The National Institute on Drug Abuse (NIDA), a branch of the National Institutes of Health (NIH), recently granted the Avant-Garde Award for HIV/AIDS and Drug Use Research to Leor Weinberger, PhD, senior investigator, William and Ute Bowes Distinguished Professor, and director of the Center for Cell Circuitry at Gladstone Institutes. This award supports highly innovative research that could transform prevention and treatment of HIV/AIDS among drug abusers.
Weinberger was selected for proposing to develop a novel type of “gene drive” for HIV, a technology that involves engineering genes to increase the likelihood that they will be transmitted to offspring. He and his team developed molecules called therapeutic interfering particles, or TIPs, that act like gene drives. Using the virus’s own tricks against it, these particles hijack infected cells and make them produce a therapy instead of replicating the virus.
For HIV/AIDS, people who inject drugs are considered a high-risk group and can be “super-spreaders” of the disease. A treatment that targets this group would have immense potential to contain outbreaks and reduce incidence of HIV. However, it is usually costly and difficult to reach this population with disease-control measures.
Weinberger’s unique strategy could offer a much-needed solution. Once a person is treated with TIPs, if the virus is spread to another person, the TIPs would be passed along with it, essentially providing the antidote with the infection.
What’s more, the treatment would only have to be administered once, as opposed to traditional antiretroviral therapies that must be taken every day to keep the virus dormant. This single administration is a huge advantage for resource-limited areas, where deployment of and adherence to antiretroviral therapy is extremely challenging.
“We sincerely congratulate Leor for receiving this coveted award,” says Gladstone President Deepak Srivastava, MD. “The therapy he is developing can reach the highest-risk populations that most need treatment, which is a fundamental barrier for current therapies and vaccines. As a result, it could help greatly reduce HIV transmission.”
The NIDA Avant-Garde Award Program supports individual scientists of exceptional creativity who propose cutting-edge approaches to major challenges in biomedical and behavioral research on HIV/AIDS that are relevant to drug abuse, and could lead to new avenues for prevention and treatment of the disease.
Weinberger has already shown that the TIP approach can work in humanized animal models. With this award, he intends to test the safety and efficacy of a prototype gene-drive hijacker therapy in patient cells to establish feasibility, as well as test tolerability in a Phase-I clinical trial.
In addition to Weinberger, three other Gladstone virologists are past Avant-Garde awardees. Melanie Ott, MD, PhD, director of the Gladstone Institute of Virology, received the award in 2014; Warner Greene, MD, PhD, director of the Center for HIV Cure Research at Gladstone, was a recipient in 2013; and former Gladstone Investigator Eric Verdin, MD, who is now president and CEO of the Buck Institute, was a 2010 awardee.
The Avant-Garde Award belongs to the same family of high-risk, high-reward NIH flagship awards as the NIH Director’s Pioneer Award and the NIH Director’s New Innovator Award. Weinberger is also a past recipient of the Pioneer (2013) and New Innovator (2008) Awards. To date, he is the only individual to win all three of these research awards.