- The full findings from a case study on the first woman to be effectively “cured” of HIV have been released by researchers.
- She is currently one of only four people whose HIV reached a state of remission after receiving stem cell transplants.
- The stem cell transplants for her procedure were the first to be derived from cord blood.
- Previous procedures used stem cell transplants that came from compatible adult donors.
Researchers at the 29th annual Conference on Retroviruses and Opportunistic Infections announced in 2022 that the fourth person was effectively “cured” of HIV by way of stem cell transplants for blood cancer, in this case, acute myeloid leukemia (AML).
Now a year later, the researchers are sharing their full findings in the journal Cell.
They paint a full picture of the so-called “New York patient,” a middle-aged woman who self-identifies as mixed race and has now been living with her HIV in remission since 2017.
She follows three others (all men), who have seen their HIV effectively be “cured” as a result of stem cell transplants.
Their stem cell transplants came from compatible adult donors, while hers were derived from cord blood.
This case study underscores our growing understanding of how modern medicine is tackling an enduring global health crisis – 38.4 million people are living with HIV worldwide, according to current data from the World Health Organization (WHO).
Given that this is the first woman of color who has achieved HIV remission, experts say it also underscores why more needs to be done to include people from underrepresented communities in studies, clinical trials, and cutting-edge research.
Dr. Deborah Persaud, co-lead study author and the interim director of Pediatric Infectious Diseases, and a professor of pediatrics at Johns Hopkins University School of Medicine, said this point is especially important.
“This individual is the first woman to be in remission following stem cell transplant for blood cancer. Women represent approximately 50% of people living with HIV globally,” Persaud told Healthline. “Just as these three men provide hope for those living with HIV, so does our case, and especially for women.”
Why this case is a breakthrough in HIV treatment
Over a decade ago, Timothy Ray Brown, or “the Berlin patient” was the first person to essentially be cured of HIV by way of stem cell transplants. He would die in 2020 when his leukemia returned. Since then, he was followed by the “London patient,” Adam Castillejo, and “the Düsseldorf patient,” whose identity has not been revealed.
Last year, it was announced a 66-year-old man living with HIV and leukemia – “the City of Hope patient” – had achieved long-term remission from both diseases as a result of stem cell treatments.
What unites these different cases is the fact that they all received stem cell transplants for their cancer treatments by way of compatible adult donors. Their matched donors had to possess two copies of the CCR5-delta32 mutation. The presence of this mutation means these people don’t have the CCR5 receptor that’s needed for HIV to enter and infect a cell.
Roughly 1% of white people have this mutation and it is even rarer in non-white populations, according to a press release. As a result, it is incredibly hard to find stem cell donor matches for people of color being treated for these blood cancers.
To rectify this, the researchers used CCR5-delta32/32-carrying stem cells from umbilical cord blood to cure her cancer, and, ultimately, HIV. She received the transplant at Weill Cornell Medicine in 2017.
Persaud added that “the haplo-cord approach with HIV-resistant cord cells is feasible in adults living with HIV” and the “less stringent” matching process for cord cells makes this process “more available for racially diverse populations.”
“What all of these individuals have in common is that their cancer-treating physicians looked specifically for these HIV-resistant cells to get to cures for two diseases, with having to replace their immune system because of their blood cancers with the new one that is resistant to HIV,” she said.
Persaud stressed the significance of the fact this person identifies as mixed race. She says this work shows that being non-white and a woman “does not mean you can’t get a stem cell transplant with HIV-resistant CCR5 homozygous delta 32 cord blood cells.”
Dr. Monica Gandhi, MPH, Professor of Medicine and Associate Division Chief (Clinical Operations/ Education) of the Division of HIV, Infectious Diseases, and Global Medicine at UCSF/ San Francisco General Hospital, said these findings are really a “proof of concept” in the wake of the other cases of stem cell treatments pulling double duty of curing both blood cancers and HIV at the same time.
“This Cell report that shows that this person has gone this long with no evidence of HIV in her body, it’s really amazing, really exciting,” said Gandhi, who is unaffiliated with this research.
Could her HIV return?
