Emergency Med News

The first phase 2 trial of gene therapy for HIV treatment has shown promising results, but it is too early to say whether such treatment would be viable, there is enough evidence to justify further research on how to improve the approach, investigators said.

The research was the work of Dr. Ronald Mitsuyasu T, University of California Los Angeles (UCLA) and colleagues at UCLA and other research centers in the United States, Australia and Germany, and was published in online in Nature Medicine, February 15.

Mitsuyasu, director of the Center for Clinical AIDS Research and Education at UCLA (CARE), a professor of medicine in residence at UCLA, David Geffen School of Medicine, and deputy director of the UCLA AIDS Institute .

Although this first randomized, double-blind against placebo-controlled phase 2 trial in 74 adults infected with HIV do not show a statistically significant difference between the viral load in treatment and placebo group in the main criterion evaluation, further analysis has revealed that the gene therapy seems to have a modest but statistically significant, the effect of reducing the viral load in subjects treated with the placebo group, “said the article, abstract, who also suggested that the trial useful guidance on measures to improve for the future.

Although highly active antiretroviral therapy (HAART) has significantly improved the quality of life and prolonged the lives of people living with HIV, there is a risk of side effects and the virus begins to mutate into forms that are less sensitive the need for a new type of treatment is more and more each day.

Gene therapy has the potential to be once only treatment that reduces the amount of HIV in the body, preserves the immune system and avoids having to be on HAART for life.

For the study, Mitsuyasu and colleagues took blood stem cells (CD34 + hematopoietic progenitor cells) from patients in the treatment group, the change to make an enzyme called OZ1, then back into the patient. OZ1 targets two proteins that inhibit the replication of HIV itself. Patients in the placebo group underwent the same procedure except they received a placebo.

The trial was double blind, so neither the patient nor the treating health care team know if their stem cells process active enzyme or a placebo.

After 48 weeks, results showed no statistically significant difference between the two groups according to viral load (the amount of HIV circulating in their blood).

But after 100 weeks, patients who received OZ1 had higher levels of CD4 + cells circulating in their blood CD4 + cells are immune cells that are targeted and destroyed by HIV.