Published October 6, 2010
UB biophysicists who are testing a protein found in tarantula venom as a potential therapy for muscular dystrophy have received foundation support for their work.
The Children’s Guild Foundation has awarded $125,000 to Frederick Sachs, PhD, SUNY Distinguished Professor and UB Distinguished Professor in the Department of Physiology and Biophysics, and his team, which is seeking to advance the peptide—called GsMTx4—to clinical trials with the goal being to gain FDA approval for use of the drug in humans.
The FDA has already approved it as an orphan drug for muscular dystrophy.
The team discovered the peptide, which is protected by a U.S.
patent awarded to UB, about 15 years ago. The Sachs lab is also
exploring the peptide’s application to several other
conditions, including the cardiac arrhythmias commonly associated
with dystrophy, neuropathic pain, Parkinson’s disease and
sickle cell anemia, all diseases that affect cell mechanics.
“Duchenne muscular dystrophy is a far too common and fatal genetic disease that affects thousands of children and devastates families,” said James A. Bourne, board chair of the Children’s Guild Foundation. “Finding a treatment for MD is an effort that the foundation supports wholeheartedly.”
Recent testing on GsMTx4 in dystrophic laboratory mice showed that the drug increases muscle strength without causing mortality, morbidity or toxicity. Sachs said it is the only known drug to act specifically on mechanosensitive ion channels.
More information about the work is available at Sach's laboratory website and in the Facebook group Rosie’s Therapy for Muscular Dystrophy.