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Health : HIV – A Functional Cure?

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A possible “functional cure” for HIV has recently been granted FDA approval for further human testing. The method uses genetic modification to cause a specific mutation in the white blood cells of HIV patients which mirrors those found in the naturally immune. It has so far shown to be both receptive and long-lasting.

The novel therapy involves taking stem cells from HIV-infected patients and using a gene editing tool to cause them to form into white blood cells with a specific mutation. The mutation affects a protein known as CCR5, and interferes with the virus’s ability to latch onto blood cells. The mutation occurs naturally in a small percentage of the world’s population and gives these individuals a life-long resistance to HIV infections. Although the virus may remain in their body, without being able to enter the T cells, it cannot replicate and therefore will stay at low numbers, uncompromising the immune system.

In theory, when these genetically edited stem cells are reintroduced into HIV patients they will repopulate the body with cells possessing the same mutation. This would give the patients the same lifetime resistance to the virus’s harm with just one procedure. The method was developed by Sangamo BioSciences Inc., but has also been tested in early human clinical trials by drug research company Calimmune, San Francisco Business Times reported.

According to IFL Science, in a small trial consisting of only 12 patients the procedure was found to be tolerable and have a low risk of adverse side effects. The genetically modified cells lasted up to four years inside the patients. Unfortunately, the trial was not large enough to test the effectiveness of the procedure, but the current FDA approval will allow the testing to be extended to treat more HIV patients. The FDA has also approved the start of a new Phase I safety study, which would consist of a multi-year, three-stage process of human trials to test a similar approach using a different method of disabling the CCR5 protein.

As reported by the SF Business Times, the trials will be conducted at the City of Hope medical center in California and is being funded by CIRM, the state’s stem cell research funding agency. It will be run by researchers from Sangamo BioSciences Inc. and the Keck School of Medicine at the University of Southern California. They will include people with HIV/AIDS who have had poor responses to standard therapies.

“This kind of work is too important to just try one method at a time and sit back and wait to see if it is effective,” explained Dr. Jonathan Thomas, chair of the CIRM governing board, Imperial Valley News reported. “We have a mission to find treatments for patients in need. By trying several different approaches, taking several shots at goal at the same time if you like, we feel we have a better chance of being successful.”

The procedure hopes to replicate what occurred in the Berlin Patient, the only person to ever be “cured” of HIV. If proven to be as effective in the trials as it is on paper, the procedure may become the world’s first “functional cure” for HIV and AIDS.

Written by Dana Dovey at Medical Daily

 


There are 26 comments

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  1. thundernuts

    Wow.

    Big pharma is NOT gonna like this….

    It seems every few years there is an announcement of a promising possibility of a cure. As usual I’ll hope for the best, but remain cautiously optimistic.

  2. douglas mosier

    From reading between the lines of the article, the most important thing about this “functional cure” is that it would eliminate the need for expensive medications to keep from dying…….but it seems that it does nothing to rid the body of the virus, which means, theoretically, the patient would remain “poz” and also, more importantly, able to infect others. Yes, being able to live my life without worrying about ruining my liver and kidneys because of drug side effects or worrying about going bankrupt to stay alive would be fantastic. But we still would not be able to have sex the way we might wish to. But, hey, one step at a time!

  3. WALTER

    Douglas,
    You missed something.. Once the new cells with the gerenic mutation is put into the body, they reproduce new cells that will take over the body cause & the old ones to eventually die or not able to reproduce new HIV cells. If you can’t produce new HIV cells you are not positive, eventually the old HIV cells will die since they can’t reproduce.

  4. John

    My friend I’ve known for 12 years died yesterday from AIDS. He was 24 years old. I wish he got treatment right away and told someone he had it so they could help him. Today is very depressing and will be for a few weeks. I only wish a cure for HIV is coming soon.

