Frederick Sachs, PhD, president of Tonus Therapeutics, holds the
source of a promising new drug targeting muscular dystrophy.
UB Spinoff Opens Headquarters in Center of Excellence
Published
January 29, 2013
Tonus
Therapeutics, a UB spinoff firm developing a promising new
therapy for muscular dystrophy, has opened its headquarters in UB’s New York
State Center of Excellence in Bioinformatics and Life
Sciences.
“Being located in a hub of research activity in Buffalo,
close to other entrepreneurs and biotech startups, is important to
us,” says Jeff Harvey, the firm's co-founder and chief
financial officer.
“The Buffalo Niagara
Medical Campus provides ready contact with experienced
entrepreneurs and potential clinical and commercial partners,
including those within the Center of Excellence.”
Promising Drug Derived from Tarantula Venom
Tonus is developing a drug derived from a peptide that UB
scientists discovered in the venom of the Chilean rose
tarantula.
The U.S. Food and Drug Administration has designated the
peptide, now made by chemical synthesis, an “orphan
drug” for muscular dystrophy, a designation recognizing
promising methods of treating rare diseases.
Peptide May Suppress Muscular Dystrophy’s Symptoms
The Tonus researchers’ findings stem from their long-term
studies of venoms targeting mechanosensitive ion
channels—tiny conduits that help control the flow of
important substances, such as calcium, into cells.
Due to a defective gene, people with muscular dystrophy lack the
protein dystrophin, which helps muscles keep their shape.
Consequently, their cell membranes stretch more easily, prompting
the ion channels to open, letting calcium flood in.
This chain reaction causes the body to start “digesting
muscle from the inside out,” says Tonus President Frederick
Sachs, PhD, SUNY Distinguished Professor of Physiology and
Biophysics.
The peptide that the UB researchers discovered, GsMTx4, keeps
the ion channels shut, offering potential for a drug that could
suppress the debilitating symptoms of a host of diseases, including
muscular dystrophy.
Drug’s Properties May Help Reduce Cost to Patient
Tonus is also exploring the peptide’s effects on sickle
cell anemia, xerocytosis and chronic kidney failure.
Researchers have gathered preliminary data showing that GsMTx4
is nontoxic in mice and does not disturb heart function in mice or
ferrets or isolated human heart muscle.
The drug is capable of staying in the body for a long time
without breaking down, so it could be possible to deliver low doses
infrequently, reducing patients’ costs, Sachs notes.
Child’s Illness Spurred Collaboration
Tonus was founded in 2009 when Harvey, a stockbroker whose
grandson has a severe form of muscular dystrophy, joined forces
with UB researchers.
In addition to Harvey and Sachs, the other co-founders, both in
physiology and biophysics, are vice presidents Thomas Suchyna,
research assistant professor, and Philip Gottlieb, research
associate professor.
“The whole project on mechanosensitive ion channels and
GsMTx4 and its use in treating muscular dystrophy is 100 percent
Buffalo,” Sachs says.
“Everything was discovered here.”
UB Partnerships Advance New Firm
Tonus has benefited from several UB resources and partnerships,
including: