New amfAR Grants Advance HIV Cure and Post-Treatment Studies

In August 2019, through the amfAR Research Consortium on HIV Eradication (ARCHE), a grant program that fosters collaboration among teams of scientists, amfAR has awarded new grants totaling $1.16 million to advance a pair of innovative research studies attacking HIV from very different angles.   

Keith Jerome, Ph.D., of the University of Washington, Seattle, was awarded $344,000 for a project that aims to advance a gene therapy strategy for curing HIV. Gene therapy is emerging as one of the most promising interventions across all of biomedical science, including HIV, but it carries a number of risks and challenges. Scientists need to find ways to improve the efficiency of appropriately altering DNA, effectively target the correct cells, and enable the therapy to safely persist long enough to have an effect.

Moreover, a substantial limitation of current approaches is their cost in the clinic, which can be as much as $2 million or more. amfAR’s ARCHE-GT consortium plans to reduce costs by developing in vivo gene therapy in which the gene-engineering tools are injected directly into the patient. These tools are delivered inside vectors, which function as “Trojan horses.”

Dr. Jerome’s team plans to compare which of 11 vectors delivers the gene-editing tools most effectively to the various specific tissues that are being targeted. These lead candidates will then be used in future studies of combination in vivo gene therapy interventions.

Another area of HIV research in which amfAR is particularly interested is post-treatment control. Post-treatment controllers (PTCs) are HIV-positive individuals who, unlike the majority of people living with HIV, are able to control their virus after stopping antiretroviral therapy (ART). But discovering the mechanisms of this control has proven difficult since the rarity of PTCs has so far precluded analysis of a sufficient number of samples.

amfAR’s ARCHE-PTC collaboration consists of the world’s leading experts in PTC research and brings together clinical cohorts of PTCs from all over the world, including an all-female cohort from Cameroon, under one streamlined analysis plan. Dr. Jonathan Li of Brigham and Women’s Hospital in Boston has assembled an impressive cohort of post-treatment controllers from one of the largest HIV clinical trial networks in the world.

Supported by an amfAR grant of $815,000, Dr Li and his team will gather and analyze samples from this multinational PTC cohort and will be able to employ cutting-edge tools to investigate whether characteristics of the virus or immunologic responses can predict post-treatment control. Discovering what leads to post-treatment control in some people could help to achieve durable ART-free control in all people living with HIV.

“We’re excited to be supporting these immensely talented research teams and their very different but very promising avenues of investigation,” said Dr. Rowena Johnston, amfAR vice president and director of research. “These research areas have enormous potential for giving us the tools to control the virus without the need for lifelong treatment or, in the case of gene therapy, to eliminate it altogether. Either outcome could dramatically alter the lives of the millions of people living with HIV worldwide.”

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