Made in Nebraska: Potential Cure to HIV Could be in Reach Thanks to Nebraska-Funded UNMC Research

Made in Nebraska Potential Cure to HIV Could be in Reach Thanks to Nebraska-Funded UNMC Research

Investigators from Nebraska Medical Center and Temple University, through a combination of drugs and novel gene-editing therapy, successfully eliminated the HIV virus in living animals for the very first time this past summer. As it turns out, a New York transplant in 1992 planted the seed in a rich fertile Nebraska research ecosystem, leading to a worldwide research hub to grow and prosper.

Top 100 Scientific Breakthrough of 2019

According to Altmertic, which tracks dissemination of research online, noted the development was one of the top 100 scientific breakthroughs in 2019. After all, just a decade ago, HIV and AIDs was a near death sentence, and today, science has advanced considerably to where often the disease can be managed. However 1.1 million people residing in America still die from the disease annually.

A Cure on the Horizon?

Chris Dunker of the Lincoln Journal Star recently reported that Howard Gendelman, University of Nebraska Medical Center (UNMC), chair of pharmacology and experimental neuroscience, recently hinted that another major milestone in the battle against the disease was “on the horizon” during a University of Nebraska Board of Regents meeting last month. He reported to the Board of Regents that “We’re getting very close to moving to human trials.”

UNMC: A Worldwide Hub for HIV/AIDS Clinical Research

As it turns out, Dr. Gendelman, who was actively treating HIV patients in New York City back in the early 1980s, moved to UNMC in 1992. He has been a key agent to establish a foundation and framework for the building of a world-class HIV research operation in America’s heartland. As the local press revealed, UNMC attracts on an annual basis world-renowned researchers, grad student and various experts from virology and immunology to chemistry and pharmacology.

Because of that momentum, they also secured a number of large federal research grants not to mention support from the state legislature, university, community and local citizens that care and contribute. They have also inked deals abroad with entities focused on this research.  Gendelman himself thinks of this unfolding collective effort as an orchestra where each player contributes crucial elements, making the symphony work.

The Therapy

Known as LASER ART—the long-acting, slow-effective release antiretroviral therapy—developed and manufactured at a specialized UNMC facility could be ready for clinical trials within a year or two. The team leveraged an existing drug as well as pioneering gene editing techniques. The existing HIV drug was utilized by the UNMC team as they modified its chemical structure transforming it into a nanocrystal effectively doubling the drug’s “half-life”—the length of time it can be effective.

Dr. Benson Edagwa, UNMC assistant professor of pharmacology, got involved, “encasing multiple nanocrystals inside a fatty coating, which allows it to easily penetrate the membranes of cells infected with HIV and facilitates the slow release in the body,” reports the Lincoln Journal Star.

Now with a “lipid tail,” the drug actually digs deeper into the body’s tissue where HIV likes to hide out—areas such as lymph nodes or bone marrow. And since they have engineered a longer life, the drug delivers antiretroviral therapy in more precise ways for longer periods of time, hence suppressing the HIV virus growth.

One benefit of this preclinical experimental treatment is that it can potentially be taken once or twice a year rather than every day. Researcher Edagwa, who originates from Kenya (where 6% of the total population lives with HIV), notes this treatment could have a profound impact on his home country.

The Study

The UNMC-led team leveraged theranostics—combining therapeutics and diagnostics—and hence successfully tracked the drug’s destination by, utilizing an MRI, studying mice with human DNA who were administered the treatment. The researchers could track where the drug reached within the mice by viewing an iron tag on each nanoparticle, which manifested as a black stain on the image.

Investigator Comment

Dr. Gendelmen notes, “It’s not simply just demonstrating we can extend the half-life” and continued, “But can we target the medicine to where the virus continues to grow and suppress it to the greatest extent we can possibly achieve.”

Funding: Nebraskans Chip In

Apparently, several biopharmaceutical companies were interested in potentially licensing or purchasing the experimental therapy but Gendelman told the regents of the university that such a deal would lead to a lengthy timetable dealing with specialized manufacturing requirements. Because they want to expedite this therapy, they are keeping it in house for now. But how did they do it? How were they able to possess the specialized manufacturing capability?

The Nebraska Nanomedicine Production Plant: Advanced Experimental Drugs Made in Nebraska

The funding for this study (and associated lab and tissue culture facility) was vital for its success. Nebraska tax payers helped contribute to this globally significant therapy. The Nebraska Research Initiative, part of an annual $11.4 million state appropriation during the 1980s and as they received $2 million of this precious capital, they creatively and pragmatically renovated a tissue culture facility in the Lied Transplant Center into a specialized manufacturing facility called “the Nebraska Nanomedicine Production Plant,” which opened earlier in 2019.

This is one of the few such nanomedicine manufacturing facilities operated by a university in America. Instituting Good Manufacturing Practice, or GMP, this allowed the team to produce the HIV drug consistently and with quality. Additionally, they can make small batches economically—sufficient for small clinical trials. This enabled a fast track for FDA approval.

Their facility very well could be a “springboard for other novel infectious and metabolic disease drugs to be developed in Nebraska.”

Clinton Health Access Initiative (CHAI)

Based on UNMC’s successful moves they attracted CHAI, which focuses on helping researchers navigate the labyrinth of regulatory requirements worldwide as well as translating experimental therapies into commercial success in the developing world. With a global focus, they seek to reduce the burden of disease in low-and middle-income countries. They support their partners to strengthen the capabilities of governments and the private sector to create and sustain high-quality health systems. They were founded in 2002 to help save the lives of millions of people living with HIV and AIDs in low-and-middle income countries.

Lead Research/Investigators

Howard Gendelman, University of Nebraska Medical Center (UNMC) chair of pharmacology and experimental neuroscience

Dr. Benson Edagwa, UNMC assistant professor Pharmacology

Call to ActionTrialSite News will track the movement of this preclinical research carefully. Sign up for the Daily Digest and receive updates. Are you a biopharma drug company interested in partnering with the new Nebraska Nanomedicine Production Plant? Contact Adam Szlachetka, Operations Manager