneCell believes in offering the best support and advice to the medical community to ensure parents are given the best and most up to date information in making an informed decision on preserving their childs stem cells that can potentially save a life of a family member.

Cord blood is rich in stem cells and there is less risk for the recipients immune system to reject these cells, because certain immune cells found in the cord blood are not mature. These cells can later be used to treat a variety of diseases and blood disorders within the immediate family, are free of ethical debate and patients can get the treatment in about three weeks - as opposed to six to eight for bone marrow from an adult donor, added Dr. Todd R. Flower, Genecells Director of Research and Laboratory Operations.

Alongside its commitment to educating the public on the benefits of stem cell preservation, GeneCell is always on the forefront in providing information for those who may require stem cells for medical treatments. With more than a decade of experience, GeneCell has maintained a large presence in Latin America - promoting the practice and encouraging families to bank their childs stem cells to help protect their loved ones.

About Umbilical Cord Blood Preservation: Umbilical cord blood preservation is a process by which blood is collected from the umbilical cord of a newborn baby and is stored cryogenically in a specially-designated bank. According to the National Marrow Donor Program, cord blood contains cells that can be transfused to a patient to treat various diseases, including lymphoma and leukemia. The list of illnesses that can be treated with cord blood continues to grow. In addition, the cord blood can be used to treat the child from whom the blood was collected as well as some first-degree relatives who are a close genetic match, such as immediate family members. Cord blood banking is regulated by the U.S. Food & Drug Administration and each year more and more parents choose to save their childrens cord blood should the medical need arise.

About Dental Pulp Stem Cells: One of the major advantages one gets from harvesting stem cells from his own body is that there will be no rejection of these cells when they are harvested and subsequently re-implanted. In the future, medical researchers anticipate being able to use technologies derived from stem cell research to treat a wider variety of diseases including Parkinsons, Alzheimers, spinal cord injuries, diabetes, heart diseases, liver disease, multiple sclerosis, muscle damage and many other diseases. The discovery that human dental pulp tissue contains a population of multi-potent mesenchymal dental pulp stem cells with the ability to reproduce quickly for self-renewal and the ability to differentiate into functional odontoblast has revolutionized dental research and opened new avenues in particular for reparative and reconstructive dentistry and tissue engineering in general.

About GeneCell International: GeneCell International, LLC is a trusted provider of collection, processing and storage of umbilical cord blood, dental pulp (teeth), and adipose (fat) from which stem cells can be extracted to treat a variety of diseases and disorders. GeneCell operates state-of-the-art laboratories and storage facilities for the cord blood of thousands of clients, headquartered in Miami, Florida and with local offices in Central Florida, Colombia, Costa Rica, Dominican Republic, Honduras, India, Peru, Puerto Rico, and Venezuela.

For more information and to learn more about cord blood, dental pulp, adipose tissue stem cell banking benefits or other services visit http://www.genecell.com.

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Aggressive cancer exploits Myc oncogene to amplify global gene activity

September 30th, 2012 6:15 pm

ScienceDaily (Sep. 27, 2012) For a cancer patient, over-expression of the MYC oncogene is a bad omen. Scientists have long known that in tumor cells, elevated levels of MYC's protein product, c-Myc, are associated with poor clinical outcomes, including increased rates of metastasis, recurrence, and mortality. Yet decades of research producing thousands of scientific papers on the subject have failed to consistently explain precisely how c-Myc exerts its effects across a broad range of cancer types. Until now, that is.

The prevailing theory emerging from this massive body of research has been that in tumor cells, c-Myc affects the expression of specific genes or sets of genes -- that so-called Myc target genes are being selectively activated or repressed, leading to aberrant cellular behavior. Now, however, researchers in the lab of Whitehead Institute Member Richard Young are dispelling this commonly held notion, showing that elevated expression of c-Myc amplifies the activity of all expressed genes in tumor cells of multiple cancer types. It turns out that high levels of c-Myc send a tumor cell's gene expression program into overdrive. Transcription increases dramatically, allowing malignant cells to overwhelm factors that might normally hamper their growth and proliferation. This surprising finding, published in this week's issue of the journal Cell, provides a simple, elegant explanation for how a single protein can have such profound effect in so many and varied types of cancer. The newly revealed mechanism may also help scientists develop novel therapeutic approaches that disrupt c-Myc's activity.

"MYC is a key driver in most major cancers, but it has been notoriously difficult to drug," says Young, who is also a professor of biology at MIT. "Now that we know the mechanism by which c-Myc acts, we can go after the components of that mechanism as potential drug targets. This research creates an even stronger impetus to find a way to drug the thing."

