After successfully removing the HIV-1 virus from living animals this past spring, researchers plan to test on human subjects.
The technology is being prepared for clinical trials on humans in the next two years.
Neuroscience department Chair Dr. Kamel Khalili is the lead doctor for the technology. He and his team were the first to eliminate the HIV-1 virus from cultured human cells in 2014, and successfully removed HIV-1 DNA from living animals this past spring.
He said his past accomplishments paved “a path toward the clinical trials.”
After several more studies testing the safety of the gene-editing technology, Khalili plans to contact the Food and Drug Administration and the National Institutes of Health to get approval to open the technology up to patients.
Khalili’s research has demonstrated the technology’s ability to edit human and animal genes and remove HIV-1 DNA, a significant step towards finding a cure for the virus.
“Our next step is to complete our preclinical studies,” Khalili said. “Then I think we may be able to enter into clinical trials, hopefully in less than two years.”
Khalili said when the time comes for clinical trials, the research method will likely be intravenous therapy, or IV injection, but the team is “still in the midst of developing these basic procedures” for treatment.
Khalili said he is working to ensure the treatment methods are simple and accessible so that in the future, areas of the world lacking access to expensive treatments are able to utilize the necessary HIV treatment.
“It’s becoming easier and easier to imagine a cure for HIV,” said Dr. Robert Bettiker, an associate professor of medicine and infectious diseases and an HIV doctor at Temple University Hospital.
Bettiker is part of Temple’s Comprehensive HIV Program, headed by Dr. Ellen Tedaldi, which provides HIV-related care to more than 1,100 patients, according to the program’s website.
Bettiker added that his HIV patients at TUH are “fired up” about the steps Temple has made in HIV research.
According to the Philadelphia Department of Health, there are about 30,000 people infected with HIV in the Greater Philadelphia Region, and the city has an infection rate five times the national average.
Khaili said the gene-editing systems he and his team have developed are not necessarily HIV-specific, and he has started researching how to adapt the technology for other viruses.
“We are expanding this strategy to go beyond just HIV,” Khalili said. “This is very serious research.”
“These are the kind of results that were a dream to us five years ago,” Khalili said. “But we still have more homework to do.”
Noah Tanen can be reached at noah.tanen@temple.edu.
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