The Gwendolyn Strong Foundation and FightSMA, two all-volunteer nonprofits working toward a treatment for the childhood neuromuscular killer spinal muscular atrophy (SMA), announced this week a jointly funded $250,000 grant to be awarded to Dr. Christian Lorson and his research team at the University of Missouri.
The grant will support Dr. Lorson’s work to increase production of Survival Motor Neuron (SMN), a protein that is critical for muscle strength in persons diagnosed with SMA.
SMA is caused by the absence of the SMN1 gene which produces SMN protein. In spinal muscular atrophy, a “backup” gene produces the protein, but in insufficient amounts, resulting in the loss of neuronal activity and muscle strength. In its most severe form (“Type One”), SMA is the leading cause of infant death.
“We have been working for several years towards the development of a potential drug to treat Spinal Muscular Atrophy (SMA),” said Dr. Lorson. “Now we have optimized a compound referred to as an “anti-sense oligonucleotide” or “ASO.” (An ASO is a synthetic stretch of nucleic acid that is highly resistant to degradation (so a single dose is active for several months) and is very specific, as it binds tightly to a segment of SMN RNA identified in the lab.)
“For our drug, the ASO binds to a powerful regulatory element called Element 1 or ‘E1.’ Inhibition of this region dramatically increases the production of the protein that protects kids from SMA,” said Dr. Lorson. “Based upon our preliminary results,” said Dr. Lorson, “our ASO exhibits no detectable toxicity and from a single injection, we observe a 400-600% increase in average survival with some animals living 10-fold longer than untreated littermates. The response we observe is truly exciting and opens the door for a completely new genetic target in SMA.”
“Dr. Lorson’s lab is seeing some of the most exciting results in the SMA field,” said FightSMA Chair Joe Slay. “We are proud to sponsor this vital work to bring new compounds into the pipeline.”
“It’s truly a new day in the world of SMA research with several potential therapeutics making their way through human clinical trials,” said Bill Strong, Gwendolyn Strong Foundation co-founder. “And we’re honored to expand that pipeline by supporting the impressive work of Dr. Lorson’s lab to move their ASO drug closer to the clinic.”
“Additional therapeutic targets such as E1 need to be developed as it is an unfortunate reality that the first drug candidates are rarely the last or only therapeutic options,” said Dr. Lorson. “Development of additional high-impact targets will be essential.”
Prior funding for this project has been provided by the National Institutes of Health (NIH) and by CureSMA.