Study: Gene Therapy for HIV Safe, But Effectiveness Still
WEDNESDAY, May 2 (HealthDay News) -- New research shows that
gene therapy can have long-lasting effects on the immune cells of
HIV patients -- a promising sign -- even though the specific
treatment being studied did not eradicate the virus.
This approach is one of several gene therapy strategies that are
being investigated by scientists as possible ways to keep the AIDS
virus from spreading in the blood.
In this case, "people were treated by gene therapy and nothing
bad happened. It was safe," said study co-author Frederic Bushman,
a professor of microbiology at the University of Pennsylvania.
In addition, he said, the treated immune cells managed to remain
around for about a decade. "The general picture that emerges about
genetic alterations to human immune cells is that they can persist
for a long time if you do it right."
The study appears in the May 2 issue of
Science Translational Medicine.
Researchers have long been exploring gene therapy -- in which
cells in the body are genetically modified -- as a possible
treatment for infection with HIV, the virus that causes AIDS. The
idea is that the therapy would offer a permanent alternative to
costly medications that come with potentially disabling side
"Just think about what an HIV patient has to do: take drugs every day for the rest of his life, and the minute he stops taking them, the virus starts coming back," said John Rossi, chair of the department of molecular and cellular biology at the Beckman Research Institute of the City of Hope, in Duarte, Calif. He was not associated with the new research.
The study looks at 43 HIV-positive patients. Between 1998 and
2002, researchers removed blood from the patients, genetically
modified it, and injected it back into them.
The plan was to program immune cells known as T cells to kill
Up to 11 years later, researchers found that all 43 patients are
healthy, and 41 still have modified T cells in their bodies. That
means the modified cells didn't cause leukemia, as has happened
with some similar gene -therapy treatments.
The treatment, however, didn't seem to have had a major impact
on the HIV in the patients and "may not have worked at all" on that
front, study co-author Bushman said.
Dr. David Looney, director of the Center for AIDS Research at
the University of California, San Diego, said the research is still
"exciting and promising" since it shows that modified immune cells
can last a long time, potentially decades.
Several research projects are continuing to examine gene therapy
for HIV patients. There's a caveat, however: If gene therapy
treatments become available, they seem likely to be extremely
But Bushman said that if the gene therapy could someday replace
antiretroviral medicines, the cost might still be lower than
keeping patients on drugs for rest of their lives.
For more about
HIV, visit the U.S. National Library of Medicine.
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