Biotechnology Company to Launch Trial of New HIV/AIDS Treatment That Would Target DNA

A biotechnology company on Monday announced plans to start human trials of a new approach to treating HIV/AIDS that targets patients' DNA, Reuters reports. California-based Sangamo BioSciences' drug SB-728-T is designed to disrupt the CCR5 gene -- the protein on the surface of immune cells to which HIV attaches. The study involves removing CD4+ T cells from people living with HIV and treating the cells with the drug, called a "zinc finger nuclease." The treated cells would then be infused back into the patient with the hope that they will flourish and multiply, making the patient's immune system resistant to HIV, Reuters reports. According to Sangamo, 12 people living with advanced stages of HIV will be recruited to participate in the Phase I trial, which will focus only on the safety of the therapy.

Carl June of the University of Pennsylvania School of Medicine, who will be involved with the trial, said, "This is the first time that we have had the ability to make a patient's T cells permanently resistant to infection by CCR5-specific strains of HIV." He added that researchers are "very excited to begin a clinical trial of this novel zinc finger nuclease-based therapy." June said, "The ability to protect immune cells from infection with HIV and the expansion of CCR5-modified T cells has the potential to provide long-term control of both the virus itself and eventually the opportunistic infections characteristic of AIDS" (Fox, Reuters, 2/2).

German researchers in November 2008 reported that an HIV-positive man who underwent a bone marrow transplant to treat leukemia had experienced undetectable HIV viral loads for almost two years. For the procedure, physicians replaced the patient's bone marrow cells with those from a donor with a naturally occurring gene mutation that provides immunity to almost all strains of HIV by preventing CCR5 from appearing on the surface of cells (Kaiser Daily HIV/AIDS Report, 11/7/08).

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