Stem Cells and the Hope for an HIV Cure

The growing list of patients cured

For decades, HIV has been managed with antiretroviral therapy, but never truly cured. Now, a series of stem cell transplant cases has begun to rewrite what is possible. A total of ten individuals are considered cured of HIV so far, with several more in long-term remission. These patients often underwent transplants as part of cancer treatment, and the unexpected result was freedom from HIV.

Among those who have stepped forward publicly are Adam Castillejo, known as the London patient, Paul Edmonds, the City of Hope patient, and Marc Franke, the Düsseldorf patient. They are now collaborating with researchers at Oregon Health and Science University (OHSU) to help scientists learn how their cures were achieved.

The OHSU project

The National Institutes of Health has awarded OHSU an $8.4 million grant to study the immune responses of these individuals. Jonah Sacha, a leading investigator, has emphasized that stem cell transplants are the only documented medical procedure linked to HIV cures. By analyzing blood and tissue samples from the cured patients, the team hopes to identify mechanisms that could eventually be applied in less risky ways.

Sacha explained, “For the first time, multiple individuals cured of HIV have released their identities and are willing to collaborate with scientists. This is an unprecedented and exciting time in HIV cure as we can reverse engineer a stem cell transplant-mediated cure.”

How stem cells are used

Stem cell transplants typically replace a diseased immune system with one from a healthy donor. In the case of HIV, donors with mutations in the CCR5 gene have played a key role. The CCR5 receptor allows HIV to enter cells, but individuals with the CCR5-delta-32 mutation are resistant to infection.

The first known cure was Timothy Ray Brown, the Berlin patient, who received a transplant from a donor with the double CCR5-delta-32 mutation while being treated for leukemia. Years later, no trace of HIV remained in his body. Since then, others have achieved remission or cures through variations of the same approach, some with donors carrying the mutation and others without.

Recent patients and their progress

At the 2025 Conference on Retroviruses and Opportunistic Infections, two more cases were revealed. The Chicago patient, a 67-year-old man, experienced viral rebound after his first attempt to stop medication but later entered lasting remission after a second interruption. The Oslo patient, a 58-year-old man, underwent a transplant from his brother with a CCR5 mutation and has remained virus-free two years after stopping treatment.

Earlier, a woman in France, known as the French patient, also achieved an apparent cure following a stem cell transplant. Monitoring revealed no HIV DNA or RNA in her blood, and she has lived for more than a year without treatment while maintaining an undetectable viral load.

What researchers are learning

These cases do not all follow the same pattern. Some patients had donors with double CCR5 mutations, some with single mutations, and some with none at all. Some endured severe graft-versus-host disease while others did not. Researchers now believe the answer may lie in a combination of factors including the size of the viral reservoir, immune system changes, and perhaps even the drugs used to manage complications.

David Cennimo, an infectious disease specialist at Rutgers, pointed out that the latent viral reservoir remains the greatest obstacle. HIV hides within DNA, making it almost impossible to remove. Stem cell transplantation might alter this reservoir in ways that help, but it remains uncertain. Cennimo said, “I hope the researchers deduce what is actually happening at the interaction of the immune system and virus, and if so, whether these mechanisms could be triggered in other less intensive ways to result in cure.”

Beyond traditional transplants

Because stem cell transplantation is risky, researchers are experimenting with gene editing. Teams at the University of California San Francisco and elsewhere are using CRISPR and viral vectors to engineer stem cells with multiple forms of HIV resistance. These efforts aim to make a scalable treatment that avoids the dangers of donor transplants.

One study modified patients’ own stem cells with three HIV-resistance genes, which persisted long-term in their immune systems. While not yet a cure, the findings suggest a path toward protecting cells against HIV in ways that could eventually be used more broadly.

The potential of stem cell research

Although stem cell transplants cannot be offered to the millions living with HIV today, every case of cure adds to the map of how HIV might one day be eliminated. Andrea Gramatica of amfAR summarized the situation: “We know that stem cell transplants are not a viable means of curing most people living with HIV, but each new case of a cure provides clues for developing more scalable interventions as well as avenues for further research.”

The ultimate goal is not just isolated cases, but a therapy that can be used widely without the risks of cancer treatment-level procedures. With patients willing to share their experiences, and with new tools like gene modification, the possibility of a true HIV cure may be closer than ever.