BUFFALO, N.Y. -- University at Buffalo biophysicists who are
testing a protein found in tarantula venom as a potential therapy
for muscular dystrophy have received foundation support for their
The Children's Guild Foundation has given $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 and ultimately to gain FDA approval for use of
the drug in humans.
The FDA has already approved it as an orphan drug for muscular
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 http://www.sachslab.buffalo.edu,
and on Facebook under Rosie's Therapy for Muscular Dystrophy.
The Children's Guild Foundation is a non-profit, private
organization that advocates and funds rehabilitative health care,
research, education and therapeutic recreation programs for special
The University at Buffalo is a premier research-intensive public
university, a flagship institution in the State University of New
York system and its largest and most comprehensive campus. UB's
more than 28,000 students pursue their academic interests through
more than 300 undergraduate, graduate and professional degree
programs. Founded in 1846, the University at Buffalo is a member of
the Association of American Universities.