You did it! $250k for SMA research
Thanks to the many people who took this cause to heart by voting everyday in March and urging their friends and family to do the same, we are happy to announce that Stop SMA has received $250,000 from Pepsi to fund a year of SMA research. We could not have done this without the support we received from the SMA community, our friends, our family, and even people who have heard of SMA for the first time. You did it. And we are forever grateful.
With the $250k donation, we are funding a coincidentally named research project called STOP SMA. The project is directed by Dr. Kathy Swoboda, part of a research team at the University of Utah’s department of Neurology. We were excited to help with the research at Dr. Swoboda’s office. This is research in the clinical trial phase and it needed funding. Research in this phase is actually getting into the hands of families for potential treatment of their children. Dr. Swoboda is one of the best SMA doctors in the country. To introduce her work is a note from Dr. Chris Lorson, Associate Professor at the University of Missouri’s Department of Veterinary Pathobiology. He is also the Science Director of Fight SMA.
Dr. Kathy Swoboda is a leading SMA researcher and has been instrumental in establishing current paradigms for SMA clinical trials. Her current proposal to FightSMA involving pre-symptomatic SMA children is an exceptionally powerful trial that addresses several fundamental aspects of SMA biology and SMA clinical trial design. Her work is ready and ongoing in the clinic – this grant will have an immediate effect upon SMA children and their families. Additionally, the early indications from this work strongly suggest that Dr. Swoboda’s trial will further support the effort to include SMA in a neonatal screening platform.
The Stop SMA research project (no relation to our organization) has studied the effectiveness of proactive treatment of infants with SMA before the infants began showing symptoms of the disease. In 2003, they began a pilot study using the drug, sodium phenylbutyrate. The results appeared promising and in 2007 they began a formal clinical trial. Enrollment for this trial ended in April 2010. Early results appear promising. One infant with SMA type 1 was able to achieve independant sitting. Two of six children who had SMA type 2 were able to achieve independent walking.
Over the next year, the trial will be completed and the results of the study will be used to plan a larger trial of early intervention of childten with SMA. This study provides a foundation for further studies and is another step towards curing spinal muscular atrophy.