Results of a small human clinical trial presented at the annual meeting of the American Society of Gene and Cell Therapy in Boston showed that a single infusion of modified immune cells kept HIV under control for years, Reuters reported. CAR-T therapy, already proven effective against certain blood cancers, could become the basis for a “functional cure” for HIV.
Non-profit organisation Caring Cross, which helped develop the approach, explained that previous “cures” for HIV required stem cell transplants from donors with a rare genetic mutation and were only suitable for cancer patients. CAR-T therapy, by contrast, could be used for a much broader range of patients.
Speaking to the New York Times, Dr. Steven Deeks, professor of medicine at the University of California, San Francisco and lead author of the study, noted that this was the first such approach tested in humans. The organisation now plans to launch a larger trial in late 2026 and is collaborating with partners in Brazil, India and other countries to reduce manufacturing costs.
In the study, T-cells were extracted from patients and modified to both kill infected cells and protect healthy ones. According to method developer Boro Dropulić of Caring Cross, this dual function turned out to be the missing link. After a single infusion, patients stopped taking antiretroviral drugs. In two of the three participants who began treatment soon after infection, the virus remained undetectable or very low – for more than two years and nearly a year, respectively.
In the third patient, following an early relapse, HIV levels were low but detectable. Three patients with chronic infection did not respond to the therapy and returned to conventional treatment. Lead researcher Steven Deeks said the key finding was that all three participants with early-stage disease were controlling the infection to varying degrees.
HIV patients receiving CAR-T therapy did not experience the severe side effects – particularly cytokine release syndrome – characteristic of cancer CAR-T therapy. However, the modified cells disappeared from the blood within weeks, so the mechanism of long-term control remains unclear. “The CAR-T cells disappeared … we are trying to find a mechanism that explains this,” Dr. Deeks said.
Approximately 41 million people worldwide are living with HIV. The virus mutates easily, hides deep in the body and rebounds whenever it gets a chance. Modern antiretroviral therapy has turned HIV into a manageable condition but requires lifelong medication – daily pills or monthly injections. CAR‑T therapy could potentially provide years-long control after a single procedure.
Dropulić noted that the goal now is to make such treatments affordable. CAR-T therapy remains a complex and expensive procedure – requiring the extraction, modification and reinfusion of a patient’s cells. However, advances in modifying cells directly inside the body could significantly lower costs.
The Gates Foundation did not fund the study. However, its representative, Dr. Mike McCune, praised the trial results. He said that while only a few people participated, even such small samples stimulate further research. At the same time, he noted that CAR-T is still too expensive and invasive for widespread use, so the foundation is looking for more scalable approaches.
In 2025, the United States significantly simplified access to innovative CAR-T cell therapy. The FDA officially removed the mandatory requirement for a special Risk Evaluation and Mitigation Strategy (REMS) programme for already approved drugs of this class. The safety protocol was introduced in 2017 due to potential serious side effects, including cytokine release syndrome, neurological toxicity and the risk of secondary T-cell malignancies.
