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New Research Indicates Gene Therapy May Be An Effective Treatment For HIV

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Robert Dominic Ventre II

Staff Writer

Earlier this month, the White House announced that a new method involving no medication was being developed by the National Institute of Health as a way to treat HIV. Judged as safe for those afflicted, the technique involves isolating key cells in HIV patients, removing and genetically modifying them, before returning them to their host. The initial research was funded by the National Institute of Allergy and Infection Diseases (NIAID – a part of the National Institutes of Health) while the first (Phase I) clinical trial for this new treatment plan was funded by Sangamo BioSciences. The trial was lead by NIAID grantee Carl. H June, M.D., as well as Bruce L. Levine, Ph.D. and Pablo Tebas, M.D.. All trial leaders are of the Perelman School of Medicine at the University of Pennsylvania, Philadelphia.

Of the 12 patients that took part, one was revealed to have lost all detectable traces of the HIV virus post-treatment.

Per Carl H. June, M.D. (who also acted as senior author of the study):

“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.”

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Certain individuals are born with a genetic mutation (known as the CCR5 mutation) that makes them effectively immune to the HIV virus. This newly-developed treatment involves replicating that same resistance in HIV-positive patients via alterations to the structure of their T-cells, which allows their bodies to effectively fight against HIV without the aid of daily medications.

Sharon Lewin of Monash University had this to say regarding the intended goal of the study and its findings:

“What most people are aiming for in HIV is a way you take treatment for a short period of time and that keeps the virus under control,”

In 2006, Timothy Ray Brown, now known colloquially as the ‘Berlin Patient’, was on his 11th year of anti-retroviral therapy for HIV, which helped to keep the virus at bay. After being diagnosed with acute myeloid leukemia and undergoing chemotherapy, which had a severely negative impact on his immune system (eventually resulting in a bout of pneumonia followed by sepsis during treatment), oncologist Dr. Gero Hutter of the Charite Hospital in Berlin chose to give Brown a stem cell transplant in order to treat his leukemia. Instead of using a matching donor, Hutter specifically chose to use stem cells gathered from a donor that had the CCR5 mutation. The result: Brown became leukemia free, and is also now considered the first person who has been “cured” of the HIV virus. He no longer receives medication and is reportedly doing well.

As this form of treatment is still undergoing early clinical trials, it is likely “still a long way off” from becoming a routine option for all patients afflicted by HIV, as stated by the University of Monash. But the results it has shown and the implications it raises are a great sign for sufferers of the disease. Other recent developments regarding the treatment of HIV, coupled with this new form of treatment, may prove to be a serious game-changer in the medical world.

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