Biochem’s Gene Therapy May Offer ‘Alternative’ to Combination Therapy
Researchers at Enzo Biochem Inc. have developed a "unique" gene therapy approach to AIDS treatment that could "offer an alternative to a lifetime" of drug combinations, Reuters reports. Enzo presented findings from Phase I clinical trials indicating that patients' cells can safely incorporate "antisense" RNA that "interferes with how the virus works." However, the patients did not show decreased viral loads. The Enzo approach involves a combination of gene therapy and bone marrow transplantation, a "common" cancer therapy. Researchers performed the study on five HIV-positive patients who had high viral levels and were not responding successfully to standard AIDS therapies. Researchers extracted bone marrow from these patients, purified out bone marrow stem cells, and inserted the antisense genes into the stem cells (Fox, Reuters, 2/6). Bone marrow stem cells are the progenitors of CD4+ T cells, the cell type infected by HIV. The study found that the genetically modified stem cells not only survived, but also successfully developed into CD4+ cells. When the modified stem cells were injected back into the patients, researchers could detect antisense RNA in the patients six months later, and beyond six months in five of these patients (Enzo release, 2/6). However, the new genes did not affect patients' viral levels. In order for its approach to reduce viral load levels, Enzo Vice President Dean Engelhardt believes researchers will have to "partially destroy the patient's bone marrow" before inserting genetically modified bone marrow, a similar approach used in treating cancer. Enzo plans to seek permission from the NIH and the FDA to attempt this "next step" (Fox, Reuters, 2/6).This is part of the KHN Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.