Woman appears cured of HIV after umbilical-cord blood transplant

A woman in the New York City area appears to have been cured of an HIV infection, joining a small group of people whose recovery is providing researchers with road maps to beat one of nature’s most resilient viruses.

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The woman has shown no detectable signs of the human immunodeficiency virus in extensive testing since she stopped antiretroviral treatment in October 2020 following a transplant of stem cells with a rare genetic mutation that blocks HIV invasion, her doctors said.

The doctors said they consider her HIV to be in long-term remission, suggesting a cure if it holds. That would mean she has no virus able to replicate in her body, unlike people who have HIV but stay healthy by keeping the virus at low levels with long-term drug treatment.

"Everything is looking very promising," said Marshall Glesby, associate chief of the division of infectious diseases at Weill Cornell Medicine and New York-Presbyterian, who is treating the woman. The details of her case were presented Tuesday at the Conference on Retroviruses and Opportunistic Infections, which is being held virtually.

The woman, who had a form of leukemia, received a transplant of stem cells from an adult relative and an umbilical-cord blood from a newborn to whom she wasn’t related. The treatment is likely to be suitable for a broader range of people than are the transplants that beat HIV back in three other patients, because cord blood doesn’t have to be a precise genetic match to a recipient patient, Dr. Glesby and other researchers said.

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Three other cancer patients have achieved HIV remission following stem-cell transplants, which like this woman’s contained stem cells with a key mutation to a gene called CCR5. Most HIV strains use normal copies of this gene to penetrate a patient’s immune-system cells. The mutation renders the gene inactive, thereby blocking HIV entry.

Transplants, which are risky and costly, are unlikely to be an option for HIV patients other than those who need them for treatment for diseases like cancer, said Sharon Lewin, director of the Peter Doherty Institute for Infection and Immunity in Melbourne, Australia, and a leader in HIV cure research who wasn’t involved in the woman’s case. It’s also not known how many HIV patients have failed to go into remission following similar transplants, Dr. Lewin said.

Researchers aspire to develop a cure that could be given not just to people in advanced medical centers in the developed world, but in sub-Saharan Africa, home to two-thirds of the world’s 37.7 million people who are living with HIV, the virus that causes AIDS.

Still, the details of the New York patient’s medical journey could help researchers who are developing gene therapies to confer the HIV-resistant mutation to patients, she and other researchers said.

Steven Deeks of the University of California, San Francisco, another longtime leader in the search for an HIV cure who wasn’t involved in the case, called the woman’s treatment "a road map for doing this using a person’s own cells, which would be far safer and more scalable."

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The woman, who is middle-aged and of mixed race, was treated by doctors at Weill Cornell Medicine and New-York Presbyterian as part of a study funded by the National Institutes of Health. She was given a transplant to treat acute myeloid leukemia, a cancer that starts in blood-forming cells of the bone marrow, as well as HIV, which she was diagnosed with four years before developing her leukemia.

Described by her doctors as a private person, she declined to comment or allow them to disclose her identity or exact age.

The first person deemed to be cured of HIV was Timothy Ray Brown, initially called the "Berlin patient," whose success inspired researchers to explore gene therapies that could cure the disease. He died in 2020 following a relapse of the leukemia that led to his transplants. In 2019, researchers reported that two other patients, men in London and Düsseldorf, had achieved sustained HIV remission after receiving transplants and stopping HIV medications.

Researchers have also discovered two women who were effectively cured of HIV by their own immune systems, which suppressed the virus without medication.

The New York patient received her transplant in August 2017. Her doctors wanted to try to cure her HIV as well as her leukemia, but the donors whom she matched with didn’t have the rare CCR5 mutation, which is normally found in Causacians in Northern Europe.

So they offered her a transplant of stem cells in umbilical-cord blood, which don’t require a perfect genetic match, said Koen Van Besien, stem-cell transplant specialist at Weill Cornell Medicine and New York-Presbyterian who designed the procedure. "Cord blood grafts are much more permissive," he said. The cord blood they used had the HIV-resistant mutation.

"She really liked the idea," said Jingmei Hsu, a stem-cell transplant specialist at Weill Cornell Medicine and New York-Presbyterian, who also treated her.

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Cord-blood transplants take up to 30 days to graft, Dr. Van Besien said. The doctors sped up the process by supplementing it with a transplant of stem cells from one of the woman’s adult relatives, which graft more quickly. Within 100 days, the patient’s blood cells were totally derived from the HIV-resistant cord-blood cells, the doctors said.

The patient stopped taking HIV medication in early April 2020, but went back on it 10 days later when her doctors decided they couldn’t safely monitor her progress in the huge Covid-19 pandemic wave that had hit New York City. She went off the medication for good later that year. She has also been free of leukemia for more than four years.

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