Stem Cell :: CNS Foundation grantee Dr. Evan Snyder repays special-needs community with stem cell publication

The CNS Foundation welcomes the publication by CNS grantee Dr. Evan Snyder of experimental results that extend the promise of stem cell therapies to degenerative brain diseases of childhood.

His publication marks the achievement of a significant milestone on the road to brain repair therapies for special-needs children.

Evan Snyder, M.D., Ph.D., and lead author Jean-Pyo Lee, Ph.D., of the Burnham Institute for Medical Research demonstrated that human neural stem cells slow the onset of Sandhoff disease when implanted in the brains of mice with the condition. The study, to be published in Nature Medicine, is now available by advanced publication at the journal?s website.

Sandhoff is a lethal neurodegenerative disorder related to Tay-Sachs disease and is among a group of lipid accumulation disorders that, in total, affects 1-in-5000 births. Sandhoff results from a genetic deficiency in an enzyme (hexosaminidase) that breaks down unneeded lipid molecules. Without these enzymes, fatty “molecular garbage” builds up to toxic levels within brain cells, disrupting their normal function and ultimately resulting in brain cell death.

The neural stem cells used in the study appeared to benefit transplanted animals in a number of ways, including delivering functional hexosaminidase enzyme to the brain, reducing inflammation and supplementing damaged brain cells by functionally integrating into recipient brain tissue. The single stem cell administration described in the study significantly extended these animals? lifespan and improved their quality of life. This therapeutic effect was further increased when drugs that independently reduce lipid accumulation accompanied the stem cell transplant.

Although principally focused on Sandhoff, Dr. Snyder?s results are expected to advance the development of stem cell transplantation therapies for more complex childhood brain disorders like some forms of cerebral palsy, autism and ataxia-telangiectasia; and even adult disorders like Parkinson?s and Lou Gehrig?s disease.

“Dr. Snyder?s team has extended the promise of stem cell therapies to children with special-needs, including those with Sandhoff disease.” said Fia Richmond, founder ofChildren’s Neurobiological Solutions Foundation and mother of a brain-injured child. “The CNS Foundation is proud to have contributed major funding for this research along with the A-T Children’s Project on behalf of the 14 million special-needs children in the United States alone.”

CNS and our partners at the A-T Children?s Project provided $348,000 to Dr. Snyder for this important research. His grant, entitled Multimodal Stem Cell Action in Inherited CNS Disease, is a direct result of our organizations? 2004 Tarrytown Initiative which identified priorities for the development of neural stem cell therapies.

“I am delighted to see our contribution to Dr. Snyder?s research paying dividends in the form of proof-of-concept for a potential brain repair therapy,” said CNS science director Shane Smith, Ph.D. “On behalf of all CNS families I congratulate Dr. Snyder and his collaborators for their commitment to science urgently needed by our special children.”

Dr. Snyder and his team believe that their study may serve as the springboard for a clinical trial for Sandhoff or Tay-Sachs. CNS has opened a dialogue with him to discuss how our organization may continue to support his efforts to bring this stem cell therapy to special-needs children as quickly as possible.

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