Charlottesville firm’s drug could be a brain cancer breakthrough

A Charlottesville-based company is hoping its drug therapy program will one day prolong and enhance the lives of cancer patients.

Tau Therapeutics, a start-up company with ties to the University of Virginia, has developed a drug treatment it believes will stop the growth of cancer cells.

The treatment, known as Interlaced Cytostatic Checkpoint Therapy, accumulates and destroys cancer cells while causing no damage to healthy cells.

It has also been successful at reducing the side effects of chemotherapy.

“By making chemotherapies more effective, we have a real opportunity to help those with cancer,“ said Lloyd Gray, one of three co-founders of Tau Therapeutics. “[We hope] to reduce the painful side effects of chemotherapies and extend both the quality and length of life.“

The U.S. Food and Drug Administration recently awarded the company an orphan drug designation for Mibefradil in the treatment of glioblastoma multiforme, a form of brain cancer. This designation will allow the company to sell the drug without competition for seven years.

Each year, 22,070 malignant tumors in the brain or spinal cord are diagnosed, according to the American Cancer Society. About 12,920 people will die this year from malignant tumors in the brain or spinal cord, with the average life expectancy of 15 months after diagnosis.

Tau Therapeutics’ treatment, if successful, will be given to newly diagnosed brain cancer patients before they start chemotherapy. Company co-founders are hoping that one day the drug platform will be available for patients with all types of cancer.

“The platform will not only stop the cancer cell from growing, but will also enhance other medications that are considered standard care of practice for cancer patients,“ said Andrew Krouse, co-founder of the company.

Gray, a former UVa professor, and UVa chemistry professor Timothy Macdonald started work more than 15 years ago to discover how to block calcium from getting into cells. Once they discovered how calcium gets into cells they were able to find a way to block it, Macdonald said.

The men, along with Krouse, formed the company in an effort to get their discovery to cancer patients. They’ve been able to use contacts at the university to raise money and gain knowledge toward improving their company.

One of those prototypes involves using Mibefradil, an FDA-approved drug used by thousands for hypertension. The medicine, which was never given to cancer patients, was taken off the market in 1998 because of interaction problems with other medication, Macdonald said. He said it’s not uncommon to discover that a drug created for one reason is actually more helpful in some other treatment.

The treatment has been successful in prolonging the life of lab mice, but it will be a year before testing begins in humans.

Before that can happen, the company must get FDA approval to test the drug on humans.

The company has also been raising funds through private investors, many of them UVa alumni, and obtaining federal, corporate and private grants. The company will need to raise $3 million before it can test the drug on humans, Krouse said.

“We want to be the front-line therapy for cancer patients,“ said Krouse, former vice president of UVa’s Darden Graduate School of Business Administration.

The company hopes to get through three rounds of testing, which must be met before the drug can go on the market, by December 2013.

“I realized that science is fun, but it is only meaningful when you can help someone,“ Gray said. “If we can give people two more years, this is a success.“

Although the company is based in Charlottesville and the original research was conducted at UVa, most of it is now being done in other parts of the country. The company is working with scientists at Duke, Tulane, Johns Hopkins, Harvard and Kings College in London.

David Reardon, associate deputy director at the Preston Robert Tisch Brain Tumor Center at the Duke University Medical Center, said Tau’s work would fill an important void.

“There simply are not many options for malignant brain tumors, and what is available does not have a large impact on the lives of those with brain cancer,“ Reardon said. “Tau’s Interlaced Cytostatic Checkpoint Therapy has the potential to help meet a large unmet need.“

Sen. Ted Kennedy and writer Robert Novak both died recently from forms of brain cancer and both were treated at Duke University.

The founders of Tau Therapeutics are excited that their years of research are now moving forward. Each of the men has had family members who suffered from some form of cancer.

Their goal is to create a medication that cancer patients can take every day to aid their care, much like heart patients do. They are also working on other medications for cancer and diabetes.

“We already know what happens to animals, and at some point you have to give it to people,“ said Gray, whose wife was recently diagnosed as having breast cancer. “My dream is to one day be behind someone in line at the drug store and see them pick up a medication that I helped create.“