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European Project to guide and investigate the potential for HIV cure by Stem Cell Transplantation (EpiStem)

EpiStem is an observational project, which studies HIV infected patients who receive stem cells from another person (Allogeneic Stem Cell Transplantation). The patients all undergo this procedure because of life-threatening hematological conditions. The project aims to improve the interventions to cure these patients with the additional aim to better understand the biological mechanisms leading to reduction of viral reservoirs in the body and to identify potential cases of HIV-1 eradication/remission. EpiStem is not a clinical trial, but systematically monitors the patients before and for extensive periods of time after the stem cell transplantation.

The EpiStem investigators form a European Consortium of hematologists, infectious disease specialists, virologists, immunologists and blood/tissue bank specialists from France, Germany, Spain, the Netherlands and the United Kingdom who collaborate with clinicians globally to enroll patients to study blood and tissue samples before and after the stem cell transplantation. The project is co-led by Javier Martinez-Picado, ICREA researcher from the IrsiCaixa AIDS Research Institute (Barcelona, Spain), and Annemarie Wensing, clinical virologist from the University Medical Center Utrecht (Netherlands). Funding comes from amfAR Research Consortium on HIV Eradication (ARCHE), a program from the US Foundation for AIDS Research, amfAR.

Twenty-four individuals are being studied. Of these, 15 patients have received stem cell transplantation. A subset of these patients have received stem cells from donors who, due to a genetic variation, do not display a protein called CCR5 receptor on their cells. This protein is commonly used by HIV to enter human cells.

This kind of donor cells was also used for the stem cell transplantation of the Berlin patient, the only person to date who has been cured from HIV-infection. As part of EpiStem cord blood units and bone marrow adult donors are continuously being characterized across Europe to increase availability of donor cells lacking the CCR5 receptor.

Initial data from the EpiStem consortium show that Allogeneic Stem Cell Transplantation systematically reduces HIV-1 reservoirs independent of the procedure and type of donor used.

In three patients who are in follow-up after stem cell transplantation for more than one year, we have performed the most sensitive analyses available to date. In two of these patients we cannot detect HIV in the blood. Like in the Berlin patient we were also able to test tissue and we could only find a trace of HIV in tissues of these patients. All patients in EpiStem are still on antiretroviral therapy; therefore, we do not know whether they are cured from HIV-infection. One could only confirm cure if therapy was discontinued and no viral rebound occurred.

Currently Stem-cell transplantation is not an option that can be widely applied, since it is a high-risk procedure. However, Annemarie Wensing explained today in Durban at the international AIDS conference that studying patients who require the procedure for other conditions could provide important new insights for eradication of HIV, which currently infects about 37 million people worldwide. EpiStem aims to prospectively continue recruiting new cases of allogeneic SCT in HIV-1-infected individuals. Information on the project can be received through or  at

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New amfAR Awards Accelerate Research Towards HIV Cure

NEW YORK, Oct. 22, 2015 --- amfAR, The Foundation for AIDS Research, on Thursday announced a new round of research grants totaling more than $1.4 million. The vast majority of the funding will support cure-focused research projects.

Renewal funding of $850,000 will go to a consortium of European researchers that aims to replicate the case of the “Berlin patient,” the first and only person known to have been cured of HIV. Diagnosed with leukemia, the patient was given a stem cell transplant with a twist: The cells he received were taken from a donor with a rare genetic mutation conferring resistance to HIV infection. He remains virus-free. 

Working within the amfAR Research Consortium on HIV Eradication (ARCHE), a research program launched in 2010 to explore potential strategies for eliminating HIV, the scientists will study the outcomes of HIV patients who undergo different types of stem cell transplants. Led by Javier Martinez-Picado, Ph.D., of IrsiCaixa in Spain and Annemarie Wensing, M.D., Ph.D., of University Medical Center Utrecht in the Netherlands, the consortium has already identified a group of patients who have undergone transplants, and continues to monitor their progress in the hope of generating new knowledge that can inform more widely applicable interventions. 

