Gene Therapy

Gene Therapy

Researchers at the University of Pennsylvania School of Medicine have reported favorable results using gene-based therapies in the fight against HIV. Gene therapy uses genetically altered CD4 cells that are resistant to HIV, protecting them from the damage caused by the virus. By protecting the CD4 cells, the immune system is preserved and remains able to do its job of fighting infection. In Phase I of the gene therapy trials, the experimental drug VRX496 was given to five patients who previously demonstrated resistance to conventional HIV therapies. When they re-examined the five after nine months of therapy, four of the five showed stable or increased CD4 counts, as well as improved immune system function. In addition, a decrease in HIV viral load was observed in three of the patients. While these results are positive, researchers will follow the five patients for up to 15 years after therapy in order to get a better picture of just how well gene therapy works. In the meantime, researchers are planning a second trial, this time using patients who have well-controlled HIV viral loads to see if the results are similar.