Probing the Untapped Potential of Gene Therapy

New Grant Cycle Nurtures Promising New Field

November 17, 2009—amfAR will enter the field of gene therapy for the first time with a new round of funding aimed at exploring how this emerging discipline can be used to target HIV.

Gene therapy holds much promise against HIV as well as other diseases, but setbacks have hindered its progress. However, a report in the February 2009 New England Journal of Medicine has generated renewed interest. The report concerns an HIV-positive patient in Berlin with acute leukemia who underwent a stem cell transplant from a donor who had a genetic mutation that rendered that donor—and now the patient—resistant to HIV.

Off all antiretroviral therapy for more than two years and off all immune suppressive medications, the Berlin patient now shows no detectable signs of HIV. “At the very least,” said amfAR’s senior scientific consultant Dr. Jeffrey Laurence, “this case represents a proof-of-principle that gene therapy approaches may one day cure HIV infection.”

For scientific researchers, the implications of this case are intriguing. By soliciting proposals relevant to the role of gene therapy in the treatment and potential eradication of HIV infection, amfAR hopes to stimulate investigation into some of the most pressing unanswered questions posed by this new development.

The new request for proposals asks researchers to address questions such as: Which are the best targets for gene therapy? How can such therapies be delivered into a patient’s body most efficiently? And what exactly led to the apparent cure in the Berlin patient? Awards are expected to be announced in February 2010.

“Eradicating HIV represents one of the greatest challenges facing AIDS research,” said Dr. Rowena Johnston, amfAR’s vice president of research. “The field of gene therapy fits perfectly with amfAR’s dedication to pursuing cutting-edge research directions with the potential to revolutionize how we treat, and ultimately cure, HIV.”