Gene Therapy Against HIV Not a Proven Cure

Experts are reacting with cautious optimism to the announcement Monday that researchers reconfigured immune cells so that they became resistant to HIV in six patients infected with the virus. But they say the jury is out on whether the technique might ever spell an end to AIDS. The goal is ultimately a cure or what's called a "functional cure" having the body permanently keep HIV at bay but "we're not there yet," stressed Dr. Michael Kolber, professor of medicine and director of the Comprehensive AIDS Program at the University of Miami Miller School of Medicine.

The trial, reported Feb. 28 at a meeting of HIV specialists in Boston, "was a proof-of-principle that they could go in and do this. They demonstrated that the cells stayed in the patients, but the patients were not cured," said Kolber, who was not involved in the new research. Another expert agreed that the treatment's true potential remains uncertain. "If successful, this probably could have wide application, but going from six patients to an entire epidemic is a ways to go," said Dr. Michael Horberg, director of HIV/AIDS at Kaiser Permanente Health Plan and vice chair of the HIV Medicine Association.

"With other successes we've already had, that makes it more promising and people are starting to have a greater vision as to what's possible." However, as Kolber pointed out, this trial was what's known as a phase I trial, which means it was primarily looking at safety, not effectiveness, although investigators do often report on initial effectiveness results at this stage. The idea came from an isolated case that first made headlines in 2009, involving the so-called "Berlin patient." This man, an American AIDS patient living in Germany, was apparently cured after receiving blood cells from a donor who happened to have a rare, natural immunity to HIV.