Two local firms head to clinical trial stage for cancer treatment medications

Cancer treatment drugs developed by two Charlottesville-based biotech firms are set to begin clinical trials in a few weeks.

Diffusion Pharmaceuticals has opened enrollment for clinical trials of its product, trans sodium crocetinate, as a first-line treatment for newly diagnosed primary brain cancer.

Also this month, Tau Therapeutics announced that its investigational new drug application for mibefradil was accepted by the Food and Drug Administration.

Dr. Michael Weber is director of the University of Virginia Cancer Center. He described the work at the two companies as “the tip of the iceberg” in breaking through a divide that’s unnecessarily separated university research from private development of potentially lifesaving medicines and treatments.

“There really are two major components to this,” Weber said. “One of them is getting not only closer, but faster and shorter relationships between UVa research and the private sector, and the other aspect of it is taking our discoveries out into the community.”

Historically, Weber explained, universities didn’t venture into developing products for market and private companies stayed out of the research realm. Along the way, collaboration was often minimal or non-existent, and good ideas sometimes fell into a vacuum. But thankfully, Weber said, that’s changing.

Although the goal is the same, the two privately held firms are taking slightly different approaches to cancer treatment.

In their research, Diffusion Pharmaceuticals found that trans sodium crocetinate can enhance the effectiveness of radiation treatments for brain cancer by improving oxygen diffusion.

“We’ve done a lot of experiments on animals, and it’s clear that the effect of adding our drug, TSC, to these animal molecules … can triple the killing power of radiation,” said David Kalergis, CEO and co-founder of Diffusion. And although he acknowledged that lab animal research doesn’t always directly translate to humans, Kalergis was still optimistic.

“If we could make radiation therapy better, you could lower the dose of radiation,” he said. “You can see why we’re so excited.”

Tau Therapeutics is hoping to repurpose an existing drug, mibefradil, which is used to treat high blood pressure, to improve the effectiveness of chemotherapy drugs. The company says mibefradil has shown excellent pre-clinical results in overcoming resistance to chemotherapy drugs in brain, pancreatic, ovarian and breast cancers.

Andrew Krouse is Tau’s founder and CEO. He said the company has raised nearly $20 million and is looking to raise an addition $4 million for the project in the coming months.

Kalergis said Diffusion has invested $21 million over the past few years and is looking to raise about another $5 million to continue their work.

From a business perspective, Krouse and Kalergis said any biotech company that sees their product advance to the clinical trial stage is poised to take a major stake in the pharmaceutical market.

As an example, Krouse hopes that Tau’s product, mibefradil, will be used along with cancer treatment drug Temozolomide, which is manufactured by Merck. Temozolomide has $1.2 billion in annual sales.

“Once a company goes from pre-clinical to humans, it significantly increases the value of the organization,” Krouse said.

And although the number of brain cancer patients is small, Krouse said the overall cancer market is predicted to be $50 billion by 2015.

“If we can get our drug on the market, we believe we can get over $1 billion a year in mibefradil sales,” he added.

Researchers and staff at the UVa Cancer Center, which is a National Cancer Institute designated research center, have worked with both companies.

Weber said the center receives about $2 million in annual federal funding and about $50 million annully in grants. The cancer center has 130 faculty members and sees about 3,000 patients each year.

“If it takes 10 years to get something into practice versus three years, I think the speed with which these things happen is critically important,” said Weber. He added that the fact the work is taking place in Central Virginia is pushing the area toward a positive “critical mass” of biotech innovation.

“It certainly has been gratifying to see all of that [research] develop,” Weber said.

Kalergis agreed.

“Primary brain cancer is a nasty disease and the prognosis is very unfortunate [for those] who get it,” he said. “It’s really quite surprising that here in our small town that we have two companies working on brain cancer trials.”

This article originally appeared at The Daily Progress.