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gene therapy via blood stem cells new treatment for hiv ???
Jun 7, 2009

Hi Dr Bob,

l am recently infected with hiv and guess what you have become my hero, please keep up the great work you do for us hivers around the world, l read all your posts and you are the man period... l need to start treatment asap cd4 300 and viroal load 85,000k.... but l have been reading about this blood stem cell treatment via gene therapy,

look l know treatment like this are at least a hope for us to try and live a normal life without meds,

Dr Bob get the crystel ball out the same one in the harry potter film and find out when can we sometime in the future get started on this cutting edge treatment,keep up the great work, you are a very special person.....

cheers

pat

Gene Therapy Holds Promise for HIV But research is preliminary and more trials are needed, study says Posted February 15, 2009 By Randy Dotinga HealthDay Reporter

SUNDAY, Feb. 15 (HealthDay News) -- Researchers are reporting promising results from the largest clinical trial of gene therapy for HIV patients. The findings, while encouraging, need to be replicated and more research must be done before an effective new treatment arrives, experts said.

People Who Read This Also Read Many Hospital Patients Can't ID Their Doctors 21983311 Love and Romance: Get the Health Benefits Even if You're Single 21983693 Recent HIV News Is Encouragingbut Also Provides Cause for Concern 21955837 Add More Facts to Drug Ads, Experts Urge 22045400 A Caution About Rapid HIV Testing 20194973 "We know it works, but we need to make it better before we launch a later study," said study lead author Dr. Ronald Mitsuyasu, director of the University of California, Los Angeles Center for Clinical AIDS Research and Education.

Existing treatments work fairly well for many people with HIV, the virus that causes AIDS. But patients have to take expensive drugs for their entire lives, and side effects can be significant.

Gene therapy is considered a possible alternative to drug treatment. Researchers hope to fight the virus by inserting a gene or genes into the body of an infected individual.

Last week, the New England Journal of Medicine reported a case that showed the potential for gene therapy and HIV. According to the journal, the body of a man in Germany with AIDS became free of the virus after he received a bone marrow transplant from a man with a gene that made him virtually immune to HIV. The treatment may not be a cure because the AIDS virus can become undetectable while still remaining in the body.

In the new study, researchers focused on a molecule that targets the AIDS virus. The scientists recruited 74 HIV-positive people and intravenously gave them either the molecule -- called OZ1 -- via blood stem cells, or a placebo. The patients then cycled on and off their HIV drugs so the researchers could see if the virus rebounded.

The patients who got the gene therapy treatment had a "modest decline" in the level of virus in their bodies, Mitsuyasu said. After 100 weeks, immune systems were also stronger in those patients who received the gene therapy vs. those who did not. In fact, some patients are doing so well that they haven't returned to drug treatment, Mitsuyasu said.

The findings were published online Feb. 15 in the journal Nature Medicine.

The study was a "phase II" trial, meaning that a third study would be necessary before the treatment could be approved.

There's more to be done, however, Mitsuyasu said.

"We need to figure out how best to perfect this approach. There are a lot of things that we could potentially do," he said. But in the big picture, the research represents "the first clear indication in a randomized and controlled study that there is a biologic effect of gene therapy" for HIV, he added.

The new approach holds promise, said Rowena Johnston, vice president of research at the Foundation for AIDS Research (amFAR) in New York City.

"One of the striking successes of this particular approach is its apparent safety, which is no small matter given the history so far of gene therapy interventions," she said. "Gene therapy will probably represent an exciting new frontier that will receive increasing attention in the coming years. There are so many unexplored avenues to pursue with the real potential to provide a lasting solution for HIV/AIDS."

More information

To learn more about gene therapy, visit the Human Genome Project Information

Response from Dr. Frascino

Hello Pat,

Thanks for your kind comments.

The results of the OZ1 stem cell gene therapy phase II clinical trial were exciting and encouraging in equal measure. Dr. Ron Mitsuyasu at UCLA is an excellent HIV/AIDS/oncologist physician specialist who has been conducting cutting edge immune-based HIV research for quite some time. (He's also a good friend of mine!) His comment that this study is "the first clear indication in a randomized and controlled study that there is a biological effect of gene therapy" for HIV should help in getting funding and approval for additional HIV-related gene therapy clinical trials. This study was encouraging but not a "homerun," as the treatment group only experienced a "modest decline" in HIV plasma viral load. However, there was evidence that the treatment group had sustained (out to 100 weeks) strengthening of a number of immune parameters. This type of immune-based therapy is exactly what's needed, in my opinion, to work with our antiretrovirals (or ideally in place of the antiretrovirals!). As for when this type of therapy will be read for primetime, unfortunately even Professor Trelawney's crystal ball can't predict. What we can say is that it's still quite a ways off (years). However, for now we should celebrate a major step forward with a big collective WOO-HOO!!!

I'll keep you posted on this as other promising treatment stories continue to evolve.

Be well Pat! Let's all be here for the cure, OK?

Dr. Bob



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