A grandfather enlists the help of UB scientists to conquer his
grandson’s muscular dystrophy.
Published February 28, 2013
Tonus Therapeutics, a UB spinoff company, has opened its first-ever headquarters in UB’s New York State Center of Excellence in Bioinformatics and Life Sciences.
Tonus develops drugs that target mechanosensitive ion channels, which are tiny conduits that help control the flow of important substances, such as calcium, into cells. The firm’s first project is developing a therapy for muscular dystrophy using GsMTx4—a peptide that UB scientists first discovered in the venom of the Chilean rose tarantula.
This research is of particular significance because the peptide, now made by chemical synthesis, is considered an “orphan drug” for muscular dystrophy, a designation that the U.S. Food and Drug Administration gives to recognize promising methods of treating rare diseases.
Tonus was founded in 2009 by three UB researchers and a local stockbroker whose grandson has a severe form of muscular dystrophy, a disease that causes patients’ muscles to atrophy.
“Being located in a hub of research activity in Buffalo, close to other entrepreneurs and biotech startups, is important to us,” says the grandfather, Jeff Harvey, who serves as Tonus’ chief financial officer. “The Buffalo Niagara Medical Campus is extremely important to us because it provides ready contact with experienced entrepreneurs and potential clinical and commercial partners, including those within the Center of Excellence.”
The opening of offices in the Center of Excellence follows other recent milestones for Tonus. In November, the company licensed UB patents relating to GsMTx4 through UB’s Office of Science, Technology Transfer and Economic Outreach (UB STOR).
“When companies like Tonus partner with the Center of Excellence, it’s a win for everyone involved,” says Marnie LaVigne, UB associate vice president for economic development. “Tonus benefits from the technological and business resources available here, and their continued growth will be an asset to Western New York’s economy. We’re excited to work with such a passionate and driven team as they navigate this important stage of development.”
Besides Harvey, the founding principals of Tonus Therapeutics are three UB faculty members: SUNY Distinguished Professor Frederick Sachs, Research Assistant Professor Thomas Suchyna and Research Associate Professor Philip Gottlieb, all members of the Department of Physiology and Biophysics in the School of Medicine and Biomedical Sciences.
Sachs’ team first came across GsMTx4 more than a decade ago while studying the effect of venoms on mechanosensitive ion channels.
These channels, which Sachs co-discovered in 1983, connect the inside of a cell with its outside. Normally, in healthy cells the channels are closed, but when a cell is stretched or contorted, the channels open and let calcium and other substances into the cell.
This is what happens in muscular dystrophy: Due to a defective gene, patients are missing the fiber-like reinforcing protein called dystrophin that helps muscle cells keep their shape, Sachs explains. This causes the cell membrane to get stretched more easily, prompting the ion channels to open, letting calcium flood in. The end result of this chain reaction is that the body “starts digesting muscle from the inside out,” Sachs says.
Tonus has 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, Sachs says. He added that the drug is quite “sticky” and is capable of staying in the body for a long time without breaking down. This means it could be possible to deliver low doses infrequently, reducing costs for patients.
Prior to their move into the Center of Excellence, the Tonus team benefited from other UB resources and partnerships. Harvey is a graduate of the UB School of Management’s High-Tech Center for Entrepreneurial Leadership program, and Tonus received funding through the UB Center for Advanced Biomedical and Bioengineering Technology (UB CAT). The Buffalo Children’s Guild and the John R. Oishei Foundation have provided additional funding.
“The whole project on mechanosensitive ion channels and GsMTx4, and its use in treating muscular dystrophy, is 100 percent Buffalo; everything was discovered here,” Sachs notes.
Besides investigating the effects of GsMTx4 on muscular dystrophy, Tonus also is exploring its application to other diseases, including sickle cell anemia, xerocytosis and chronic kidney failure.