When asked whether this HIV remission will persist, Gandhi said it’s significant that Timothy Ray Brown went over a decade until his passing with the resurgence of his leukemia “with no evidence of HIV rebound.”
He was completely off antiretroviral therapy (ART) for that period of time.
The other individuals whose HIV went into remission also have yet to see their HIV return to detectable levels and have also been off therapies for the virus, she added.
“Adam [Castillejo], they did everything on this poor man, rectal biopsies, looked at his cerebrospinal fluid for this virus, and couldn’t find anything, so he gives the most hope that ‘no HIV will not come back’ for these three individuals,” Gandhi said, putting in context the chances that the “New York patient’s” HIV could return to detectable levels.
Persaud said it’s been 27 months since this woman has been off antiretroviral treatments for HIV.
“The longer she goes without rebound the more likely it is that the virus won’t return in her blood to detectable viral load levels with our standard clinical assays,” she said.
Why studying underrepresented communities is essential for the development of new treatments
The fact that it is so challenging to find donor matches for patients of color when it comes to seeking these rare genetic mutations for cancer treatments, Persaud said this research can stand as an important beacon for more down the line.
“By studying understudied populations as in this case, we identified a key component of the treatment success for HIV control, as having a new immune system replaced with HIV-resistant cells, which is what all four participants have in common,” she said. “This also demonstrates racial equity in care of patients, especially [for] those living with HIV to achieve this amazing outcome of HIV remission and likely cure of two major illnesses.”
Gandhi explained it is long well-known that HIV is an epidemic that, in the United States and the world at large, has “disproportionately affected people of color” across the board.
That being said, current HIV cure trials feature “less representation of women, less representation of people of color,” Gandhi added.
She said it presents a medical and research blind spot because this lack of representation in those being studied doesn’t mean that a cure and advanced treatments can’t be possible for people of color. They can, as this latest person to achieve HIV and leukemia remission proves.
It means that these populations will be less understood as long as they are kept in the rearview of scientific research and not put equally in the center stage with other populations that are being observed.
“I urge all cure researchers to increase representation in their trials,” she said. “Rowena Johnston from amfAR has said we need to increase the representation of women in our cure trials. We’ve made some progress but definitely not enough.”
“It increases the urgency that we need representation, not just in this country [but] for the 38.4 million people living with HIV worldwide. This is an epidemic that is disproportionately affecting people in sub-Saharan Africa, for instance. We don’t want cure initiatives to only be available to just the very few, to only be available to only white men, essentially,” she added.
What this means for the future of HIV treatment
Obviously, this work applies to a narrow subset of people living with HIV. Stem cell transplants only apply to people being treated for cancer and are not something replicable in the population at large.
Still, the case of the “New York patient” offers a ray of hope for the future.
“Our growing understanding is that HIV reservoirs can be cleared sufficiently from an individual through treatment for blood cancers,” Persaud said. “Most of these cases had AML… and that control off ART can be attained with replacement with HIV-resistant immune cells.”
Gene therapy replacement approaches could be a path down the line, Persaud added.
“We are definitely not going to give people bone marrow transplants or cord transplants unless they need it for their own health, but could we do CRISPR, could we do gene therapy?” Gandhi posited. “Could we cut out the CCR5 in people living with HIV in a safer way? And that’s what’s being explored by the cure programs.”
She added the path to a cure has been rocky at best. At the end of last year, Johnson & Johnson announced it was ceasing its phase 3 trial for an HIV vaccine.
“I think that this news following on the vaccine HIV vaccine trial failure is really positive. The vaccine trial failure makes the possibility of the vaccine seem even more dim. We just have not had good luck with HIV vaccines,” Gandhi said. “This, however, should give us faith that giving more funding to a cure is still important.”
For the relatively high number of women of color living with HIV, this latest news also offers hope that underrepresented populations in medical research won’t be left out in the cold.
By thinking outside the box and finding stem cell matches for a patient whose odds were statistically lower than the men before her, the researchers found a solution that could pave the way forward for more research.
“Cure [in this form] is achievable, remission is achievable by using a more widespread strategy,” Gandhi added. “She [the ‘New York patient’] gives us hope.”
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