Approximately two miles north of Main Campus on North Broad Street, researchers at the Lewis Katz School of Medicine took another step in the fight against HIV.
The research team, led by Dr. Kamel Khalili—who chairs the neuroscience department—uses a gene-editing technology it developed to snip the virus out of cells. This month, the technique successfully stopped HIV-1 virus replication in the T-cells of blood from HIV-positive patients.
“I think it’s a very [big] first step toward the strategy which can be developed toward [the] cure,” Khalili said.
Dr. Wenhui Hu, an associate professor in neuroscience, said because HIV has “been integrated into the host cell,” the gene-editing technology is the best way to eliminate any further spread of the virus.
“Once the virus infects, the viral genome integrates into the host gene and becomes part of the chromosome and part of the DNA,” Khalili said. “The only way you can cure that is to eliminate the viral DNA by excision.”
In 2014, Khalili and his team of researchers eradicated the virus from cells through a DNA-snipping enzyme and guide RNA. There are many differences, however, between the gene-editing research and the research published in 2014.
“One really important thing is that the experiment was done in the patient samples—a proof of concept it can get into the clinic,” Khalili said. “The second one is the off-target and ensuring that there is no off-target effect, means the system is safe and it is not introducing any abnormality to the host. And then the third one is … that it significantly suppresses viral replication.”
Dr. Jeffrey Jacobson, a nationally and internationally known clinical researcher on HIV, has recently been appointed to professor of Medicine, professor of Neuroscience and professor in the center of Neurovirology in order to further the research on HIV.
“It couldn’t be better for our team, [and] it couldn’t be better for Temple,” Khalili said of hiring Jacobson.
By adding Jacobson to their team, Khalili added the next step is moving toward clinical trial.
“First we have to make sure that our systems are efficient and safe,” said Dr. Rafal Kaminski, a research scientist at the Lewis Katz School of Medicine.
Khalili added their systems would also have to be FDA-approved in order to begin clinical trials.
Currently, however, he said they are seeking funds in order to perform experiments on larger and smaller animals.
“To start a clinical trial?” Khalili said. “It’s hard to say. But my hope is within two years, but it can be three years, it can be one-and-a-half years. … So many parameters are involved in taking this one to clinic. But what I can tell you is, that right now we have the right people here, we have a good infrastructure and a process to build. And it should take us to that clinic. We will do the clinical trial right over here in Temple.”
For this research, they were funded by the National Institutes of Health, the Comprehensive NeuroAIDS Center and even some doctors chipped in, Khalili said.
According to the Centers for Disease Control and Prevention, there are 1.2 million people living in the United States with HIV, including 156,300 who are unaware of their infection. In addition, about 658,507 people have died overall from AIDS in the United States.
Khalili said their discovery is another reason for those infected with HIV to remain optimistic about their future.
“It’s a hope for the patients who never saw any light [at] the end of the tunnel,” Khalili said. “So it seems that at least the technology is in place to eradicate the virus. We just have to find the system and take it to the clinic. I think that would be the ultimate goal of our laboratory here as a whole.”
Tom Ignudo can be reached at firstname.lastname@example.org or on Twitter @Ignudo5.