University of Rochester Scientists Receive $5 Million Grant to Test Off-Label Drugs’ Effectiveness in Treating HIV-Related Dementia
The National Institute of Mental Health and the National Institute of Allergy and Infectious Disease have awarded to a team of researchers at the University of Rochester a four-year, $5 million grant to study the efficacy of drugs approved for treating other diseases in alleviating HIV-related dementia, the Rochester Democrat and Chronicle reports. The researchers will study the effectiveness of the drugs, used "off-label," in an effort to speed treatment of HIV-related dementia, a condition characterized by memory loss, confusion, changes in behavior and decline in motor skills. Dementia, for which there is no known effective treatment, is believed to occur because HIV kills nerve cells in the brain and central nervous system. Less than 10% of those on antiretroviral medications develop the condition, but about 95% of people with HIV worldwide do not have access to such drugs. The Rochester group will test "at least" four drugs, including an epilepsy drug called sodium valproate. The compound is believed to inhibit the activity of an enzyme that takes part in communication between brain cells, which appears to "trigger" a process that can lead to the destruction of brain cells in people with HIV. The drugs will first be tested on mice with a specific type of HIV infection to determine which drugs may be effective on humans. About 1,500 HIV-positive people already receiving treatment in the Rochester area may then be able to participate in human trials in which researchers will monitor the drugs' effects on cognitive behavior, motor skills and brain inflammation. "This very streamlined model for developing new and established drugs could be a stepping stone for something much bigger," lead researcher Dr. Harris Gelbard, a University of Rochester professor of neurology, said, adding that the project is "not beholden to any company or any drug. We can screen a wide variety of agents and approaches and may the best agent win." Drug companies are providing the drugs that will be evaluated. By testing drugs that have already received FDA approval for treating other conditions, the "expensive and time-consuming requirements of long-term testing for safety" will be shortened, the Democrat and Chronicle reports (Wentzel, Rochester Democrat and Chronicle, 10/23).This is part of the KHN Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.