Published October 20, 2014
A drug developed by University at Buffalo scientists from a small protein found in spider venom is moving forward as a promising treatment for Duchenne muscular dystrophy, a fatal genetic disease affecting boys.
To help develop the drug, Sachs co-founded the UB spinoff firm Tonus Therapeutics with two colleagues in his department — Thomas Suchyna, PhD, research assistant professor, and Philip Gottlieb, research associate professor — along with Jeff Harvey, a local stockbroker whose grandson has Duchenne muscular dystrophy.
The GsMTx-4 protein has since been modified and chemically synthesized in the laboratory to produce AT-300, a potential therapy designed to slow the muscle deterioration that characterizes muscular dystrophy.
Sachs’ team is now studying AT-300’s effectiveness in dystrophic mice. When these studies are completed in 2015, the researchers will apply to the U.S. Food and Drug Administration (FDA) for approval of an Investigational New Drug and the first clinical trials in humans.
The FDA has already declared the protein an “orphan drug” for muscular dystrophy, a designation recognizing promising methods of treating rare diseases.
The biopharmaceutical company Akashi Therapeutics Inc., based in Cambridge, Mass., recently acquired the rights to the AT-300 protein from Tonus Therapeutics, bringing the therapy another step closer to reality.
“Akashi Therapeutics has the funding and resources to propel development of this new potential drug,” says UB Vice Provost Robert J. Genco, PhD, DDS. Genco oversees UB’s Office of Science, Technology Transfer and Economic Outreach (STOR), which commercializes technologies developed at the university.
Akashi aims to develop a cocktail of medications to transform Duchenne muscular dystrophy into a chronic, manageable condition.
Both Tonus and UB will receive royalties on sales of any treatments resulting from AT-300.
Sachs will continue to help oversee research and development.
In patients with dystrophy, genetic mutations weaken the membrane of muscle cells, enabling large amounts of calcium to infiltrate the cells. This sets off a chain reaction that leads to muscle degeneration.
Laboratory studies show that AT-300 stops the dangerous calcium influx by keeping mechanosensitive ion channels shut when muscle cells are stretched, says Sachs.
The protein also holds potential for preventing cardiac failure, one of the most common causes of death in dystrophic boys, he adds.
In addition, the modified form of the drug is designed to remain stable for a long time in the human body. This could mean patients would need infrequent doses, which could hold down costs.