Very Personalized Medicine: Genetically Customized Bone Marrow Transplant May Have Eradicated Patient’s HIV

In a way, organ transplantation is the one branch of medicine that has already been personalized, because doctors must carefully match the immune systems of donor and recipient to prevent rejection. Now transplant physicians in Germany have taken that procedure a step further by engineering not just a successful bone marrow transplant, but one that appears to have cured their patient’s HIV infection as well. The doctor who conceived the operation has suggested that in principle, the accomplishment could inspire a gene therapy for HIV. But it will take much more research, and a lot of luck, for that to happen. This week Dr. Gero Huetter and his colleagues at the Charite Hospital in Berlin announced that they were treating a 42-year-old patient who required a bone marrow transplant for leukemia, but had also been HIV-positive for a decade. The doctors knew of a gene, CCR5, that confers resistance to the most common form of HIV. People who have a particular mutation, known as Delta32, on both copies of CCR5 can be exposed repeatedly to HIV-1 without becoming infected. The doctors reasoned that if they could find a donor who was not only immune compatible with their patient, but also had two copies of the Delta32 mutation in the CCR5 gene, perhaps they could simultaneously eradicate his leukemia and his HIV infection. Remarkably, such a donor existed. And 600 days after his bone marrow transplant, the patient is both leukemia- and HIV-free. Is this a cure for AIDS? Not a chance. Doctors do not consider the procedure a potential treatment for HIV, because bone marrow transplants are expensive and risky – about one patient in four does not survive the procedure. But the case does provide inspiration and hope for researchers who are working on ways to harness the resistance conferred by CCR5Delta32 to treat the infection.
  • This is cool.

    I am writing a story where the main character basically gets an immune system reboot. Due to the longevity of the people in the story, they are more susceptible to auto-immune diseases as the immune system breaks down or goes awry. Therefore, folks get regular immune system resets and required re-immunizations.

    This gene therapy route is cool too.

  • This almost sounds too good to be true! I wonder if the HIV is completely cleared from his system, or if there is a chance that it will come back. At any rate, this is amazing.

  • Matthew

    A recent news article (after 2 years of tracking) reported he still had no sign of HIV in his system, so they’re assuming he’s still completely free of the virus. Addtionally, given the immune system “reboot” of the CCR5 genes, I wonder if his susceptibility to ever being reinfected is much lower?

    Pretty amazing stuff…. not a practical treatment, but in the meantime the medication developments have already been amazing based on this.

  • One more comment – has 23andme ever considered facilitating those found to be homozygous with the CCR5-delta32 variant getting registered as bone marrow donors? Nothing mandatory, of course, but the impact you could have on an HIV+ person in need of a transplant as a bone marrow donor is pretty profound!

  • Jewel

    I am delta32 ccr5 (homogeneous) and cannot seem to find anyone doing these transplants. Only drug research, but meanwhile at least someone’s prayer can be answered, one at a time while we wait for the drug companies…

    My husband died of HIV in 1994… When I never became infected I searched out genetic testing and found I was indeed a mutant. Wish I could have saved my sweet Mark, but would love to offer hope to another.

    Any insights?