One potential drawback to thwarting c-Myc's activity is the important role it plays in normal cell division. That role is so powerful that cells co-evolved an emergency death pathway to keep c-Myc expression in check. If c-Myc's production spins out of control in an otherwise normal cell, the cell immediately commits suicide through a process called apoptosis. But in cancer cells in which c-Myc is overproduced, this suicide pathway is compromised, allowing the cell to survive and proliferate.

"MYC is the most deregulated gene in cancer," says Charles Lin, a graduate student in the Young lab and co-author of the Cell paper. "It's been called a bad-boy, a Swiss army knife, and a jack-of-all-trades because, according to previous research, it could do everything under the sun in a cancer cell. But most of the different attributes ascribed to MYC are contradictory or seemingly incompatible."

Propelled by its earlier research that identified c-Myc as an important regulator of transcription in embryonic stem cells, the Young lab began to focus on c-Myc's activity within cancer cells. Lab members found that as the expression of c-Myc increases in these cells, the protein attaches to the promoters and enhancers of all active genes, thereby amplifying the active genes' transcription. The heightened transcription produces cells bloated with excessive RNAs and proteins capable of altering normal cellular functions. Researchers observed this phenomenon in cells from a host of cancers, including Burkitt's lymphoma, small cell lung cancer, multiple myeloma, and glioblastoma multiforme.

"The previous research now makes sense -- finally!" says Jakob Lovn, co-author and postdoctoral researcher in the Young lab. "Our findings provide a way to unify everybody's seemingly conflicting data. I think that's really nice. Instead of saying 'you're all wrong,' we're saying 'you're all right, and here's why.' The model makes a lot of sense in terms of the biology that has been described so far."

With a better understanding of how c-Myc can wreak so much damage, the Young lab is turning its efforts to disrupting c-Myc's activity. Although cancer cells that overproduce c-Myc are associated with poor clinical outcomes, their reliance on c-Myc for survival may represent an Achilles' heel. When these "Myc-addicted" cells are deprived of c-Myc in vitro, even for a short period of time, they quickly die. Research in mice has shown that, Myc-addicted tumors deprived of the protein shrink dramatically. Despite c-Myc's necessary role in normal cell division, particularly in tissues with rapid cell turnover, such as the intestine and blood, these mouse studies have shown that if c-Myc activity is restored after a brief period, normal tissues quickly bounce back, while tumors are unable to regain their footing.

"So what we think now is that potentially, if drugs can tune down the levels of transcription just slightly, this might be catastrophic for the Myc-addicted cancer cells," says Peter Rahl, co-author and postdoctoral researcher in the Young lab. "You wouldn't need to abolish all transcription because that would be toxic to your other cells. So we're hoping that our model will show us ways to create a therapeutic window where the Myc-addicted cells just won't be able to adapt to lower levels of transcripts."

This work was supported by National Institutes of Health (grants HG002668 and CA146445), Swedish Research Council, American Cancer Society, and Damon-Runyon Cancer Research Foundation.

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MultiCell Technologies To Announce Positive Preclinical Results at 2012 ASCB Meeting

September 30th, 2012 6:15 pm

WOONSOCKET, R.I., Sept. 27, 2012 /PRNewswire/ --MultiCell Technologies, Inc. (OTC Bulletin Board: MCET) is pleased to announce the acceptance by the American Society for Cell Biology (ASCB) of abstract "Short Synthetic Double Stranded RNA with Dual Activity - Oncolytic and Immune Modulatory - for Hepatocellular Carcinoma." The preclinical research results will be presented by Anand Ghanekar M.D., Ph.D., Division of Cellular & Molecular Biology, Toronto General Hospital Research Institute, at the 2012 ASCB Annual Meeting in San Francisco, CA, December 15-19, 2012.

Poster Session: Cancer Therapy II Day/Date of Presentation: Tuesday, December 18, 2012 Time of Presentation: 12:30 PM - 2:00 PM PST Place: Exhibit Halls A-C Presentation Number: 2444 Board Number: B1425

Dr. Ghanekar's research was supported by MultiCell via a sponsored research grant with the University Health Network, Toronto General Hospital Research Institute, Ontario, Canada. The research results to be presented by Dr. Ghanekar support further mechanistic and in vivo studies exploring the safety, effectiveness and utility of MCT-465 and MCT-485 as novel therapeutic agents as a treatment for hepatocellular carcinoma and other cancers.

About MCT-465 and MCT-485 MCT-465 and MCT-485 are the first of a family of prospective cancer therapeutics based on the use of our patented TLR3 signaling technology. MCT-465 and MCT 485 are in preclinical development, and are being investigated as prospective treatments for primary liver cancer and triple negative breast cancer.