“We’re very excited to continue our support of the scientists in the European consortium,” said amfAR Chief Executive Officer Kevin Robert Frost. “They have made good progress since we began supporting their work last year, and they have real potential for significantly advancing the field of HIV cure research.”  

In addition, amfAR awarded a total of $600,000 to four promising young scientists who will each receive $150,000 over two years. These Mathilde Krim Fellowships in Basic Biomedical Research, named in honor of amfAR’s Founding Chairman Dr. Mathilde Krim, are awarded annually to nurture new talent within the HIV/AIDS research field. 

Two of the Fellows will study aspects of the reservoirs of latent virus that are the main obstacle to eradicating HIV. 

Luis Agosto, Ph.D., of Boston Medical Center, will explore a mechanism that involves the covert shuttling of HIV between cells, which could be an important factor by which the virus evades the immune response and thus may maintain the viral reservoir. Liang Shan, Ph.D., of Yale University in New Haven, CT, will use a humanized mouse model to test the efficacy of latency reversing drugs, studying their ability to reactivate HIV so that the immune system can kill those cells that harbor the virus. 

Louise Scharf, Ph.D., at the California Institute of Technology in Pasadena, CA, will study the molecular structure of broadly neutralizing antibodies isolated from two HIV-infected patients to better understand how these powerful antibodies can help in the development of a vaccine against HIV. 

And Amit Sharma, Ph.D., of the Fred Hutchinson Cancer Research Center in Seattle, WA, will explore how Rhesus macaques can be better utilized as an animal model in vaccine studies. Since the macaques are not susceptible to HIV and therefore cannot be used to study HIV specific antibodies, scientists have made viruses that are part SIV (the simian version of HIV) and part HIV, called SHIVs. However, not all SHIVs replicate efficiently, which limits their usefulness in the lab. Dr. Sharma is looking into what restricts the replication of some SHIVs but not others. His findings could help accelerate the field of vaccine research. 

“The Krim Fellows are doing work that could produce major contributions to HIV/AIDS cure and vaccine research,” said amfAR Vice President and Director of Research Dr. Rowena Johnston. “Their projects are exciting and innovative, and we look forward to closely following their progress.”

About amfAR

amfAR, The Foundation for AIDS Research, is one of the world’s leading nonprofit organizations dedicated to the support of AIDS research, HIV prevention, treatment education, and the advocacy of sound AIDS-related public policy. Since 1985, amfAR has invested $415 million in its programs and has awarded grants to more than 3,300 research teams worldwide. Learn more about amfAR at

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Start of an international research consortium to reproduce the only successful case of a person cured of HIV

Funded by the amfAR Research Consortium on HIV Eradication (ARCHE)

Start of an international research consortium to reproduce the only successful case of a person cured of HIV

  • European researchers co-led by the Institute for AIDS Research IrsiCaixa, in Barcelona, and the University Medical Center Utrecht (UMCU), in the Netherlands, have initiated an ambitious project with the aim to reproduce the only case of a person cured of HIV, the Berlin patient. Experts from the Catalan Institute of Oncology (ICO),the Gregorio Marañón Hospital, in Spain, the Oxford University, in the United Kingdom, the University Medical Center Hamburg and Celle xGmbH, in Germany are collaborators in this project.
  • The Berlin patient received a stem cell transplant from a donor naturally resistant to the virus to treat him of leukemia in 2007. However, this is a high-risk procedure, only indicated for patients with life-threatening cancer disease. Doctors around the world have tried to cure other patients with similar conditions, but no other patient has been cured of HIV infection so far.

  • The international consortium, named EPISTEM, is sponsored by the amfAR Research Consortium on HIV Eradication (ARCHE), a program from the US Foundation for AIDS Research amfAR. The project aims to improve the interventions to cure these patients and to better understand the implication of stem cell transplants in the control and eradication of HIV.

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For more information you can contact
Antoinet van Kessel
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The IciStem project is supported by AmfAR Research Consortium on HIV eradication (ARCHE) Research Grant # 109858-64-RSRL and the Aidsfonds

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