HIV, the AIDS virus (yellow), infecting a human cell. Photo by National Cancer Institute on Unsplash
HIV, the AIDS virus (yellow), infecting a human cell. Photo by National Cancer Institute on Unsplash

EXPERT REACTION: Stem cell transplant sends another HIV patient into remission

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Peer-reviewed: This work was reviewed and scrutinised by relevant independent experts.

Case study: A study involving observations of a single patient or group of patients.

People: This is a study based on research using people.

A patient who was treated for leukaemia with stem cell transplantation has also shown nine years of persistent suppression of HIV, according to international researchers, who say this patient could follow the famous ‘London patient’ and ‘Berlin patient’ who underwent similar treatments.  The team describe the 53-year-old male with HIV who was diagnosed with acute myeloid leukaemia in January 2011. During his stem cell transplant for the cancer, he received bone marrow from a donor with a mutation in the gene for the HIV-1 receptor CCR5, which makes cells resistant to HIV infection. HIV became undetectable in the patient's blood cells, so antiretroviral therapy was stopped in November 2018 to see if the infection would return, but the patient remained in remission for another four years. While stem cell therapy is a high-risk procedure, the team says this case study adds to previous work that could one day allow HIV treatment and remission.

Journal/conference: Nature Medicine

Link to research (DOI): 10.1038/s41591-023-02213-x

Organisation/s: Düsseldorf University Hospital, Germany

Funder: The IciStem program (www.icistem.org) is funded through the amfAR Research Consortium on HIV Eradication program (amfAR 109858- 64-RSRL) and the Dutch Aidsfonds (P60802). University Hospital Düsseldorf was supported by the Heinz Ansmann Foundation for AIDS Research. The German Center for Infection Research (DZIF TTU 04.816, 04.819) supported University Hospital Cologne and the University Medical Center Hamburg-Eppendorf. J.S.z.W. was supported by the European Research Council (ERC H2020 grant no. 681032), the H.W. & J. Hector Foundation (project no. M2101), the German Research Foundation (SFB1328 project A12) and Hamburg Investment and Development Bank (IFB Hamburg, PROFI no. 51167035). F.P.-C. was supported by Institut Pasteur’s Roux Cantarini program. T.H. and C. Mummert were supported by the German Research Foundation, Graduate School 1071, project B1. M.S. was given a grant by the Miguel Servet Fellowship (CP22/00038) and I+D+I RTI-A project (PID2020-115931RA-I00) from the Ministry of Science and Innovation. Research in the Martínez-Picado laboratory is supported by the Spanish Ministry of Science and Innovation (grant nos. PID2019- 109870RB-I00 and CB21/13/00063), NIH/NIAID (1 UM1 AI164561-01), European Union HORIZON-HLTH-2021-DISEASE-04-07 (grant no. 101057100) and the Research Centers of Catalonia (grant no. 2017 SGR 252). C.G. was supported by the PhD fellowship of the Spanish Ministry of Education, Culture and Sport (FPU15/03698). J.D.E. and the Oregon National Primate Research Center were supported by the NIH/NIAID (grant no. R01AI143411) and the NIH/Office of the Director (grant no. P51OD011092), respectively. The funders had no role in the conceptualization, design, data collection, analysis, decision to publish or preparation of the paper. The authors thank IciStem no. 019 and all volunteers who participated in this study; K. Pohl, E. Bäcker, O. Adams, T. Feldt, F. Hüttig and N. Freise of University Hospital Düsseldorf for their assistance with patient care and/or sample logistics; S. Kummer and R. Woost of the University Medical Center Hamburg-Eppendorf for technical assistance with sample preparation and flow cytometry; M. Angin of Institut Pasteur for initial assistance with flow cytometry analyses; G. Hütter of Cellex, J. L. Diez Martin of Hospital Gregorio Marañón Madrid, J. Kuball, D. de Jong and M. van Luin of the University Medical Center Utrecht for contributing their expertise to the discussion; M. A. Fernandez Sanmartin, Flow Cytometry Service of the Institut de Recerca Germans Trias i Pujol for his contribution to the cytometry panel discussions and sample sorting; Á. Hernandez Rodrıguez, V. González Soler and B. Rivaya Sanchez of the Microbiology Department of the Hospital Universitari Germans Trias i Pujol for their assistance with the murine outgrowth assay and HIV antibodies; J. Diaz Pedroza and Y. Rosales of the Comparative Medicine and Bioimage Centre of Catalonia for their contribution to the development of the mVOA experiments; M. del Carmen García and I. González of the AIDS Research Institute IrsiCaixa for their assistance with the HIV-DNA ddPCR; J. Blankson of the Johns Hopkins University for his contribution to the development of the mVOA protocol; and the Oregon Health & Science University Knight BioLibrary for the lymph node sample.