The immune system is composed of two synergistic elements: the innate immune system and the adaptive immune system. Stimulation of the innate immune system through key receptors plays a critical role in triggering the adaptive immune response stimulating T and B cells to produce antibodies. In cancer, this integrated defense system does not work well, resulting in suboptimal activation of innate immunity and thus, late or inefficient adaptive immunity. The innate immune system is composed of a family of ten receptor molecules, the Toll-like Receptors (TLR1-TLR10), which act as sentries to identify invaders and signal the alarm to mobilize the body's array of immune defenses.

Within the tumor lesion, there may be infiltrating monocytes, dendritic cells and leukocytes in general, that have the capability to mobilize an adaptive or innate immune response but they are either silent or immune suppressive in the absence of select immune interventions. Such infiltrating non-cancerous immune cells may express TLR3, other TLRs, RIG-I and/or MDA-5. In addition, within tumor lesions, there may be cancerous cells or stromal cells or cancer stem cells which express TLR3, other TLRs, RIG-I and MDA-5 (representing RNA-sensing molecules).

Cancer stem cells are thought to play a role in a tumor's resistance to therapy. While significant progress has been made in developing cancer therapies that result in cytoreduction and thus tumor regression, the control of cancer over a longer interval and especially of metastatic disease, remains a key goal. Cancer stem cells are believed to be responsible for cancer relapse by being less sensitive to conventional therapies.

MultiCell owns exclusive rights to two issued U.S. patents (6,872,389 and 6,129,911), one U.S. patent application (U.S. 2006/0019387A1), and several corresponding issued and pending foreign patents and patent applications related to the isolation and differentiation of liver stem cells. The role of liver stem cells in the carcinogenic process has recently led to a new hypothesis that hepatocellular carcinoma arises by maturation arrest of liver stem cells.

Double stranded RNA (dsRNA) provides a therapeutic avenue for cancer treatment through (a) activating intra-tumoral leukocytes, abrogating their immune suppressive activity and/or (b) interacting with cancerous cells and directly inducing apoptosis, or indirectly through mobilization of immune effector mechanisms.

MCT-465 is a high molecular weight synthetic dsRNA (polyA:polyU, of 70bps) with immune enhancing properties. The mechanism of action of MCT-465 is pleiotropic and mediated by RNA sensors such as TLR3, 7/8, MDA-5 and RIG-I - expressed by antigen presenting cells and select cases, by tumor cells:

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MultiCell Technologies To Announce Positive Preclinical Results at 2012 ASCB Meeting

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Study Shows Stem Cells May Prevent And Cure Alzheimer's

September 30th, 2012 6:15 pm

SEOUL, South Korea, Sept. 26, 2012 /PRNewswire/ --In the first study of its kind, researchers at Korea's leading university and the RNL Bio Stem Cell Technology Institute announced this week the results of a study that suggests an astounding possibility: adult stem cells may not only have a positive effect on those suffering from Alzheimer's disease, theycanprevent the disease.Using fat-derived adultstem cells from humans [scientific term:adMSCs, orhuman, adipose-derived mesenchymal stem cells], researchers were able to cause Alzheimer's disease brains in animal models to regenerate. The researchers, for the first time in history, used stem cells toidentify the mechanism that is key to treatment of Alzheimer's disease, and demonstrated how to achieve efficacy as well as prevention of the symptoms of Alzheimer's with adult stem cells, a "holy grail" of biomedical scientists for decades.

Alzheimer's disease, the most common form of dementia (loss of brain function), is the 6th leading cause of death, and affects 1 in 8 people -- more than breast cancer. As of 2010, there were 35.6 million people with Alzheimer's disease in the world, but this number is expected to double every 20 years. It is estimated that the total cost of Alzheimer's is US $604 billion worldwide, with 70% of this cost in the US and Europe. To put that in perspective, Alzheimer's care costs more than the revenues of Wal-Mart (US$414 billion) and Exxon Mobil (US$311 billion), according to the British World Alzheimer's Report of ADI. The cost of Alzheimer's is at the top of health economists' list of the disorders of aging that could topple nations' entire economies, and that regularly ruin not only the lives of patients but of their relatives.

According to the results of this first major study, Alzheimer's may soon be a preventable disease, or even a thing of the past. Equally important, the safety human administration of the kind of adult stem cells used in this experiment has been established in multiple articles and government-approved clinical trials.

THE RESEARCH:

The study was jointly led by Seoul National University Professor Yoo-Hun Suh and RNL Bio Stem Cell Technology Institute (SCTI) director Dr. Jeong-Chan Ra.

