New Gene Therapy to Control HIV

When somebody is infected with HIV, their only defense is a cocktail of antiretroviral drugs that they will continue to take for the rest of their lives. While these antiretroviral drugs have helped infected patients live long and relatively healthy lives, keeping AIDS at bay, they are still very powerful drugs that can sometimes wreak havoc on a patient’s body. A new study that has been released in the online version of Nature Medicine has demonstrated that perhaps one day, lifelong antiretroviral treatments might be a thing of the past.

The new study conducted by the UCLA AIDS Institute has used gene therapy as a safe way to fight HIV. Those scientists and researchers involved with this study have revealed that a gene transfer done with cell delivery could be the key to having patients treated only one time when they are infected with HIV. This treatment would also keep the immune system intact while reducing the viral load. Dr. Ronald Mitsuvasu, a medical professor, the director of the Center for Clinical AIDS Research and Education (CARE) and lead researcher in the study realizes the results of the study are a bit modest, but they do show that gene therapy could have its place as scientists continue to develop effective and promising treatments for HIV. He states, “It is the first randomized controlled study done with gene therapy in HIV. What we were able to demonstrate was that the patients who received the gene-modified cells had a somewhat better suppression of their HIV viral replication after discontinuing their highly active antiretroviral therapy (HAART) treatment, compared with the controls.”

The study had 74 participants, all who are infected with HIV. Each participant was a recipient to their own harvested blood stem cells; these stem cells where either as-is or treated to harbor a molecule named OZ1. OZ1 is responsible for helping to prevent HIV from replicating by pinpointing a main HIV gene. As the trial progressed over 100 weeks, OZ1 was deemed to be safe with no ill side effects. Once a participant had stopped highly active antiretroviral therapy, or HAART treatments, after eight weeks, the differences in viral load between those participants in the placebo group and those in the OZ1 group were not very noteworthy. However, other parameters showed more significance. For example, HIV was better suppressed and there was a noted improvement in the CD4+ lymphocytes counts (these are the cells that HIV eradicates).

Mitsuyasu is well aware that this gene therapy needs to be worked on much more. He states, “Part of the reason that we didn’t see a larger effect is that the persistence of the anti-HIV gene in the patient’s blood was not as long as we would have liked. We need to find better ways to get the genes into the patients and maintain them, which could include using different vectors to get the gene into the cells or conditioning the patients prior to gene transfer.” The study’s co-author, Dr. Thomas Merigan, adds, “But in the way we set up the trial with randomized placebo controls, we could dissect out that there was a positive effect in patients who had the gene successfully installed. This could be a first step in developing a new method of controlling a chronic infectious disease.”