HIV Stem Cell Breakthrough Could Have Enormous Impact

A woman from New York City has reportedly been cured of HIV. This is the first time that someone has been cured of HIV in the United States (and the 3rd time in the world). Doctors say that her case is a breakthrough and could offer new hope for other patients with the virus.

To understand the process we first need to do a quick science review.

• Immune cells or white blood cells in our blood are what fights off diseases
• All of these white blood cells are made from stem cells in your bone marrow.
• There is a genetic mutation to DNA that can make people naturally resistant to HIV. This mutation affects “CCR5”, a protein in our white blood cells.
• People with this mutation have white blood cells that attack HIV, and have a greatly reduced, if almost zero, chance of catching HIV.

Traditionally, if you get Leukemia or blood cancer, sometimes it gets so severe that they have to use radiation and chemotherapy to wipe out all the cancer cells and also all of the immune white blood cells in your body. Then the doctors do a bone marrow transplant to give you bone marrow that will create new white blood cells in your body, in order to fight off future infections.

In this recent case in NYC (and two others now), the patient had HIV and also Leukemia, and needed the radiation/chemotherapy and then a bone marrow transplant. As part of the test, the bone marrow was specially selected to come from someone with the CCR5 genetic mutation, so the bone marrow would create white blood cells that were resistant to HIV. The bone marrow transplant was successful, and the new white blood cells appeared to attack the HIV and removed all symptoms of the HIV from the patient. [see news article here.]

Research Laboratory PhotoIstock Photo

Moving forward, researchers are using gene editing to learn how to induce this CCR5 mutation.

The goal is to be able to draw blood from a patient, edit the patient’s blood to induce that CCR5 mutation, and then turn that blood into stem cells using what is called “Yamanaka factor” therapy.

This would allow them to use the patient’s own blood to create mutated stem cells that are resistant to HIV, and inject them back into the patient without having to take the risks that come with a bone marrow transplant. This is called autologous stem cell therapy, using your body’s own blood to create stem cells. (see added info here.)

Researchers are hopeful that the combination of these two processes, gene editing and autologous stem cell therapy, could lead to big breakthroughs in treatments for HIV, blood cancer, and even aging.