SMA researchers receive grant for Spinal Muscular Atrophy research

Nationwide Children's Hospital receives $250,000 grant for SMA research

Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital, along with a team of Spinal Muscular Atrophy (SMA) researchers and clinicians, recently received a $250,000 grant for SMA research and clinic development from Sophia's Cure Foundation via the Pepsi Refresh Project.

Spinal Muscular Atrophy (SMA) is a group of inherited debilitating neurological diseases that cause progressive muscle degeneration and weakness throughout the body. There is no treatment for the progressive weakness caused by the disease. It is estimated that SMA occurs between one-in-6,000 and one-in-20,000 births. One-in-40 to one-in-80 "normal" men and women carry the gene for SMA, and if both a man and woman carry the gene, there is a 25 percent chance that any of their children will manifest SMA.

Sophia's Cure Foundation, a not-for-profit organization created to assist in funding clinical research for SMA and to offer support to families affected by SMA by providing advocacy, awareness and education, received the $250,000 grant from the Pepsi Refresh Project and awarded it to Dr. Kaspar and his team. The Pepsi Refresh Project has committed to award more than $20 million in 2010 to projects and research that will "move communities forward." Individuals can apply for these grants to benefit a variety of projects while web site visitors vote for the best ideas.

"We are extremely honored to be able to fund such an incredibly promising program," said Vincent Gaynor of Sophia's Cure Foundation. "Dr. Kaspar and his team are some of the most talented and devoted researchers in the field. It was our mission to educate the community on their promising work, and to bring together a united effort to see Dr. Kaspar's SMA research funded. There is a tremendous amount of work yet to be done, and we look forward to continuing our support of this research."

"The Pepsi Refresh Project is an innovative funding mechanism that is clearly making a major impact for community projects," said Dr. Kaspar, also a faculty member at The Ohio State University College of Medicine. "Spinal Muscular Atrophy research and therapeutic development stands to gain from this important and generous investment, and we all look forward to translating our research to advance human clinical trials."

Both Dr. Kaspar's team and Sophia's Cure Foundation are extremely thankful for everyone that voted for their application via the Pepsi Refresh program. It is clear that the SMA community worked together and united for a common goal to push this research and cause to first place.

"This is a major win for the SMA community," said Dr. Kaspar. "We are honored and extremely appreciative for the support from both of these organizations."

The grant of $250,000 will assist Dr. Kaspar and his team at Nationwide Children's in research and development, moving their gene therapy work toward scaleable production and purification methods to perform the pivotal safety studies and produce clinical grade material as they advance this program to the clinic.

Through his SMA research, Dr. Kaspar has worked closely with, and will continue to collaborate with a host of doctors and researchers in the field, all with the common goal to find a treatment for SMA.


Nationwide Children's Hospital


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