A new UCLA AIDS Institute study has found that gene therapy can be developed as a safe and active technique to combat HIV.
Researchers involved in this first-of-its-kind study found that gene therapy the potential to be a once-only treatment that reduces viral load, preserves the immune system and avoids lifelong antiretroviral therapy. The study appears in the current online edition of the journal Nature Medicine.
Though modest, the results do show some promise that gene therapy can be developed as a potentially effective treatment for HIV, said lead investigator Dr. Ronald Mitsuyasu, professor of medicine and director of the Center for Clinical AIDS Research and Education (CARE) at the David Geffen School of Medicine at UCLA.
"It is the first randomized controlled study done with gene therapy in HIV," said Mitsuyasu. "What we were able to demonstrate was that the patients who received the gene-modified cells had a somewhat better suppression of their HIV viral replication after discontinuing their highly active antiretroviral therapy (HAART) treatment, compared with the controls."
While there was nominal difference in viral loads between the test patients and placebo patients, researchers found that T4 counts were improved among the group receiving gene therapy, when compared to the placebo group.
"Part of the reason that we didn't see a larger effect is that the persistence of the anti-HIV gene in the patient's blood was not as long as we would have liked," he said. "We need to find better ways to get the genes into the patients and maintain them, which could include using different vectors to get the gene into the cells or conditioning the patients prior to gene transfer."
Still, the results indicate that gene therapy could eventually be a useful tool in the fight against AIDS, said study co-author Dr. Thomas C. Merigan, the George and Lucy Becker Professor of Medicine emeritus at the Stanford University School of Medicine. He agrees that more needs to be done to perfect it.
This study was funded by Johnson and Johnson Research Pty Limited. Grants from the National Institutes of Health also helped support part of the research. Courtesy Insciences.org
Researchers involved in this first-of-its-kind study found that gene therapy the potential to be a once-only treatment that reduces viral load, preserves the immune system and avoids lifelong antiretroviral therapy. The study appears in the current online edition of the journal Nature Medicine.
Though modest, the results do show some promise that gene therapy can be developed as a potentially effective treatment for HIV, said lead investigator Dr. Ronald Mitsuyasu, professor of medicine and director of the Center for Clinical AIDS Research and Education (CARE) at the David Geffen School of Medicine at UCLA.
"It is the first randomized controlled study done with gene therapy in HIV," said Mitsuyasu. "What we were able to demonstrate was that the patients who received the gene-modified cells had a somewhat better suppression of their HIV viral replication after discontinuing their highly active antiretroviral therapy (HAART) treatment, compared with the controls."
While there was nominal difference in viral loads between the test patients and placebo patients, researchers found that T4 counts were improved among the group receiving gene therapy, when compared to the placebo group.
"Part of the reason that we didn't see a larger effect is that the persistence of the anti-HIV gene in the patient's blood was not as long as we would have liked," he said. "We need to find better ways to get the genes into the patients and maintain them, which could include using different vectors to get the gene into the cells or conditioning the patients prior to gene transfer."
Still, the results indicate that gene therapy could eventually be a useful tool in the fight against AIDS, said study co-author Dr. Thomas C. Merigan, the George and Lucy Becker Professor of Medicine emeritus at the Stanford University School of Medicine. He agrees that more needs to be done to perfect it.
This study was funded by Johnson and Johnson Research Pty Limited. Grants from the National Institutes of Health also helped support part of the research. Courtesy Insciences.org
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