PRECIOUS MEMORIES: AS AGING AMERICANS FACE ALZHEIMER'S, HIGH POINT'S VTV THERAPEUTICS RACES TO FIND A TREATMENT.

• Author: Tosczak, Mark
• Position: NC TREND: Life sciences

It's been 14 years since the Food and Drug Administration approved a new Alzheimer's drug--a long, frustrating drought for more than 5 million U.S. patients and their families. Despite the injection of hundreds of millions of dollars into research--including Microsoft founder Bill Gates' recent pledge of $100 million--there is no approved treatment to alter the relentless course of the disease.

Of some 17 drugs in the final stages of testing before the FDA considers approval, one is from vTv Therapeutics, a High Point-based company that expects to report the results of the first of two critical trials this spring. vTv is close to completing testing on the anti-Alzheimer's drug Azeliragon that it has been developing for nearly 20 years, funded in large part by billionaire investor Ronald Perelman.

With Alzheimer's and dementia care costing more than $250 billion a year in the U.S. alone, the payoff of a successful treatment is significant. So are the risks: Tested Alzheimer's therapies between 2000 and 2012 had a 99.6% failure rate, while the failure of cancer drugs in the same period was 81%.

"There's still a lot about the disease we don't understand," says James Hendrix, director of medical and scientific relations for Global Science Initiatives at the nonprofit Alzheimer's Association, a Chicago-based research and advocacy group. "That makes it challenging to really design and develop therapeutics." Scientists don't know exactly what causes Alzheimer's, but they know more than ever about the effects of the disease. Proteins accumulate in the brain and damage brain cells, interfering with memory and other cognitive functions. Though Alzheimer's isn't the only cause of dementia, it's the most common, responsible for an estimated 60% to 80% of all dementia cases.

"We have only a few treatments, and they're not very good," says Daniel Kaufer, chief of Cognitive Neurology & Memory Disorders at UNC Health Care and director of the UNC Memory Disorders Program. "The only real hope we have of turning this around is identifying an intervention early in the disease process."

By the time cognitive impairments are evident and Alzheimer's diagnosed, the disease is already several years into its course, says Kaufer, who has consulted for another North Carolina company that has pursued Alzheimer's treatments, Chapel Hill-based Zinfandel Pharmaceuticals Inc.

Zinfandel had partnered with Japan-based Takeda Pharmaceutical Co. in an $87 million deal to test...