  5. dean

    only thing is there is so much money in the drugs now the drug co are going to buy up the research and will it ever happen big money always rules

  6. drops

    i am hiv poz for 16 years. no meds no infections or any illness. i take care of myself by taking herbs and living an active lifestyle and vegan lifestyle. until a cure comes, i will take nothing to weaken any other system of my temple. i look forward to many years with my partner.
    if you smoke or do drugs of any kind, you will certainly need meds. hiv is nothing serious, just eat and live clean.
    a dormant virus is a virus. obly take the cure guys!

  7. Dr. Feelgood

    This is indeed promising but I believe the news last month (http://www.nature.com/nature/journal/v519/n7541/full/nature14264.html) about the 100% effectivity (at least in macaque monkeys) of the passive vaccine modality through similar gene-therapy induced genomic changes but attacking 2 different weak points in HIV (i.e. CD4 and CCR5 attachments) holds more promise in fending out any adaptive mutation by this notoriously protean virus. Another concern with this particular technique is how one would have to theoretically re-infuse the genetically modified T cells eventually after their life cycles have run their course (and to keep on doing so every time it happens) given that the changes are effected on fully mature cells and not on their stem cells, which would not have to be done theoretically with vaccination outside of a booster dose or two during a person’s lifetime, if it is even needed at all.

    But of course, this still needs to be celebrated if not only for it being realistically closer to be used clinically than the new one but also as an excellent bridge to more efficacious, efficient, safe and cost-effective modalities in the future.

    Additional sources:

    http://directorsblog.nih.gov/2015/02/24/study-suggests-alternative-approach-to-aids-vaccine/

    http://www.nytimes.com/2015/02/19/health/new-approach-to-blocking-hiv-raises-talk-of-an-aids-vaccine.html

    http://news.sciencemag.org/biology/2015/02/stopping-hiv-artificial-protein

  8. azzisblack

    Can someone send me info on what exactly tropical strains of HIV are?
    I’ve never heard of this
    I’m on A4A also

  9. Michael

    Thank you for posting this news.

    We’ve been working toward a gene therapy approach to curing or controling HIV in Seattle since 2011. http://www.defeathiv.org if you’re curious about our work.

    Still, it’s hard to reach many people to get them up to speed about the science so they can help advocate for the research and eventual clinical trials we will need to see the cure or control of HIV become a reality for all people living with the virus.

    So, thank you for posting this news about an HIV functional cure.

    May a cure for HIV that is accessible to everyone who needs it come within our lifetimes!

    Michael

  10. Leo

    @Douglas Mosier – Right now it’s just in clinical trials. If the method is further study and is approved we would be able to remove the virus from the body completely by giving the patient radiation (similar to cancer therapy) before administering the treatment. (Google “The Berlin Patient”)

  11. Scott

    Not a bad idea, but like silver fox mentioned what about cxcr4? And CCR5 delta 32 mutations come with their own problems. They are known to cause strokes. But not a bad idea

  12. Fred

    It sounds promising and is based on the same process that cured the Berlin Pateint Timothu Brown and so it’s based on something a bit more concrete than the usual promising research which doesn’t pan out.

    Cautious Optimism

  13. Mark

    Big Pharm HAS to be kept out of this! They are the modern day Robber Barons. Keeping the R&D funded at the research level can keep Pharm out and progress continuing. Trust Big Pharm to stall if they can.

  14. HSV 1 & 2

    Great. HIV will be cured soon. Herpes next please. Since, you know, that’ll be the next thing to spread around like an even bigger wildfire than it already is since it’s not curable.

  15. Robert

    My specialist in Dallas, TexAs says that there is a basic cure that is in the preliminary stages of being tested and approved . Even if there was a release of the drug it would become so expensive that we would not be able to really afford it. I hope that this is not truth and that we as a human race would be smarter than that to see a fellow brother suffer and die eventually.

    Staying in good health and excising on a regular basis and taking vitamins at an early age is wise in somewhat slowing the process down .


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