The researchers and their teams injected stem cells into mice genetically designed to have the core symptoms and physiology of Alzheimer's disease. They were able to identify that these human stem cells, derived from adipose tissue, behave in a very special way when injected into the tail vein of mice subjects. The cells migrated through the blood brain barrier, thought by many to be impossible for adult stem cells to cross, and went into the brain. In fact,fluorescent labeled cells were monitored for distribution in subjects and the team identified that the infused cells migrated throughout the bodiesincluding brainexcept the olfactory organ, and therefore confirmed that IV infused stem cell can reach to the brain across the blood brain barrier.

The team infused human adipose stem cells intravenously in Alzheimer model mice multiple times two weeks apart from three month to 10 month.Once there, the mice who received cells improved in every relevant way: ability to learn, ability to remember, and neuropathological signs. More important, for the first time ever, Alzheimer model mice showed the mediation of IL-10, which is known for anti-inflammation and neurological protection.

The team also found that stem cell restored special learning ability from Alzheimer model subjects with great reduction of neuropathy lesions.This was found using tests used for Alzheimer's disease: behavioral assessment. In assessment it was found, amazingly, that stem cells' therapeutic effect on Alzheimer's disease was tremendous. This was also found in pathological analysis. The key though was prevention: the scientists showed that stem cells, when infused into Alzheimer's mice, decreased beta amyloid and APP-CT, known to cause brain cell destruction, leading to dementia and Alzheimer's disease. In the lab it was clear that stem cells increased neprilysin, which hydrolyzes toxic proteins. No other compound or treatment has ever suggested so strongly the potential to prevent, as well as stop, this epidemic of incurable dementia sweeping across suffering patients and their families.

Stopping Alzheimer's disease, let alone preventing it, is the focus of thousands of researchers worldwide. Speaking of their breakthrough discovery,Professor Yoo-Hun Suh, who led the study, said, "It is a ground breaking discovery that such a simple method as IV injection of the safest autologous adipose stem cells, without causing any immune rejection, or any ethical issues, opened a new door to conquering Alzheimer's disease, one of the most horrible, expensive and incurablediseases of our time." Joining him, leader of the RNL Bio Stem Cell Technology InstituteDr. Jeong-Chan Ra said, "It has never been more clear that it is an ethical imperative for governments to provide patients with incurable diseases with their right to participate not only in studies like this but in therapies with such obvious potential, once they have been tested as many times for safety as has our technology." Both scientists stressed that the real breakthrough in their complex research is the prevention of the onset of symptoms.

Specifically, stem cells grafted in the brain, in another part of the study, were identified to induce cell division and neuro differentiation of endogenous neuro progenitor cells around the hippocampus and its surrounding cells and increase in great deal the stability of dendrites and synapses. Stem cell also contributed various anti-inflammatory and neuro growth factors, especially increased the expression of IL-10. This again suppressed apoptosis of brain neurons, the prevention effect against Alzheimer's disease.

See the original post here:
Study Shows Stem Cells May Prevent And Cure Alzheimer's

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Anticipated short-term cell therapy industry clinical milestones

September 30th, 2012 3:56 pm
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What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.  


There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.


CellCoTec (http://www.cellcotec.com)
  • Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application.  The CE mark application is under review and they anticipate a response in October.  
  • This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.



ERYtech Parma (http://www.erytech.com)

  • Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2012. 


GamidaCell (http://www.gamidacell.com)

  • Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012.  They have not been shy about the fact they expect to be in the market in 2013.


Innovacell (http://www.innovacell.com)

  • They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed.  Status unknown.


Miltenyi Biotec (www.miltenyibiotec.com)

  • Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.


NovaRx (http://www.novarx.com)

  • Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.


NuVasive (http://www.nuvasive.com)

  • They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.


Sanofi's Genzyme (http://www.genzyme.com)

  • Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.


Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  



--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

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The cost of clinical trial data bias/loss, FDA’s new job and the need for bold leadership.

September 30th, 2012 3:56 pm
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The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here's the underlying premise as succinctly described by author Ben Goldacre:

"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."

Authors M. Todwin and J. Abramson summarize it thusly:

"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."

Here is a great list of very recent resources that may convince you of the merits of this concern:

Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced  requirement to submit all relevant clinical trial data to http://www.clinicaltrials.gov

For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless - and if not powerless than certainly without sufficient resources - to successfully enforce its new power.  This requires collective industry leadership.  Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.

Here's what I wish.  

I wish that as a cell therapy industry we - through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc - would take a leadership position on an issue like this.

I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors.  Of course it requires the political will and cajones.

The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data.  Perhaps even more important would be the public profile such leadership would provide the sector.  Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history. 

Just my thought for the day...

