HIV Wiped Out? Nebraska Researchers Say They've Taken a Big Step

July 2, 2019, 7:30 p.m. ·

Dr. Howard Gendelman, researcher at the University of Nebraska Medical Center. (Photo courtesty of UNMC)

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Researchers at The University of Nebraska Medical Center in Omaha and Temple University say they’ve taken a critical step in finding a way to eliminate the HIV infection. A combination of therapies in a new study has wiped-out the virus in so-called “humanized” lab mice whose immune systems mirror those in humans. Dr. Howard Gendelman is a researcher at UNMC and is a senior investigator on the new study. He spoke with NET’s Jack Williams.

NET News: This is a big day for research at the University of Nebraska Medical Center and Temple University. Can you briefly explain the combination of therapies that appear to have eliminated the HIV infection in mice?

Dr. Howard Gendelman: What we’ve done is to transform the anti-retro viral drugs from a water-soluble, short half-life medicine to a nanocrystal and encased it into a nanoformulation that can be given infrequently and is long-lasting over a period of months up to as long as a year. These nanocrystals can reach lymphoid tissues, like the lymph nodes, the spleen and the gut, as well as the brain, in very high concentrations over long periods of time.

NET News: When we talk about humanized mice, why is that so important in this research?

Dr. Gendelman: HIV has a proclivity to infect only human cells and some non-human primates, so if we were to use small animal models that mirror disease or develop therapies, we would have to find a way to place human cells in the background of a small animal. We’ve been able to accomplish that task by taking an immunodeficient animal, an immunodeficient mouse that is incapable of rejecting a graft and repopulating that animal’s immune system with human cells. So essentially we have a mouse and a human immune system in a single model.

NET News: How confident are you that this combination therapy will even work in humans?

Dr. Gendelman: The confidence is only as strong as the data. Our first step is actually to take this into other species, use animal models that are more reflective of the human condition, of the size, the weight, the shape and the metabolic structure and configuration of drug metabolism and what we call affirm or confirm the data sets that we have done in mice in other species. The second thing is that there is a lot more virus in a human than there is in a mouse. No matter what we do or how we repopulate these mice, it can’t reflect the actual mechanism of disease and viral dissemination and immunology and all the characteristics of HIV infection are different in a human that must be mirrored if we are to move that forward, so those kinds of studies must proceed.

NET News: One of the key parts of this research is so-called CRISPR technology. How has this gene-editing capability changed the game for researchers like you and your team?

Dr. Gendelman: CRISPR technology is a revolution in bio-medical research. I think we all understand that it’s not simply for HIV. It’s for metabolic diseases, it’s for developmental diseases, it’s for genetic disorders, neurodegenerative and degenerative as well as a variety of infectious and inflammatory schemes that could be affected by gene manipulation at different times under different conditions with different approaches. So I suspect we’re going to hear a lot more about CRISPR in the time ahead because of what this technology can do and the impact it has on biomedical research.

NET News: How much closer are we now to finding a cure for HIV?

Dr. Gendelman: I do not have an answer of how long or how effective or how excited or what I think. There’s still a lot more research to be done. But what I can say is this research does provide proof of concept. It does tell us that we have it in our power to eliminate HIV from an infected animal, infected person. The technology exists. How we develop that technology, how effective that technology is, what our approach is, how significant or the end-organ of off-target we call, adverse events, that would preclude development, we don’t know yet. But we do know that it is possible through this research and research like it to eliminate HIV from an infected animal. Where this goes next will require a significant amount of research to move the science to the next level.