Gene research could be key to HIV treatment advances
Procedure could block HIV replication in cells
It’s a long way from being used in a clinical setting, but Philadelphia researchers have had some success in testing a process that could actually render HIV harmless.
"We tested a concept that it might be possible to block replication of HIV in cells that are already infected," says Stuart Starr, MD, professor of pediatrics at the University of Pennsylvania Medical School and chief of immunologic and infectious diseases at The Children’s Hospital of Philadelphia.
Most of the latest genetic research into HIV treatments has focused on protecting uninfected cells from becoming infected with HIV. Starr’s group instead focused on infected cells and whether a genetic approach might be helpful, along with other therapies.
"So this study is a proof of the concept that a genetic approach might also be considered for infected cells," Starr says.
The researchers already knew that the HIV tat gene is essential for the virus to replicate in the infected cells. So, they designed an anti-tat gene that blocks the HIV tat gene’s function. Then they inserted the anti-tat gene into a mouse retrovirus that can enter cells that are sites for HIV replication. Researchers took blood immune cells taken from patients infected with HIV and inserted the beneficial gene into those cells. This effectively blocked the AIDS virus from replicating in those cells and prevented the virus from activating.
The research done so far is only a first step, Starr says.
"In order to test this concept further, we need to move into an animal model and see whether we can achieve similar results," he explains. "There are formidable obstacles to getting this to work in a whole animal, as well as in the test tube, and at the moment I would say this approach is going to require a lot of work to bring it into the clinic."
If the process continues to work on animal models and then in humans, it could offer HIV patients and their physicians an alternative to antiretroviral therapy, or at least a longer latency period in which patients are free of symptoms before antiretroviral drugs are necessary, Starr says.
"One reason for developing this approach is that the other approaches have given outstanding results in terms of short-term clinical improvement and outlook for quality of life, but unfortunately, the drugs don’t help everyone," Starr says. "And they have side effects and they are very expensive, so while they’ve been a tremendous improvement, they may not be the total answer."
Starr estimates the research still is three to five years away from being developed for clinical use.
"Even though we’re a long way away from having a genetic approach that has a reasonable chance of working in humans, we thought it was important to take the first step, because this approach eventually may be an important complement to other treatments," Starr adds.
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