January 4, 2011
San Francisco researchers led by Dr. Jacob Lalezari are looking for HIV-positive volunteers to participate in a groundbreaking study that uses gene therapy to modify patients' immune systems.
The study is based on work conducted in Germany on an HIV-positive man treated for leukemia. In 2007, the man received a bone marrow transplant from a donor with a rare genetic mutation that eliminates the CCR5 protein from the immune system. Without CCR5, HIV is unable to enter and infect T-cells. Three years after the transplant, HIV is undetectable in the patient.
Lalezari, medical director at Quest Clinical Research and assistant clinical professor of medicine at the University of California-San Francisco, and his team are exploring a less invasive approach. Rather than undergo a costly and painful bone marrow transplant, volunteers will have their blood filtered to extract immune cells. Those cells will then be treated with a zinc finger nuclease that will remove the gene that produces the CCR5 protein. Following cultivation for about three months, a large dose of treated immune cells will be re-infused in the originating patient in the hope they "take root" and replace vulnerable cells.
The treatment is expected to be painless and carry a relatively low risk of side effects. By contrast, the patient in the German study received chemotherapy and immunosuppressive drugs to prevent transplant rejection.
Interested volunteers must fit a very specific profile. Researchers are looking for HIV-positive people who have not taken antiviral drugs in the last 12 weeks, who are negative for hepatitis B and C, and whose T-cell counts are higher than 500. For more information, contact Quest at 415-353-0800 or e-mail Lalezari at firstname.lastname@example.org.