The Gwendolyn Strong Foundation (GSF), an all-volunteer nonprofit organization created to change the future of the genetic disease spinal muscular atrophy (SMA), has awarded a targeted research grant to Dr. Alex MacKenzie of Children’s Hospital of Eastern Ontario Research Institute (CHEO). The $30,000 grant will directly fund an open-label, dose response clinical trial in Canada investigating the effect of low-dose Celecoxib in patients with SMA type II and III.
SMA is a degenerative disease that causes weakness and wasting of the voluntary muscles. Although most common in infants and young children, SMA can impact adults. And the disease does not discriminate based on race or gender.
There are several primary types of SMA – types I, II, III, and IV – typically correlating to severity, age of onset, and physical milestones achieved by patients. Regardless of type, patients with SMA are missing a gene that produces SMN2, a protein critical to the function of the nerves that control the muscles. As the motor neuron network breaks down, the ability of the brain to control muscles diminishes and with less control and use, muscles weaken and waste away. SMA eventually impacts every muscle in the body hindering the ability to walk, stand, sit, eat, breathe, and swallow.
Although there is no treatment for SMA, research from the bench to the bedside is extremely mature and there are several promising potential therapeutics currently working their way through human clinical trials.
Celecoxib is a nonsteroidal anti-inflammatory drug (NSAID) that has been approved by the U.S. Food and Drug Administration (FDA) and Health Canada for treatment of pain caused by conditions such as arthritis. Although not developed specifically for SMA, favorable pre-clinical studies at CHEO in SMA mice and human SMA cells indicate that low-dose Celecoxib may ‘upregulate’ SMN2 protein and, therefore, indicate a new, cost effective disease modifying treatment for SMA. The aim of the study at CHEO is to investigate which dose of Celecoxib is most effective in improving SMN2 gene expression in patients with SMA.
“In previous work funded in part by the GSF, my colleague showed a particular cellular pathway demonstrated the common FDA approved arthritis drug, Celecoxib, could activate that pathway therefore raising SMN2 protein levels in SMA,” said Dr. Alex MacKenzie, Senior Scientist at CHEO. “Through this study, we shall now establish whether Celecoxib increases SMN2 in actual SMA patients hopefully paving the way for a new therapeutic widely available around the world.”
“We’re extremely excited to support the important work of Dr. MacKenzie and his team at CHEO,” said Bill Strong, co-founder of the Gwendolyn Strong Foundation. “There are several exciting potential therapeutics on the horizon for SMA, and the Celecoxib avenue, if proven effective through this study, would provide a low cost, low risk approach that would be immediately available globally to all impacted by this disease. There’s no doubt this should be explored.”
CHEO is planning to begin enrollment in the Celecoxib open-label, dose response clinical trial in early 2016.
For more information on CHEO or the Celecoxib SMA clinical trial, contact Adrienne Vienneau: avienneau(at)cheo(dot)on(dot)ca
The Gwendolyn Strong Foundation (GSF) (http://www.theGSF.org) is a unique all-volunteer, nonprofit organization created to change the future of spinal muscular atrophy or SMA. Inspired by their incredible daughter, Gwendolyn, Bill and Victoria Strong started the GSF to fill voids in the SMA ecosystem by utilizing technology, social media, and branding to raise funds to further SMA research, provide technology grants, advocate for and support families impacted by SMA, and raise global awareness of this brutal disease.
For more information about the GSF or SMA, contact Bill Strong: bill(at)theGSF(dot)org