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Utah scientists develop drug that could prevent, treat HIV

Technique to create treatment may work in battling COVID-19, researcher says

Dr. Amanda Smith, University of Utah, is shown testing drug candidates against HIV in a biosafety cabinet. University of Utah researchers say they have developed a drug that appears to prevent HIV and treat the virus with fewer side effects than therapies currently in use. After successful tests in primates, the drug will soon undergo human trials.
Dr. Amanda Smith, University of Utah, is shown testing drug candidates against HIV in a biosafety cabinet. University of Utah researchers say they have developed a drug that appears to prevent HIV and treat the virus with fewer side effects than therapies currently in use. After successful tests in primates, the drug will soon undergo human trials.
Dr. Michael Kay

SALT LAKE CITY — University of Utah researchers say they have developed a drug that appears to prevent HIV and treat the virus with fewer side effects than therapies currently in use.

The injectable drug, called CPT31, is headed for human trials after successfully undergoing tests in primates.

The first of its kind, the drug blocks HIV from entering cells to either prevent an infection or to treat an existing infection and keep it from spreading within a patient’s body, said Dr. Michael Kay, a senior author of the study and a University of Utah Health professor of biochemistry.

It’s based on a newly discovered type of compound called a D-peptide, which is a mirror image of a natural peptide that exists in our bodies and has a longer lifespan than others, Kay said. The drug has been in development at the university for five years.

“It’s exciting because it works differently than any of the other drugs that are commonly used to treat HIV, and so it would be helpful for patients who have drug-resistant HIV, which is a common problem with the existing drugs,” Kay explained.

“This is one of the very first of this type of drugs to make it this far, and is the only example we’re aware of that’s being used against HIV so far,” he said.

While the curve of new HIV cases in the U.S. has fallen since 2010, Salt Lake County’s rate has remained flat at around seven cases per 100,000 people per year. Last year, 79 new cases of HIV were reported in the county, according to Salt Lake County Health Department data. Likewise, Utah has not seen a drop in its rate of new cases.

The numbers in Utah, however, are relatively small, Kay noted, making it difficult to measure the trends. But his team is working with the university’s AIDS prevention clinic, which serves a large portion of Utah’s HIV patients or those at risk of getting HIV, to find out what they need. If the drug proves safe and effective in humans, they would work with patients to work to “get down to zero new HIV infections,” Kay said.

“Which really does seem like an achievable goal, but the more tools we have to do that, the easier that will be to achieve,” the doctor said.

The current therapy used by HIV patients consists of a “drug cocktail,” which often has serious side effects and requires daily pills, Kay noted. CPT31 enables new formats of treatment, including long-acting versions to prevent patients from needing to take pills daily.

“We’re envisioning that this might be a suitable component as an injectable cocktail that patients could get every month, maybe even every three months or even less frequent,” he said.

Because it is created through a chemical process, the drug is also less expensive to make than antibody treatments that are undergoing clinical trials for HIV treatment, Kay noted.

The drug will be applicable to multiple strains of HIV, Dr. Sarah Apple, postdoctoral fellow, noted.

“In this case, this drug is targeting a part of the virus that doesn’t change very much as far as we’ve seen so far, and so that means that it would have a really broad reach for HIV specifically,” Apple said.

Dr. Sarah Apple, University of Utah, stands next to a stack of dozens of peptides made during a drug discovery process. University of Utah researchers say they have developed a drug that appears to prevent HIV and treat the virus with fewer side effects than therapies currently in use. After successful tests in primates, the drug will soon undergo human trials.
Dr. Sarah Apple, University of Utah, stands next to a stack of dozens of peptides made during a drug discovery process. University of Utah researchers say they have developed a drug that appears to prevent HIV and treat the virus with fewer side effects than therapies currently in use. After successful tests in primates, the drug will soon undergo human trials.
Dr. Michael Kay

When the drug was given to macaque monkeys and they were exposed to a simian form of HIV called SHIV, the monkeys were protected and never developed signs of infection, the researchers noted.

In monkeys that already had untreated SHIV infections, researchers said they had a significantly lower level of the virus in their bloodstream after 30 days.

Kay said human trial will help researchers determine who will benefit most from the drug. They believe CPT31 alone could potentially prevent the virus in humans at risk of HIV, but will be needed with a cocktail of therapies in those who already have the virus.

The extensive research put into the drug has come with other benefits, the researchers said.

Now they are using the technique they developed when creating the drug to target several other viruses — including the coronavirus. They can do that because many viruses — including SARS-CoV-2 — have mechanisms of entry into a cell similar to HIV, Apple explained.

Now that the techniques, which took years to develop, are in place and validated, Kay said, “we’re really excited about applying those validated methods and technologies to a new virus like for COVID-19. And what we think will be dramatically faster than the first time, with HIV.”

Apple said that research, which she is leading, has been moving quickly despite restrictions during the pandemic.

Researchers believe there will be future strains of coronavirus and want to be prepared for that, Kay said. Using the methods they’ve created, they hope to develop a drug that can be stockpiled and ready to go if there’s another outbreak of viruses in the coronavirus family.

U. scientists led the study in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), Beth Israel Deaconess Medical Center in Boston, and Navigen, Inc.