--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

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Fortune Magazine on California Stem Cell Agency: Warm, Personal and Favorable

September 30th, 2012 3:55 pm


California's $3 billion stem cell
research effort today garnered a handsome dollop of favorable
national news coverage– a lengthy piece in Fortune magazine that
spoke of looming stem cell cures and the leading role of the state
stem cell agency.

The article led the Fortune web page online at one point this morning and
likely will be read by tens of thousands of persons, although it was not the cover story on the print product. 
Written by a former senior editor of
the magazine, Jeffrey O'Brien of Mill Valley, Ca., the piece was warm
and personal. He began with the story of his 95-year-old
grandmother and her health issues, ranging from arthritis to macular
degeneration. And he wrote,

“The citizens of California have
spoken. If my grandmother and I had the power to get the rest of the
country to follow, we would.”

O'Brien also discussed the science and
finances of the stem cell business. He said,

“To be clear, the earliest stem cell
therapies are almost certainly years from distribution. But so much
progress has been made at venerable research institutions that it now
seems possible to honestly discuss the possibility of a new medical
paradigm emerging within a generation. Working primarily with rodents
in preclinical trials, MDs and Ph.D.s are making the paralyzed walk
and the impotent virile. A stem cell therapy for two types of macular
degeneration recently restored the vision of two women. Once they
were blind. Now they see!

“Some experts assert that AMD could
be eradicated within a decade. Other scientists are heralding a
drug-free fix for HIV/AIDS. Various forms of cancer, Parkinson's,
diabetes, heart disease, stroke, and ALS have already been eradicated
in mice. If such work translates to humans, it will represent the
type of platform advancement that comes along in medicine only once
in a lifetime or two. The effect on the economy would be substantial.
Champions of stem cell research say it would be on the order of the
Internet or even the transistor.”

O'Brien continued,

“The obstacles along the road from
lab rat to human patients are many, of course, but the biggest by far
is money. With the dramatic events in the lab, you might think that a
gold rush would be under way. That's far from true. Long time
horizons, regulatory hurdles, huge R&D costs, public sentiment,
and political headwinds have all scared financiers. Wall Street isn't
interested in financing this particular dream. Most stem cell
companies that have dared go public are trading down 90% or more from
their IPOs. Sand Hill Road is AWOL. The National Venture Capital
Association doesn't even have a category to track stem cell
investments.”

As for the California stem cell agency
itself, the article contained remarks from its Chairman J.T.Thomas,
President Alan Trounson and former chairman Robert Klein about the origins and progress of the California Institute for Regenerative Medicine (CIRM).
O'Brien wrote, 

“The $1.7 billion awarded so far has made one obvious mark on the state: a dozen gleaming research institutions. CIRM has proved adept at getting billionaires to donate funds to the cause.”

O'Brien interviewed a several
prominent businessmen who have contributed tens of millions of
dollars to stem cell research “about the prospects of a legitimate industry emerging.” One was “bond genius” Bill Gross, who has
contributed to UC Irvine. Gross replied.

“Goodness, you're talking to the
wrong guy. Our donation had nothing to do with business.”

Eli Broad, another big stem cell donor,
said pretty much the same thing. And Andy Grove, the former chairman
of Intel, was “surprisingly full of doom and gloom.” O'Brien
wrote,

“For close to two hours, Grove argues
passionately about how the FDA is enabling predatory offshore
industries by impeding progress and the many reasons financiers want
no part of stem cells. "VCs aren't interested because it's a
shitty business," he says. Big Pharma? Forget it. CIRM? "There
are gleaming fucking buildings everywhere. That wasn't necessary."
When I press him to be constructive, he wearily offers one possible
solution. Rather than courting billionaires to put their names on
buildings, we need a system of targeted philanthropy in which the 99%
can sponsor the individual stem cell lines that matter to them.”

O'Brien said, however,

“It was clear during our talk that
Grove wants an economic model for stem cell research and development
to emerge, even if he's not willing to bet money on its happening.
And that puts him in good company.”

While the Fortune article has its
negative points about stem cell research, it is about as laudatory as
it is going to get at this point for the California stem cell agency.
The piece recognizes and even celebrates much of the work of the
agency. The article clearly details the void in financing
for commercialization of stem cell research, bolstering support for
efforts like those in California. Importantly, it also helps to push
the activities of the stem cell agency more fully into the national
discussion of stem cell research and its future. That should pay off
again and again in future news coverage and also benefit the stem
cell agency as it explores the possibility of additional funding –
either private or public – after the cash for new awards runs out
in 2017.

(The story is in the Oct. 8, 2012, edition of Fortune.)

Source:
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$700,000 Blue-ribbon Study of CIRM All But Finished

September 30th, 2012 3:55 pm


The $700,000 study of the $3 billion
California stem cell agency is nearly concluded and is expected to be
released sometime in November.

