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U-Buffalo spinout advances tarantula venom-based therapy for muscular dystrophy

Biophysicists at the University at Buffalo (NY) have found a protein in tarantula venom that shows promise as a therapy for muscular dystrophy (MD) and have formed a start-up to advance the drug to clinical trials. Fredrick Sachs, PhD, professor of physiology and biophysics at UB, and colleagues discovered the peptide, called GsMTx4. Because therapies for MD are classed as orphan drugs by the U.S. Food and Drug Administration, allowing a shorter testing period than normal drugs, Sachs anticipates that Buffalo-based Rose Pharmaceuticals may obtain FDA approval of the peptide for human use within two years. In addition to MD, the peptide has potential as a therapy for several other conditions, including neuropathic pain and atrial fibrillation.

In collaboration with the Wellstone Muscular Dystrophy Center at Children’s National Medical Center in Washington, DC, Sachs’ team tested the effect of GsMTx4 on mice with MD. Results showed that the drug increased muscle strength and caused no mortality, morbidity, or toxicity. Rose Pharmaceuticals now is developing methods to administer the drug. The peptide and its mirror image are covered by U.S. patents obtained by UB’s Office of Science, Technology Transfer, and Economic Outreach (STOR) and licensed to the start-up. No other drugs are known to act specifically on mechanosensitive ion channels — the target of GsMTx4, according to Sachs.