Gwendolyn Strong Foundation Awards $135,000, Partners On Promising Gene Therapy Program

The Gwendolyn Strong Foundation, a Santa Barbara, CA based nonprofit organization striving to increase awareness of Spinal Muscular Atrophy (SMA), the #1 genetic killer of young children, and accelerate cure focused SMA research, has awarded $135,000 to two promising research programs: $75,000 to FightSMA for their SMA gene therapy partnership with Dr. Brian Kaspar at Nationwide Children's Hospital and The Ohio State University and $60,000 to Dr. Hans Keirstead at University of California, Irvine for his stem-cell based motor neuron replacement program.

Online PR News – 20-May-2010 – – Santa Barbara, CA – The Gwendolyn Strong Foundation (GSF), a nonprofit organization striving to increase awareness of Spinal Muscular Atrophy (SMA), the #1 genetic killer of young children, and accelerate cure focused SMA research, has awarded $135,000 to two promising research programs: $75,000 to FightSMA for their SMA gene therapy partnership with Dr. Brian Kaspar at Nationwide Children's Hospital and The Ohio State University and $60,000 to Dr. Hans Keirstead at University of California, Irvine for his stem-cell based motor neuron replacement program.

“We are thrilled at the opportunity to continue partnering with incredible organizations and researchers in our drive to have a direct, material impact on moving groundbreaking SMA research towards a cure for all people impacted by SMA,” said Bill Strong, GSF co-founder. “But it’s important to recognize that none of this is possible without the tireless support of thousands of people around the world – some impacted by SMA and some not – who have rallied around our efforts to help end this brutal infant killer.”

SMA is a terminal, degenerative genetic disease that impacts the voluntary muscles in infants and children including the ability to walk, sit, stand, eat, breathe, and even swallow. Ninety percent of children born with SMA die before the age of two. Although SMA is not a household name, the gene responsible for SMA is carried by 1 in 40 people or nearly eight Million Americans. There is currently no treatment or cure and although SMA research is underfunded, it is extremely mature. GSF is focused on helping the SMA community make sure resources are not an obstacle to attaining that cure.

Dr. Brian Kaspar’s Gene Therapy Program – $75,000 Award

FightSMA (http://FightSMA.org), a leading Virginia based nonprofit organization focused on SMA research, has a long-standing stake in gene therapies focused on SMA. They are now embarking on the next phase of this promising research through a partnership with Dr. Brian Kaspar at Nationwide Children's Hospital and The Ohio State University. FightSMA’s program is a multi-year endeavor with a goal of helping move the program closer to human clinical trial. GSF’s $75,000 award will be used specifically to help fund a critical initial portion of the program: pre-clinical work of proving safety and toxicity of the science.

But it’s important to recognize that none of this is possible without the tireless support of thousands of people around the world – some impacted by SMA and some not – who have rallied around our efforts to help end this brutal infant killer.

“We are tremendously excited about the potential for gene therapy in SMA and our continuing collaboration with the Gwendolyn Strong Foundation,” said Martha Slay, FightSMA President. “Gene therapy research is a marathon run, but the work of Dr. Kaspar will advance the field significantly for SMA.”

In addition to this $75,000 award, GSF has announced a long-term partnership with FightSMA to continue fundraising for their commitment to Dr. Kaspar’s gene therapy program.

Dr. Hans Keirstead’s Motor Neuron Replacement Program – $60,000 Award

GSF’s $60,000 award to Dr. Hans Keirstead at University of California, Irvine will be used exclusively to fully fund critical experiments requested by the Federal Drug Administration, which will enable the human clinical trial for this promising stem-cell based motor neuron replacement program. Dr. Keirstead’s program will initially focus on SMA Type I, the most severe form of the disease, but the pre-clinical and clinical data will be directly relevant to SMA Type II and III, ALS-Lou Gehrig’s disease, chronic spinal cord injury, and polio, all of which are characterized by motor neuron loss.

"We are forever grateful to the SMA community for their support in helping us move our discoveries from the bench to the bedside," said Dr. Hans Keirstead, Associate Professor at the Reeve-Irvine Research Center. "Together, we have pioneered a first for SMA, and for the stem cell field."

This most recent $60,000 award is in addition to over $170,000 GSF and its programs have donated in support of Dr. Keirstead’s research over the past 12 months.

About Gwendolyn Strong Foundation (GSF) – GSF is 501(c)(3) public charity based in Santa Barbara, California dedicated to increasing global awareness of Spinal Muscular Atrophy (SMA), the #1 genetic killer of infants and young children, and accelerating research toward a cure. To learn more about SMA and GSF, please visit http://GwendolynStrongFoundation.org or contact GSF at (805) 679-1679.

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