University of Pennsylvania Team Receive Prestigious National Clinical Research Award for HIV Breakthrough

Researchers from the Perelman School of Medicine at the University of Pennsylvania and the Penn Center for AIDS Research (CFAR) are among the 2015 recipients of the prestigious Clinical Research Achievement Award for their personalized gene therapy work in HIV. The team included Carl H. June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine, Bruce L. Levine, PhD, the Barbara and Edward Netter Professor in Cancer Gene Therapy in the department of Pathology and Laboratory Medicine and the director of the Clinical Cell and Vaccine Production Facility, and Pablo Tebas, MD, director of the AIDS Clinical Trials Unit at the Penn CFAR.

The Clinical Research Forum recognized the year’s 10 most outstanding research papers written by teams from across the nation at its 4th annual awards ceremony in Washington, DC, on April 16. The winning papers were chosen based on their degree of innovation from a pool of more than 50 nominations from 30 research and academic health centers nationwide.

The Penn team’s work, published in the New England Journal ofMedicine in March 2014, was the first successful clinical test of any gene editing approach in humans. In the phase I study, they engineered the immune cells of 12 HIV positive patients to resist the HIV infection, by mimicking a naturally occurring mutation occuring in a very small number of people that renders their cells resistant to HIV infection. Researchers used a zinc finger nuclease technology to induce the genetic CCR5 mutation in patients’ T cells to lock out HIV. The modified T cells persisted in all patients, they found, and reduced viral loads in some taken off treatment entirely.

“This study shows that we can safely and effectively engineer an HIV patient’s own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs,” said June, the study’s senior author. “This reinforces our belief that modified T cells are the key that could eliminate the need for lifelong antiretroviral drug therapy and potentially lead to functionally curative approaches for HIV/AIDS.” For more on the study, read the press release here.

The Forum and its supporters believe these papers represent the best and brightest work in the field, and will lead to advancements in medicine that will change lives and patient outcomes worldwide.

Click here to view the full release.

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