Gene Therapy as Possible Cure for HIV/AIDS

American Gene Technologies (AGT) has submitted an application to begin Phase 1 clinical trials with the FDA for gene therapy that might be able to cure HIV in patients who are already HIV-positive.

According to an article by NewNowNext, AGT’s drug, called AGT103-T, is a “single-dose vector-based gene therapy” that might be able to eliminate infected cells and disrupt HIV-positive patients’ need to take lifelong antiretrovirals.

AGT, based in Rockville, Maryland, submitted an Investigative New Drug application and if it is approved, the company would begin trials of the drug.

Antiretrovirals were a breakthrough in treatment, but if use isn’t maintained, the virus returns within just a few weeks. Chief Science Office C. David Pauza said that the goal is “to treat HIV disease with an innovative cell and gene therapy that reconstitutes immunity to HIV and will control virus growth in the absence of antiretroviral drugs.”

However, some doctors expressed concern about the idea of treating HIV with bone marrow transplants.

“A bone marrow transplant is an extraordinarily toxic and life-threatening intervention which you do if someone has an illness that’s clearly going to be fatal,” Kenneth Freedberg, MD, a professor of medicine at Harvard Medical School and Massachusetts General Hospital, told NewNow Next. “There must be no other treatment options available. It puts people at massive risk for infections and toxicity complications.”

So far, there have been three cases of patients being cured of HIV when they received bone marrow transplants for unrelated instances of cancer. However, bone marrow transplants are an untenable solution. The procedure is risky, as it makes patients susceptible to many serious, possibly fatal, and long-lasting complications.

Until trials are approved and the drug is available for wide use, the search for a cure continues. Hopefully, solutions emerge with more research and funding. As of now, antiretrovirals and PrEP are tools for people navigating their health and protection against HIV.