SANTA BARBARA, CALIFORNIA -- 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 of Children'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.
About Children’s Neurobiological Solutions Foundation: CNS Foundation is a non-profit research foundation whose mission is to develop the potential to repair the damaged brain so that all special-needs children may experience a lifetime of possibilities. Since its founding by special-needs families in 2000, CNS has grown into a dynamic parent-driven organization that fosters an essential sense of community while funding scientific research that lays the groundwork for brain repair and regeneration. For more information about CNS visit www.cnsfoundation.org, e-mail info@cnsfoundation.org, or call (866) 267-5580.
Journal
Nature