‘New York Patient’ Appears to be Cured of HIV Following UCB Transplant

A woman appears to be cured of HIV after receiving a transplant of rare, HIV-resistant umbilical cord blood (UCB) stem cells, investigators reported last week in the journal Cell. The so-called “New York patient,” a middle-aged biracial woman treated for leukemia and HIV, has been without HIV in her blood since her transplant at Weill Cornell Medicine in 2017.

Other patients who have been cured of HIV as part of their cancer treatment – most recently the “Dusseldorf patient” – received stem cells from matching adult donors that exhibited a rare mutation in the CCR5 gene (CCR5Delta32 mutation). The mutation results in the absence of a docking site for HIV in immune cells, which protects against infection with HIV.

This mutation is found in around 1% of white people (mostly in individuals of Central and Northern European descent) and is even rarer in other populations. According to investigators, the chance of finding a compatible adult donor for the New York patient was less than 20%. Instead, the investigators transplanted CCR5-delta32/delta32-carrying stem cells from banked UCB to try to cure both her cancer and HIV simultaneously.

The treatment team transfused UCB cells with the CCR5-delta32 mutation alongside haploidentical adult stem cells from one of the patient’s relatives to increase the procedure's chance of success. The transplant successfully put both the patient’s HIV and leukemia into remission, which has now lasted more than four years. The patient stopped taking HIV antiviral medication after 37 months and has remained HIV-negative for more than 30 months.

According to investigators, the results have implications for racial health equity, demonstrating the potential to cure blood cancer and HIV via stem cell transplantation in people of all racial backgrounds. However, they noted that stem cell transplants should not be considered for curing HIV in isolation but rather for people who need a transplant for other reasons. Additionally, they emphasized that more effort needs to go into screening stem cell donors and donations for the CCR5-delta32 mutation.

“With our protocol, we identified 300 cord blood units with this mutation so that if someone with HIV needed a transplant tomorrow, they would be available,” said Yvonne Bryson, MD, of UCLA, a co-author of the study, “But something needs to be done [on] an ongoing basis to search for these mutations, and support will be needed from communities and governments.”