A draft of the report has been sent out
for “peer review” and no additional public meetings are
scheduled, according to a spokeswoman for the Institute of
Medicine(IOM)
, which is conducting the study. The IOM did not respond
to questions from the California Stem Cell Report about the number of peer reviewers or how they were selected.
The study began last year under a contract with the stem cell agency, which commissioned the effort, in
part, because agency directors hoped the findings by the blue-ribbon
panel would bolster efforts to win voter approval of another multi-billion dollar state bond issue. More recently the agency has
explored the possibility of private financing to continue operations.
The agency is expected to run out of
funds for new awards in 2017. It currently has something in the
neighborhood of $700 million for awards that is not already committed
in one fashion or another.
Christine Stencel, senior media
relations officer for the IOM, said in an email,

There will be no
further information-gathering meetings. The committee members have
finished drafting their report and it is now undergoing peer review.
Reviewers are anonymous to study staff and committee members; they
will be listed in the front matter of the report when it’s finished
and released.”

She said the stem
cell agency will not be given an opportunity to comment further.
Stencel said,

Sponsors are not
treated as peer reviewers; that is, they’re not afforded an
opportunity to comment on IOM draft reports prior to public release.
IOM is aiming for a public release in November (the exact time frame
will hinge on the duration of the peer review, which is influenced by
people’s schedules and adherence to deadlines). IOM is looking at
options for how best to hold this release, whether there will be an
event of some sort. Once plans are set, they’ll be noted on the
project web pages and IOM will alert the various stakeholders and
interested parties of the plans. The study is moving along and we’re
looking forward to the report’s debut in the not too distant
future.”

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October CIRM Board Meeting Moved to Burlingame

September 30th, 2012 3:55 pm


The location of the October meeting of
the governing board of the California stem cell agency has been
changed from Irvine to Burlingame, near San Francisco International
Airport, in an effort to save travel costs.  

CIRM Chairman J.T. Thomas said the
one-day meeting is being moved because the session will require the
attendance of a large number of CIRM staffers who are based in the
agency's San Francisco headquarters. They will be involved in
presentations involving the agency's new strategic partnership fund and other matters.
The date of the meeting remains
unchanged – Oct. 25. Look for posting of the agenda on the CIRM web
site on Oct. 15. The site of the meeting is the Hilton Bayfront
Hotel
, 600 Airport Blvd.

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CIRM Sponsoring Online Session with FDA on Thursday

September 30th, 2012 3:55 pm


One of the lesser known activities of
the California stem cell agency is webinars that put researchers
together with the folks who make the federal decisions about whether
stem cell research will be turned into therapies.

One of those sessions is coming up on Thursday, and it is not too late for scientists and other interested
parties to get on board.
Writing on the stem cell agency's blog,
Cynthia Schaffer, CIRM's contract administrator and compliance officer
had this to say today about the webinars.

“The FDA very graciously donates
their time to speak on these webinars because they too have pledged
to maintain an active dialogue with the industry and provide
education on their regulatory expectations for product development in
the regenerative medicine field. CIRM science officer Kevin
Whittlesey
 recently
wrote a paper
with Celia Witten of the FDA about the role of the
FDA in reaching out to regenerative medicine community, including
webinars such as these. 

“In that paper they point out that
the communication goes both ways:

“'Appropriate regulation requires a
strong understanding of the latest scientific developments to meet
current and future regulatory needs and challenges.'

“So the FDA benefits by learning from
the other speakers in the webinar – what is the current state of
the technology, what are investigator’s current thoughts on best
practices and the latest research findings, etc. They also learn what
the industry is facing by listening to the questions asked and the
discussion of the challenges during the Q&A sessions. A group of
FDA employees attend each of these CIRM sponsored webinars, and the
wide variety of other workshops and meetings that CIRM hosts
throughout the year.”  

(Editor's note: An earlier version of this item incorrectly identified Cynthia Schaffer as Cynthia Adams.)

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$1.5 Billion in Stem Cell Awards Goes to Directors’ Institutions

September 30th, 2012 3:55 pm


The Sacramento Bee today published an article that reported that $1.5 billion, more than 90 percent of the amount dispensed by the California stem cell agency, has gone to institutions linked to past and present directors of the agency.

The piece was carried on the front page of the newspaper's Sunday Forum section and was written by David Jensen, publisher-editor of the California Stem Cell Report.

The text of the Forum article is below. The Bee also carried a chart listing the top 10 recipient institution. The full text of the comments from Alan Trounson, president of the California stem cell agency,  and two other persons quoted in the article can be found here.

