HIV Cure & Treatment Found? FDA Approves Gene-Editing Method

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The U.S. Food and Drug Administration (FDA) has approved a new trial on blood-producing stem cell gene editing treatment for HIV. The therapy is focussed on causing specific mutation in the white blood cells (WBC) of patients with HIV that would prevent the virus from attacking the cells, lasting for a minimum of four years in the body.

The gene editing technique involves altering the gene of blood-producing stem cells of patients infected with HIV by mutating CCR5, a protein present on the surface of these cells. The technique is inspired from a naturally occurring mutation in few people with HIV wherein CCR5 is altered; thereby preventing HIV from attaching to the blood cells, according to The Body.

The blood-producing stem cells are obtained from the bone marrow of the patient and the proteins that viruses use to infect are edited using an enzyme zinc finger nucleases referred as molecular scissors, reports San Francisco Business Times.  

Using the technique developed by Richmond's Sangamo BioSciences Inc. to cause mutation, HIV present in the body wouldn't be able to enter the T cells and replicate in them. Therefore, the immune system of the HIV-infected patients will remain uncompromised. The T-cell is a type of WBC that finds, destroys and stores information of foreign antigens that enter the human body.

Paul Knoepfler a stem cell researcher at the University of California, Davis, said that the FDA's approval of the new drug application is a great step to move forward. According to San Francisco Business Times "'Cure' is a big word, but there's hope. First step is to assess safety, but IND approval is (a) major positive step. Then determine efficacy", said Knoepfler.

The clinical trial would be conducted in the City of Hope medical center in California, sponsored by the California Institute for Regenerative Medicine (CIRM), the state's stem cell program. It is to be noted that the CIRM, that has funded about $ 5.6 million of the City of Hope medical center's clinical trial, is also funding another similar program by Calimmune, a Tucson, Ariz. company, reports U-T San Diego.

The study, that includes HIV infected patients who don't respond well to antiretroviral drugs, would be carried out by researchers from Sangamo BioSciences, Inc., and Keck School of Medicine at the University of Southern California. According to San Francisco Business Times, Sangamo has already treated about 70 HIV-infected people using the zinc-finger technology providing functional cure for the ailing patients.