Update from Dr. Lorson at University of Missouri on Spinal Muscular Atrophy Program being Funded by Cure SMA

Originally published on August 6, 2014.

Christian Lorson PhD, investigator in the Bond Life Sciences Center and Professor of Veterinary Pathobiology at the University of Missouri, has been working on the development of a novel compound found to be efficacious in animal models of disease. The compound is an antisense oligonucleotide (ASO). It increases life span in severe mouse models of SMA by four times and in intermediate mouse models of SMA by seven times. In April, a patent was filed for Lorson’s compound for use in SMA.

Cure SMA awarded $150,000 to Dr. Lorson and Dr. Burghes to investigate these new antisense therapies for Spinal Muscular Atrophy. The goals of the current Cure SMA funding for the program are two-fold:
1) to optimize the nucleic acid sequence of the drug, and
2) to establish the optimal dose of the ASO in severe mouse models of SMA.

“We are very grateful to Cure SMA for funding our ASO project as we hope we can continue to develop therapeutics that target an additional genetic element within SMN2. SMA is a very complex disease and it is possible that more therapeutic options will be required to effectively combat this disease,” stated Chris Lorson, Ph.D., Professor at University of Missouri.

Dr. Lorson’s therapeutic antisense oligonucleotide repairs expression from the back-up gene for the disease, called SMN2. The research was published May in in the Oxford University Press, Human Molecular Genetics. The drug developed by Lorson’s lab is conceptually similar to ISIS-SMNRx already in clinical trial developed by Isis Pharmaceuticals and a team of investigators at Cold Spring Harbor Laboratory headed by Dr. Adrian Krainer. Isis recently announced they have started Phase III clinical trials infants with SMA with their drug.

The Lorson drug is still in preclinical stages of drug development and several years from early stage clinical trials for safety. As mentioned, the Cure SMA funding was awarded to optimize the candidate drug. Once the final drug candidate is identified, a full battery of biodistribution and safety studies will need to be completed for the FDA in order to initiate clinical trials on this new drug.

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