Stem cell cash mostly aids directors' interests

Special to The Bee

By David Jensen

With its latest round of awards earlier this month, California's stem cell agency has now handed out $1.5 billion to enterprises linked to its directors.

The figure amounts to 92 percent of the $1.7 billion awarded by the agency. The grants and loans range from $261 million to Stanford University, whose medical school dean, Philip Pizzo, sits on the agency's governing board, to $170,500 to Children's Hospital in Oakland, whose president, Bert Lubin, also is a member of the board.

The University of California, Davis, has received $128 million. Claire Pomeroy, chief executive officer of UC Davis Health System, is another one of the 29 board members. In all, 27 institutions with past or present representatives on the agency board have received funding.

None of this is illegal. And none of it is likely to change. The situation was created by Proposition 71, the 2004 ballot measure that established the state's $3 billion stem cell agency, formally known as the California Institute for Regenerative Medicine, or CIRM. The initiative was crafted so that virtually all of the institutions that stood to benefit from the state's largesse had seats at the table where the money is handed out.

The built-in conflicts of interest at CIRM have perturbed some experts in California government, but concerns have also reached into the scientific community. The prestigious journal Nature, in 2008, editorialized against what it called cronyism at CIRM. It said the agency "must fight the tendency of the academic institutions on the board to hoard dollars."

Some California scientists, wary of offending those who control the lifeblood of their research, privately grumble about an "old boys network."

Joe Mathews, co-author of "California Crackup," a study of major issues in state government, said last week: "California ballot initiatives are a terrible way to make public policy. And they are even worse as a method for making scientific policy."

The stem cell agency has a different view. Alan Trounson, president of the San Francisco-based enterprise, said: "There is no evidence that any of CIRM's funding decisions have been driven by conflicts of interest. Indeed, CIRM rigorously enforces its conflict of interest rules at each stage of the funding process to ensure that all decisions are made on the merits of the proposal for funding and not as a result of any conflicts of interest."

Mathews, California editor of Zocalo Public Square, and others point to the creation of the California stem cell agency as an example of abuse of the initiative process by special interests. The 10,000 words in Proposition 71 were written in private by Bay Area real estate investment banker Robert Klein and a handful of associates, who quietly determined the composition of the board. Klein later served six years as the first chairman of the stem cell agency, leaving in June 2011.

Klein later argued publicly that placing medical school deans and university and research institution executives on the board provided the expertise needed to make the decisions about how to spend the research money. However, the makeup of the board also served to win the support of institutions that envisioned the prospect of fresh cash – in this case money that the state borrows via bonds.

Mathews described the state's initiative process this way: "Essentially, to win the support of various groups whose money and backing is important to passage of a bond, a sponsor of an initiative bond will set up rules and include money specifically intended for each group. This is a form of pay-to-play. Agree to back the initiative, and you're in."

Bob Stern, who co-wrote the California Political Reform Act, said, "It would have been better had institutions receiving grants not to have had their representatives on the board awarding grants."

Trounson said the board follows "best practices" when it comes to grants and legal conflicts of interest. The agency has worked out an unusual procedure to prevent its directors from violating conflict of interest laws as they vote on applications that seek as much as $20 million each. Before each public session, agency attorneys determine which board members cannot vote on a proposal because of legal conflicts of interest. Applications to be approved are considered as a group. Each board member then votes on the entire group by saying, "Yes, on all those except with which I have a conflict."

No final tally is announced. The public can ferret out the overall vote a month or two later in the minutes of the meeting on the CIRM website (http://www.cirm.ca.gov). But the minutes do not list individual votes or conflicts of interest.

Domination of the board by academics and nonprofit institutions has led to bitter complaints from business. Less than 7 percent of all awards have gone to for-profit enterprises. Currently, however, the agency is embracing industry more warmly in an effort to commercialize stem cell research, which raises another set of coziness problems. They surfaced in July and again this month.

Klein, who led the stem cell ballot campaign before becoming chairman of the agency, appeared before his old board to lobby on behalf of a $20 million request from StemCells Inc. of Newark. The California firm was founded by the eminent Stanford stem cell scientist Irv Weissman. He sits on StemCells Inc.'s board, and he and his wife hold 273,821 shares of stock in the firm. Weissman was also an important backer of Proposition 71, working the "billionaire circuit" and raising more than $1 million for the campaign, according to an article in San Francisco magazine.

CIRM's reviewers had rejected StemCells Inc.'s application. After Klein made his pitch in July, the board sent the application back for re-review, an unusual procedure.

When the application returned to the board early this month, reviewers again rejected it. Klein again importuned his former colleagues, and – following a closed door session – the board approved the award, 7-5.

