An early-stage trial has provided some important proof that gene therapy could be an effective way to fight HIV. Taking five patients who had already failed to respond to standard therapies, the researchers removed immune cells from each of the participants and injected modified lentivirus in each, changing the genetic code of the DNA of the cell so it can fight HIV and prevent the infection from spreading. In Phase II, a larger number of volunteers with HIV will get more than one infusion of the modulated cells. The scientists, led by Dr. Carl June, found that one of the volunteers responded with a significant reduction in the virus while immune cells multiplied and the immune system strengthened in the other four. The fact that they can introduce genes into cells without toxicity or provoking cancer, as has happened in earlier trials, is exactly the kind of proof of concept that gene therapy experts have been working toward.
- read the Washington Post report on gene therapy