Media release

From: Springer Nature

Medicine: Sustained HIV-1 remission after stem cell transplantation

A recipient of an allogeneic hematopoietic stem cell transplant to treat leukemia has shown persistent suppression of human immunodeficiency virus type 1 (HIV-1) more than 9 years after the transplantation and 4 years after the suspension of anti-retroviral therapy. The results are reported in Nature Medicine this week.

Allogeneic hematopoietic stem cell transplantation (HSCT) is a procedure used to treat certain cancers, such as leukemia, by transferring immature blood cells from a donor to repopulate the bone marrow of the recipient. CCR5Δ32/Δ32 HSCT involves the transfer of cells from a donor with two copies of the Δ32 mutation in the gene encoding the HIV-1 co-receptor CCR5; such cells are resistant to HIV-1 infection. So far there have been two published cases of patients experiencing remission of HIV-1 after cancer treatment involving the transplantation of CR5Δ32/Δ32 hematopoietic stem cells: the ‘London patient’ and the ‘Berlin patient’.

Björn-Erik Jensen and colleagues now present a detailed longitudinal analysis of blood and tissue samples from a patient demonstrating remission of both leukemia and of detectable HIV-1 after transplantation of CR5Δ32/Δ32 hematopoietic stem cells. The patient, a 53-year-old male, was diagnosed as having acute myeloid leukemia in January 2011. The patient underwent transplantation of CR5Δ32/Δ32 stem cells from a female donor in February 2013, followed by chemotherapy and infusions of donor lymphocytes. After the transplantation, anti-retroviral therapy was continued, but pro-viral HIV-1 was undetectable in the patient’s blood cells. Anti-retroviral therapy was suspended in November 2018 with the patient’s informed consent, almost 6 years after the stem cell transplantation, to determine whether infectious HIV-1 persisted in the patient. The authors did not observe a resurgence of HIV-1 RNA or a boosting of the immune response to HIV-1 proteins that might have suggested a lingering, yet undetectable, viral reservoir 4 years after the suspension of anti-retroviral treatment.

The authors conclude that although HSCT remains a high-risk procedure that is at present an option only for some people living with both HIV-1 and hematological cancers, these results may inform future strategies for achieving long-term remission of HIV-1.

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Expert Reaction

These comments have been collated by the Science Media Centre to provide a variety of expert perspectives on this issue. Feel free to use these quotes in your stories. Views expressed are the personal opinions of the experts named. They do not represent the views of the SMC or any other organisation unless specifically stated.

Dr Ioannis Jason Limnios is from the Clem Jones Centre for Regenerative Medicine at Bond University

During the pandemic, the public learned that viruses use special "door handles" called receptors to get into our cells. Just as COVID-19 uses the ACE-2 receptor to infect cells of the lungs and other tissues, HIV uses a different receptor called CCR5 to infect cells of the immune system. What happens if the door handle is missing? For about 30 years it has been known that some individuals are resistant to HIV infection because they lack CCR5 receptors on their cells; HIV can’t open the door and get inside. 

This study shows that transplanting blood stem cells from an HIV-resistant donor has led to the development of a new, HIV-resistant immune system in an HIV+ patient. By following the patient for a decade after transplantation, researchers have shown that their HIV resistant immune system is stable and working well, and that the patient remains healthy after stopping antiviral therapy for 4-years.

Over the past 10 years, stem cell and gene editing technologies (such as CRISPR) have advanced medical science to a point where we can now engineer stem cells for such therapies. Rather than harvesting stem cells from donors with rare and special genetics, they can be created in specialised facilities under highly controlled conditions, and in greater quantities.

This is important and exciting progress in the fight against AIDS, however the researchers carefully state that HIV remains hidden in other tissues of the body. So, it’s not yet clear if type of therapy is a life-long “cure,” and the risk of passing on HIV, whilst extremely low, will never be zero using this therapy alone.

Last updated: 20 Feb 2023 11:51am
Declared conflicts of interest:
Jason declares that he has no conflicts of interest.

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