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UC-Davis Receives Adult Stem Cell Grants

News Release, Huntington's Disease Society of America, August 22, 2009

The California Institute for Regenerative Medicine (CIRM), the state legislative stem cell agency, has announced today the awarding of two separate multi-million-dollar, multi-year grants to the University of California at Davis for the R&D of adult stem cell therapies in the treatment of neurodegenerative diseases as well as inherited blood and metabolic disorders.

The two grants include $2.7 million to be devoted to the development of a treatment for Huntington's disease using the type of adult stem cell known as mesenchymal stem cells (MSCs), and a separate grant worth $4.2 million to develop a safe treatment of infants in utero who may be predisposed to inherited blood cell disorders such as sickle cell anemia and thalassemia, among others.

According to Dr. Jan Nolta, director of the Stem Cell Program at UC-Davis and a principal investigator for the neurodegenerative diseases grant, "Today's award from CIRM gives us a terrific boost because it opens the door to developing an innovative and effective therapeutic tool for victims of neurodegenerative disorders like Huntington's disease. Diseases of the brain are incredibly challenging to treat. What we hope to do is create a stem-cell-based treatment that can deliver therapeutic molecules directly into damged cells in the brain."

In the U.S., Huntington's disease occurs with a frequency of one person per every 10,000 people. Dr. Nolta has been working with MSCs for the past 20 years, during which time the cells have demonstrated their vast therapeutic versatility. Under the new grant, Dr. Nolta and her colleagues intend to re-engineer the MSCs to deliver siRNA (small interfering ribonucleic acid) to damaged areas of the brain, where the siRNA can prevent the mutant proteins that are characteristic of Huntington's disease from being produced by cells. Hoping to exploit the natural "search and rescue" capacity of MSCs to target damaged regions of the brain, Dr. Nolta expects that such a therapy will preclude the need for systemic infusion of cell-based therapies into the CNS (central nervous system).

According to Judy Roberson, president of the Northern California chapter of the Huntington's Disease Society of America, "This is the best news. Families like mine have been waiting for an intervention for Huntington's since the gene was located 16 years ago. My husband and his brother and thousands of others died waiting for something, anything. Now, thanks to CIRM support and the research at UC-Davis, there's real hope for the first time."

In the second grant, a team of UC-Davis researchers led by Dr. Alice Tarantal will be exploring an in utero model to assess the viability of transplanted stem cells in the treatment of inherited blood disorders, before irreversible damage from the diseases has occurred. Professor of pediatrics and associate director of the UC-Davis Stem Cell Program, Dr. Tarantal explains, "Our team is poised to address crucial bottlenecks that have delayed these therapies from reaching our youngest patients. We greatly appreciate the support provided by CIRM to help us work toward these goals. Infants with congenital blood diseases can be diagnosed before they are born. We hope it may be possible to treat these young patients early and thus avoid the many problems such diseases can cause."

With more than 125 scientists and physicians on its faculty, UC-Davis is currently in the process of constructing a new Institute for Regenerative Cures in Sacramento, a facility that is supported by the CIRM and which will comprise 90,000 square-feet of state-of-the-art research laboratories and clinics.



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