$30,000 Targeted Research Grant Funds SMA Celecoxib Clinical Trial

$30,000 Targeted Research Grant Funds SMA Celecoxib Clinical Trial

We're excited to announce 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.

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, Principal Investigator 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@cheo.on.ca

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