A Step Forward for Gene Therapy to Treat HIV

May 3, 2012

Bloomberg Businessweek

A new Penn Medicine study shows that treating HIV patients with genetically modified versions of their own T cells – a strategy that aims to turn patients’ own immune systems against the virus – is safe and does not lead to any dangerous side effects more than a decade after receiving the new cells. All 43 patients studied for the new paper, published in Science Translational Medicine, remain healthy 11 years after the new cell infusion, and 41 of those patients show long-term persistence of the modified T cells in their bodies. Previous uses of gene therapy in experiments have suggested that leukemia caused by the viruses that transfer the new genes to the cells might be a risk. The new findings allay that concern, enabling researchers to move the research beyond immediately life-threatening illnesses, such as HIV and cancer. The research was led by Carl June, MD, a professor of Pathology and Laboratory Medicine. “We turned those cells into heat-seeking missiles directed against HIV-infected cells,” said Bruce Levine, PhD, an associate professor of Pathology and Laboratory Medicine and director of the Clinical Cell and Vaccine Production Facility, in a Bloomberg BusinessWeek article. The approach is not a cure, but the new data provide important clues about how patients fare in the long run after receiving the modified cells. "It looks like if you do this, it's going to be safe because we have not seen any toxicity in 16 years," Pablo Tebas, MD, director of Penn’s AIDS Clinical Trials Unit, told NPR. "And two, the genetically modified cells are still circulating. They perpetuate. Those are two important things this study is telling us... Read More

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