Gene-base therapy may be effective against HIV, U.S. researchers report. An investigational gene-based immunotherapy called VRX496 suggests it can fight the virus, according to a phase I, open-label, non-randomized clinical trial conducted at the University of Pennsylvania School of Medicine.
The trial evaluated the safety and tolerability of VRX496 — a CD4 T cell treatment — in five people with chronic HIV infection who had all failed to respond to at least two antiretroviral drug regimens.
In response to the treatment, the five patients experienced decreases in viral load and showed stable or increased CD4 T cell counts.
The findings were published Monday in the journal Proceedings of the National Academy of Sciences.
“Gene therapy has long been discussed as an alternative treatment to HIV,” study senior author Dr. Carl June said in a prepared statement. “The results from this phase I trial are encouraging — particularly since these are late-stage patients — and demonstrate that gene therapy has the potential to treat HIV and other serious human diseases.”
The therapy is being developed by VIRxSYS Corporation of Gaithersburg, Md. Phase II trials to test the safety and tolerability of single and repeated doses of VRX496 are currently under way, and preliminary results should be available in 2007.
Dr Bruce Levine, who also worked on the study – published online in Proceedings of the National Academy of Science – said: “Just because this has produced encouraging results in one or two patients doesn’t mean it will work for everyone. We have much more work to do.”
Dr George Schmid, a specialist in HIV at the World Health Organization, said it could take many years to determine whether the technique was safe and effective, but he said the results were “encouraging”.
The research raises the prospect that gene therapy might provide an alternative to antiretroviral drugs.
The long-term effectiveness of the drugs is under threat from the growing problem of drug resistance.