Combination Major Advance: Gene Therapy for HIV Passes Phase 2 Clinical Trial

The therapy aims to stop HIV re-producing

A one-time gene therapy that puts an anti- HIV RNA weapon into blood cells is safe and, in higher doses and stronger form, could make the body resist the AIDS virus, a clinical trial suggests.

This “major advance in the field” is the largest clinical trial ever to test genetically altered cells in humans, say UCLA researcher Ronald T. Mitsuyasu, MD, and colleagues.

“This study indicates that cell-delivered gene transfer is safe and biologically active in individuals with HIV and can be developed as a conventional therapeutic product,” the researchers report in the Feb. 15 advance online issue of Nature Medicine.

The treatment calls for patients to get shots of a growth factor that stimulates growth of white blood cells. Then the cells are taken from their blood. Blood stem cells are separated out and put in cell culture dishes.

In the culture, the patients’ own blood stem cells are infected with OZ1, a genetically engineered mouse virus that gives them an anti-HIV gene. This gene encodes an RNA molecule called a ribozyme, which specifically targets and inactivates HIV genes.

Once equipped with the anti-HIV gene, the blood stem cells are transfused back into the patient. The idea is for these stem cells to home in to the bone marrow and populate it with HIV-resistant T cells. As the older T cells die off or are killed by HIV, more and more of the body’s T cells should be HIV resistant.

Now that researchers have shown this kind of gene therapy can work, future treatments will increase the dose, improve homing to the bone marrow, and carry an even more powerful anti-HIV gene. And in the future, patients would be treated before starting anti-HIV drugs.

This gene therapy boosting or altering of blood cells could also be effective in treating cancer.

LIFT is an investigational new cancer treatment
that will transfer naturally-occurring cancer-killing activity (CKA) in the granulocytes of a selected donor into the body of a cancer patient.

Granulocytes are a category of white blood cells characterised by the presence of granules in their cytoplasm.

BBC news had more coverage of the gene therapy against HIV clinical trials.

He said: “Gene therapy has the potential of needing only a one-time or infrequent administration of product and would allow the patients to control their own HIV internally without the need for continuous drug therapy.

“While this treatment is far from being perfected, it is not yet as effective or as complete as current antiretroviral therapy in controlling HIV, the study did show proof of concept that inserting and administering a single anti-HIV gene in the patients’ own blood stem cells and giving it back to them could reduce viral replication to some degree when anti-HIV medications are stopped.”

However, Professor Mitsuyasu said long-term follow up was needed to ensure the therapy was safe.

Keith Alcorn, of the HIV information service NAM, said: “The viral load responses in this study were very modest, and for any other sort of product would not justify going forward.

“However, the researchers have shown enough of an effect for us to be hopeful that a gene therapy approach to HIV treatment might eventually deliver effective treatments for the disease.”

HIV Prevention: Researchers are enrolling thousands of participants around the world in clinical trials in a massive effort to test whether a once-daily pill can prevent HIV. Cassandra Willyard explores why they are optimistic the strategy will work and why it might be difficult to implement.

0 thoughts on “Combination Major Advance: Gene Therapy for HIV Passes Phase 2 Clinical Trial”

  1. Very useful in the tar sands and shale oil fields. Energy independence!

    Still, should gradually wean ourselves away from excess reliance on hydrocarbon transportation fuels.

    JP Straley

  2. bw, Could the build up of charge on the ion grid be dissipated using an electron gun such as seen here:

    I thought that the problem was the wearing down of the grid as the ions strike it on the way out of the drive.

    Does the slowness of the acceleration really matter. For example an it is said about Deep Space 1 that:

    “The effect of the gentle thrust slowly builds up, eventually attaining speeds far beyond the reach of conventional propellants”.

    Also, what is it about VASIMIR which ultimately gives it greater ultimate speed?

  3. For ion drives, I believe the power source is not the limiting factor. It is things like build up of charge on the ion grid.

    Ion drives could get up to 100 km/s. The issues are the slow acceleration and problems scaling beyond not just the power source but other parts.

    Vasimr scales better than ion drives in terms of acceleration, ultimate speed and in terms of power.

  4. What would 100 MW do for us in terms of final speed for a space craft if we could use that to accelerate ions? Ion propulsion accelerates ions to 100,000 km/hr but if using some sort of accelerometer we could get much higher than that. But suspect this would be difficult.

  5. 8 tons is just the reactor “hot-tub” as I read it, roid.

    When are nuclear power producers, proponents and innovators going to realise that elaborate over-precaution makes people nervous? Like sirens, like KI pills, like alerts based on trivia? The uranium hydride battery-reactor does not, not, NOT need to be buried deep underground. The maximum burial needed is to have the top of the enclosing chamber flush with the surface, but even that is probably more expensive than building above ground without changing the safety/security by any noticeable degree.

  6. Your figures for the Uranium Hydride reactor – assuming the whole system is included in that 8 tons – then that’s a 16x greater power density (by weight) than the Safe400 reactor.


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