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HIV Gene Therapy: A Second Time for Celebration

Yamna Rizwan

posted:March 25th, 2019

Earlier this month, news broke of the second patient ever to be free of HIV infection. The
unnamed patient had been receiving stem-cell transplants in which donor HIV−resistant stem
cells took the place of their own white blood cells. Eighteen months after stopping the use of
antiretroviral drugs−a common treatment for HIV−the patient remains free of any signs of the
virus (Warren, 2019). The therapy itself is elegant in its simplicity: donor transplant of cells
containing a homozygous mutation in the CCR5 gene, which, without the mutation in both
copies, codes for the receptor upon which HIV binds (Gupta et al., 2019).

What remains to be seen is if this is a temporary remission of the virus lying dormant or a
permanent cure. The current “London Patient” follows ten years after the “Berlin Patient”,
Timothy Ray Brown. Both were given chemotherapy and a cancer-specific drug along with the
bone marrow transplants for Hodgkin’s lymphoma and leukemia, respectively, but had received
donor cells with the CCR5 mutation, treating their HIV in the process. Brown remains virus-free
but suffered serious side effects from an additional rigorous radiotherapy procedure for treating
his cancer (Warren, 2019).

At first glance, this seems to be the long-awaited cure for HIV. But a deeper look shows that the
bone marrow transplant and its side effects, as well as the strong drugs required to suppress the
immune system after the procedure, will prevent this from becoming a widely available first-
point cure. What the scientific community and society at large can take away from this
extraordinary finding−apart from further supporting a treatment option for a small group of HIV-
positive patients who require bone marrow transplants for other reasons−is it being a powerful
stepping stone for gene therapy research that may one day find an extensive, final cure (Warren,

Gupta, R. K., Abdul-Jawad, S., McCoy, L. E., Mok, H. P., Peppa, D., Salgado, M., … Olavarria,
E. (2019). HIV-1 remission following CCR5Δ32/Δ32 haematopoietic stem-cell
transplantation. Nature. https://doi.org/10.1038/s41586-019-1027-4
Warren, M. (2019, March 5). “Second patient free of HIV after stem-cell therapy” Nature.
Retrieved from https://www.nature.com/articles/d41586-019-00798-3