U.Va. Inventors Receive FDA Orphan Drug Approval for Pancreatic Cancer Treatment

University of Virginia start-up company Tau Therapeutics LLC has been granted Food and Drug Administration orphan drug status for a pioneering treatment for pancreatic cancer.

Developed by U.Va. inventors Timothy L. Macdonald, Ph.D., Lloyd S. Gray, M.D., and collaborators, the treatment takes a revolutionary approach to the fight against cancer.

Unlike many existing cancer therapies, which are designed to attack cancer cells and often damage healthy cells in the process, the
researchers’ treatment inhibits tumor growth by blocking the entry of calcium into cancer cells.

Although calcium has long been known to be necessary to the growth ofcells, it was Gray who first discovered calcium's pathway into some cells through what is known as a T-type calcium channel, or T-channel. Gray partnered with Macdonald to develop chemical tools to block this channel, effectively stopping calcium from entering cells and bringing cell growth to a halt.

“When cells decide to grow, they transfer calcium into the cell,” said Macdonald, professor of chemistry at U.Va. “If we block the mechanism through which calcium enters the cell — in this case, a T-channel — we keep the cells from growing.”

While several types of cells rely on T-channels for calcium entry, Macdonald and Gray decided to target their efforts on oncology. Their anti-cancer calcium channel-blocking technology was patented by the U.Va. Patent Foundation and licensed to Tau Therapeutics, which the researchers founded with President and CEO Andrew J. Krouse to further develop and commercialize their discovery. Krouse is an alumnus of the Darden Executive MBA program.

“Macdonald and Dr. Gray have taken a truly novel approach to fighting cancer,” said Robert S. MacWright, J.D., Ph.D., executive director of the U.Va. Patent Foundation. “Their targeted drug therapies could have a great impact on the future of cancer treatment.”

The pancreatic cancer treatment for which Tau Therapeutics recently received FDA orphan drug status is the existing drug mibefradil, trade-named Posicor™. The T-channel-blocking drug was once marketed as ahypertension medication. Although it was withdrawn from the market due to drug-drug interactions with cholesterol-lowering medications, mibefradil has demonstrated efficacy in treating pancreatic cancer in animals and has also received FDA orphan drug status for the treatment of ovarian cancer. Further, because mibefradil is non-toxic, it could be an excellent alternative to existing cancer drugs.

“Mibefradil will be among the safest of oncology drugs,” Macdonald said. “It holds great promise in the treatment of cancer.”

In addition to pancreatic and ovarian cancer, mibefradil may also be effective in treating glioblastoma, considered the most lethal form of brain cancer, and metastatic melanoma.

About the University of Virginia Patent Foundation

The University of Virginia Patent Foundation is a not-for-profit corporation that serves to bring U.Va. technologies to
the global marketplace by evaluating, protecting and licensing intellectual property generated in the course of research at U.Va. The Patent Foundation reviews and evaluates nearly 200 inventions per year and has generated approximately $85 million in licensing revenue since its formation in 1978.

About Tau Therapeutics

Tau Therapeutics LLC is a development stage biopharmaceutical company built on a revolutionary means of inhibiting the growth of cancer cells. Spun out of the University of Virginia Medical Center by faculty members Lloyd S. Gray and Timothy L. Macdonald, and founded on technology licensed from the University of Virginia Patent Foundation, the company is focused on the research, development, repositioning and commercialization of T type calcium channel antagonists as cytostatic anti-cancer drugs. Tau
Therapeutics is the world leader in developing calcium T-channel blockers for oncology and other indications. Tau offers a new way of treating cancer. Its goal is to treat cancer like any other chronic disease — a daily pill for the maintenance of cancer.