Big Bolus for HIV/AIDS Cure Research

Three collaborations will receive up to $70 million over the next 5 years to advance the search for an HIV/AIDS cure, the U.S. National Institutes Health (NIH) announced today. This is the largest single investment yet made into finding a way to rid the virus from the body or at least reduce levels to the point that infected people can stop taking anti-HIV drugs—which many researchers until recently viewed as a hopeless quest.

The three grant recipients of what's known as the Martin Delaney Collaboratory include teams organized by the University of North Carolina (UNC), Chapel Hill, the Fred Hutchinson Cancer Research Center (FHCRC) in Seattle, and the University of California, San Francisco (UCSF), working with the Vaccine & Gene Therapy Institute of Florida (VGTI) in Port St. Lucie, Florida. "The three collaboratories are using very different but largely complementary approaches," says UCSF's Steven Deeks, one of the principal investigators for that project. "Since many of us believe a cure will require combination therapy, it is my hope—as well as the hope of others—that three groups can merge their work whenever possible."

The best-funded and largest group, led by UNC's David Margolis, will receive $6.3 million per year for 15 different projects. The researchers will both conduct basic research and attempt to develop small molecule drugs that can reduce the reservoir of cells infected with latent HIV that stubbornly persist even in people who receive the best antiretroviral treatments available. The 19 collaborators Margolis leads come from nine universities across the country and the Merck Research Laboratories. "We're very excited to try and approach this important and complicated problem as a group," says Margolis

The other two collaborations will each receive a shade over $4 million per year. The UCSF and VGTI project, which is also with Merck, plans to use immune-based treatments in addition to small molecules to shrink reservoirs. Headed by VGTI's Rafick-Pierre Sékaly and UCSF's Deeks and Mike McCune, the project includes academic collaborators in Australia and Sweden.

The FHCRC project, led by Keith Jerome and Hans-Peter Kiem, involves two distinct but potentially complimentary approaches. One partners with California's Sangamo Biosciences and the City of Hope to create a bone marrow transplant that mimics the treatment given to Timothy Brown, aka the "Berlin patient" -- the first and only person who apparently has been cured of HIV/AIDS. (The case indeed helped catalyze the new interest in cure research, as this Science article details.) Specifically, they will engineer stem cells to cripple a key receptor the virus uses to infect cells and then transplant those cells into monkeys and, eventually, humans. The second strategy aims to deliver an enzyme called an endonuclease that specifically clips HIV DNA lurking in chromosomes.

Funding for the Martin Delaney Collaboratory comes primarily from the National Institute of Allergy and Infectious Diseases (NIAID), with a small contribution from the National Institute of Mental Health. Initially, the two institutes committed just $42.5 million to the collaborations and said that, at most, two would receive funding. NIAID Director Anthony Fauci explains that they scoured their budget to find what now totals $70 million for three groups because there was so much interest in cure research. "We asked our budget people if, without damaging other programs, can you scrape up a little bit here and a little bit there?" says Fauci. "At the end of the day, we came up with significant cash. We need to get people energized in this and show that we're putting the money up."