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, received $650,000 in grants in 2012 from Sophia’s Cure Foundation for SMA research and Phase 1 clinical trial development which is set to launch in 2013. Working together with Nationwide Children’s, Sophia’s Cure Foundation has raised more than $2.3 million dollars for Dr. Kaspar’s program during the last three years alone.
“Grant dollars from Sophia’s Cure Foundation are being used to support the pivotal studies required to submit an Investigational New Drug application to initiate human clinical trials in SMA patients which we hope to do in 2013,” said Dr. Kaspar, also a faculty member at The Ohio State University College of Medicine. “SMA research and therapeutic development stands to gain from this generous investment as we all look forward to translating our research to advance human clinical trials. Sophia’s Cure Foundation has been the lead funder of this program and their incredible investment in this lab has accelerated our program by many years. We are honored and extremely appreciative for this significant support.”
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 which is 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 is a non-profit public charity which was formed by the Gaynor family in New York City shortly after their daughter, Sophia, was diagnosed with Spinal Muscular Atrophy in 2009. The foundation was created to assist in funding for clinical research towards finding a cure for SMA and to offer support to families affected by this disease by providing advocacy, awareness, education and support. The foundation was formed three years ago by Vincent Gaynor, a One World Trade Center construction worker, and Catherine Gaynor, who left her job to care for Sophia when they learned she was sick. With their leadership and 100 percent volunteer involvement the foundation has raised more than $3 million for SMA research to date.
“We run our foundation from the heart, because when you lead with your heart good things will always happen,” said Vincent Gaynor, co-founder of Sophia’s Cure Foundation. “People gravitate toward that. They believe in us and Sophia and know we will get the funds into the right hands to find a cure. Our partnership with Dr. Kaspar and Nationwide Children’s Hospital will yield great results for Spinal Muscular Atrophy research.”