Eleven members were disqualified from voting because of legal conflicts of interest. It was the first time in the board's eight-year history that it approved an application twice rejected by reviewers.

Mathews said no likelihood exists of changing the board structure at CIRM. He said it is "baked in" by Proposition 71. That's because Klein and company wrote into the initiative a requirement for a super, super-majority vote – 70 percent – of each house of the Legislature to make any modifications.

Another initiative could be mounted, but that possibility is also exceedingly remote. 

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Text of Comments on Awards to Stem Cell Directors’ Institutions

September 30th, 2012 3:55 pm


Here is the full text of comments made
by the California stem cell agency, Joe Mathews, co-author of
California Crack-Up” and Bob Stern, former president of the
Center for Governmental Studies and co-author of the California
Political Reform Act
, in connection with the Sept. 23, 2012, article
in The Sacramento Bee headlined “Stem Cell Cash Mostly Aids Directors' Interests.” The comments were abbreviated for
publication in The Bee because of newspaper space constraints.

Comments by Alan Trounson, president of
CIRM:

“To make sure we do the best job of
managing taxpayer's money it's natural that we turn to people who
know most about stem cells and stem cell research. In fact, as the
state's own Little Hoover Commission reported in its analysis of
CIRM: “The fact that CIRM funding has gone largely to prestigious
California universities and research institutes is hardly surprising
and should be expected, given the goals of Proposition 71 and the
considerable expertise resident in these research centers.” But in
recruiting the best minds, we also adopt best practices to ensure
that there is no conflict of interest. Every board member has to
recuse themselves from voting on, or even being part of a discussion
on anything to do with their own institution, or to an institution or
company that they have any connections to. All this is done in
meetings that are open to the public. CIRM’s conflict of interest
rules have been subject to multiple reviews – by the Bureau of
State Audits, the Little Hoover Commission and the Controller – and
there is no evidence that any of CIRM’s funding decisions have been
driven by conflicts of interest. Indeed, CIRM rigorously enforces its
conflict of interest rules at each stage of the funding process to
ensure that all decisions are made on the merits of the proposal for
funding and not as a result of any conflicts of interest. 

“In addition all funding applications
are reviewed by an independent panel of scientists on our Grants
Working Groups, all of whom are out-of-state and meet strict conflict
of interest requirements, and it is their recommendations that help
guide the ICOC (CIRM governing board) on what to fund.”

Joe Mathews' comments:

“California ballot initiatives are a
terrible way to make public policy. And they are even worse as a
method for making scientific policy. 

“It's not merely that
this initiative was drafted in such a way as to benefit the
enterprises of its directors. It's that, under this initiative's own
provisions and the California constitution, it's so hard to change
Proposition 71 and fix what ails CIRM. Effectively, these provisions are
baked in, and nothing short of another vote of people can really make
the change. (Yes, there are provisions, as you know, that permit the
legislature by super-majority to do things, but supermajorities are
effectively out of reach in California). 

“Sadly, initiatives
like Proposition 71 are not uncommon. Many measures are drafted to benefit
the people who would support the measure, or oversee the program
established. This has been very common with bonds. Essentially, to
win the support of various groups whose money and backing is
important to passage of a bond, a sponsor of an initiative bond will
set up rules and include money specifically intended for each group.
This is a form of pay-to-play. Agree to back the initiative and
you're in. And it happens because there's no rule against it and
because passing initiatives in California require difficult,
expensive campaigns. 

“And this sort of thing will continue
to happen. There is no serious push to do anything about this.
Indeed, good government groups and reformers in California have
opposed changes to the initiative process -- because they want to use
the process for their own schemes.”

Bob Stern's comments:

“It would have been better had
institutions receiving grants not to have had their representatives
on the board awarding grants. On the other hand, we want to have the
most knowledgeable people on the board overseeing this very important
program. The question: Were these people the only qualified ones to
sit on the board?”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

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Anticipated short-term cell therapy industry clinical milestones

September 30th, 2012 3:52 pm
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What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.  


There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.


CellCoTec (http://www.cellcotec.com)
  • Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application.  The CE mark application is under review and they anticipate a response in October.  
  • This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.



ERYtech Parma (http://www.erytech.com)

  • Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2012. 


GamidaCell (http://www.gamidacell.com)

  • Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012.  They have not been shy about the fact they expect to be in the market in 2013.


Innovacell (http://www.innovacell.com)

  • They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed.  Status unknown.


Miltenyi Biotec (www.miltenyibiotec.com)

  • Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.


NovaRx (http://www.novarx.com)

  • Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.


NuVasive (http://www.nuvasive.com)

  • They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.


Sanofi's Genzyme (http://www.genzyme.com)

  • Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.


Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  



--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

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