Lifesaving HIV treatment could reach millions more people following landmark study

July 4, 2013 — Millions more people could get access to life-saving HIV drug therapy, following a landmark study led by Australian researchers based at the Kirby Institute at the University of New South Wales (UNSW).The researchers have found a lower daily dose of an important HIV drug therapy is safe and as effective in suppressing the virus as the standard recommended dose.The findings have been presented at the International AIDS Society Conference in Kuala Lumpur, Malaysia.”This has the potential to affect the treatment of millions of HIV positive people,” says UNSW Professor Sean Emery, the protocol chairperson of the study, known as ENCORE1 and Head of the Therapeutic and Vaccine Research Program at the Kirby Institute.”A reduced daily dose should translate into a lower cost of treatment and permit more effective and efficient use of health care resources. Essentially, more people could receive this life-saving treatment for the same amount of funding.”HIV-positive people from 13 countries in Africa, Asia, Australia, Europe and Latin America took part in the trial. Half these people took two-thirds of the current standard daily dose of the antiretroviral (ART) efavirenz, a commonly used treatment for HIV; the other half took the standard daily dose. The 630 participants were observed regularly for a year. The results indicate that a reduction in daily dose of one third is both safe and effective compared to the higher dose currently recommended for people with HIV infection.The research was part of a program funded with a grant of US$12.42 million from the Bill & Melinda Gates Foundation.

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Gene variant may provide novel therapy for several cancer types

June 6, 2013 — A novel gene variant found in human and animal tissue may be a promising treatment for cancer, including breast and brain cancer, according to scientists from the Icahn School of Medicine at Mount Sinai. The variant, called PTEN-long, may contribute to a cell’s healthy function and also suppress tumor cell development.This landmark study is published in the June 6, 2013 issue of the journal Science.Ramon Parsons, MD, PhD, Professor and Chair of Oncological Sciences led the team that discovered a mutation in the tumor suppressor gene PTEN, which has subsequently been recognized as the second most common mutation in cancer, especially in breast, prostate, and brain cancers. PTEN encodes a 403 amino acid lipid phosphatase protein that is critical to cellular growth, proliferation, and survival. Genetic inactivation of PTEN causes tumor development.In the current study, Dr. Parsons and his team analyzed human cells and discovered a PTEN variant that has an additional protein sequence and is 43 percent longer than normal PTEN. They called this new variant PTEN-Long. Like PTEN, the long form has the same enzymatic activity, but unlike PTEN, it is secreted by the cell and can enter other cells, indicating that the added protein sequence acts as a delivery system for the tumor suppressor gene.”This study culminates more than a decade of research that began soon after we learned the therapeutic potential of PTEN and the PI3K pathway,” said Dr. Parsons. “We are excited about the potential of PTEN-Long as a therapy for multiple cancer types.”Using human breast and brain tumor cells that lacked PTEN and PTEN-Long, the research team introduced and overexpressed PTEN-Long and PTEN into the cells. They found that, similar to PTEN, PTEN-Long decreased the signaling activity on the PI3K pathway, thus reducing cellular proliferation. …

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