U Penn pioneers HIV gene-therapy trial

Earlier this month, researchers from the University of Pennsylvania unveiled a research study in which they’re attempting to create HIV-resistant blood cells — and so far are seeing encouraging results.

The Penn scientists undertook the gene-therapy trial in July 2009, and the study is still accepting participants.

Researcher Dr. Pablo Tebas explained the study involves the physical manipulation of the T-cells in HIV-positive patients’ blood to remove the receptor through which HIV gains access to the cells.

“We separate the cells in the lab and the protein in the membrane of the cell where the HIV virus uses to enter the cells is taken out,” Tebas said. “So we remove that particular gene from the cells and then monitor the patients to see if the HIV continues to expand or not.”

The initial results have indicated that, after being placed back in the patients’ blood, the engineered cells did not become infected by HIV and “expanded” or multiplied, accounting for up to 6 percent of some patients’ T-cell count.

Tebas noted that, while the results are preliminary and much more research is needed, the study is promising.

“We’ve seen that it is doable,” he said. “We can take cells and genetically modify them and reinfuse them and it’s safe, nothing bad has happened to the patients. The cells are alive when they go back into the patient and, while we don’t have all the data yet, the cells have expanded so far as normal cells as we can tell, which means that they are protected from HIV infection.”

Tebas said the ultimate goal of the study is to get HIV-positive patients completely off their medications and free of the virus, and while that outcome is a long way down the line, the current study is necessary to eventually reach that end.

“We’re basically looking to cure HIV infection but we’re far away from a cure. Right now we’re looking to answer the question of if you can make an immune system, in a patient, that is resistant to HIV. In order for there to be an actual cure, we’d need all cells in the patient to have this gene removed, and we can’t do that yet — we’ve only removed a very small fraction. But this is the first step of going in the direction of building an entire immune system that’s resistant to HIV.”

Penn’s AIDS Clinical Trials Unit is still recruiting for the study. For more information, call 215-349-8092.

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