WDOM Physician-Scientists Play Key Role in Possibly Curing HIV Infection by Special Stem-Cell Transplant


WDOM physician-scientists Drs. Koen Van Besien, Jing-Mei Hsu, and Marshall Glesby have led a clinical trial involving a patient living with HIV who has now been free of the virus for 14 months.

The patient, who was given a blood stem cell transplant for high-risk acute myeloid leukemia, received stem cells from a healthy adult relative (used to quickly restore her blood cell population to reduce infectious complications) and umbilical cord blood from an unrelated newborn. The umbilical cord blood served in providing long-term blood reconstitution and contained an HIV-resistance gene variant. There have been two other successful cases reported in which transplanted donor cells were used that contained an HIV infection resistant mutation, but they did not involve the use of cord blood.

Drs. Van Besien, Hsu, and Glesby ran tests on their patient, who is the first woman to receive cord blood cells and who identifies as mixed-race, at the three-month mark post-transplant. At that point, the patient’s blood cell population was entirely derived from the HIV-resistant cord blood cells, and eventually, she was able to stop taking antiretroviral drugs. To date, there have been no signs of HIV re-emergence and she has been leukemia-free for more than four years.

The investigators have concluded that cord blood containing the HIV resistant variant offers a possible cure for both hematological malignancies and HIV. However, it is considered too risky for HIV-positive patients who otherwise would not need such a transplant.

Dr. Van Besien and Dr. Hsu are stem cell transplant specialists in the WDOM’s Division of Hematology and Medical Oncology. Dr. Glesby is an infectious diseases specialist in the WDOM’s Division of Infectious Diseases.

Funded by the NIH, this clinical trial was conducted at Weill Cornell Medicine in collaboration with investigators at University of California Los Angeles, Johns Hopkins University School of Medicine, and several other institutions.

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Drs. Van Besien, Hsu, and Glesby

L to R: Dr. Koen Van Besien, Dr. Jing-Mei Hsu, Dr. Marshall Glesby.
Photo credit: Benjamin Ryan/NBC News