UCLA researchers find gene therapy that can prevent cells from HIV infection

Patients with HIV/AIDS are one step closer to controlling their disease without a regimen of daily medications.

A new gene therapy developed by UCLA researchers led to increased counts of CD4 T-cells, which prevent infection, in patients who were unable to maintain normal immunity levels with HIV drugs.

The results, which are seen by some as a functional cure, were announced in late September at the 51st Interscience Conference on Antimicrobial Agents and Chemotherapy in Chicago.

Current drugs for HIV/AIDS aim to keep virus replication as low as possible or below the point of detection, said Ronald Mitsuyasu, associate director of the UCLA AIDS Institute and the study’s principal investigator.

During the gene therapy process, researchers took white blood cells from the patients and inserted a gene that prevents the cell from getting infected with HIV.

When the cell replicated, it created more non-functional HIV genes. From there, the cells were frozen in a laboratory before being infused into a patient through an IV. In many cases, the T-cell immunity counts doubled and, for some, tripled, Mitsuyasu said.

Eventually, a population of cells that prevent HIV were created, as T-cells die after being infected with the virus.

In the long run, Mitsuyasu said he hopes the process can be simplified so the laboratory component of the gene injection can be eliminated, and the gene could be injected directly into the patient.

This treatment could be a better alternative for younger patients with HIV/AIDS, said Erin Ward, a fourth-year psychology student and president of the UCLA Pediatric AIDS Coalition.

“Part of the problem is taking pills every day,” Ward said, adding that individual HIV strains can become immune to treatment if patients forget to take their medicine. “(This treatment) could deviate emotional and cognitive consequences.”

The study has already been touted as a functional cure. For chronic diseases, a functional, or “non-sterilizing,” cure allows patients to maintain their normal life expectancy and die with the infection but not from the disease, said Carl June, director of translational research at the Abramson Family Cancer Research Institute at the University of Pennsylvania Perelman School of Medicine, one of the study sites, in an email statement.

But in spite of the positive connotation, Mitsuyasu said the results are simply a stepping stone for patients to sustain their immune systems at a normal, or near-normal, level on their own.

“It’s not a cure by any means for HIV,” he said. “We hope it will become part of a functional cure, but we’re not there yet.”

Though the possibility of a functional cure is promising, Ward said it won’t solve the entire problem of HIV/AIDS.

“We need to advocate for the removal of the stigma (of the disease),” she said. “If we can mark one thing off the list, we can put more effort into educating the public.”

With the results of the past study still fresh, Mitsuyasu and his team are now into their second study, which will test the technique on patients who have not yet taken anti-HIV medication to see if gene therapy can achieve a true anti-HIV effect.

This additional research will give a better sense of the treatment’s effectiveness, Mitsuyasu said.

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