UM researchers developing process to inhibit HIV

University of Miami researchers recently developed a molecular process that blocks HIV from spreading and infecting new cells.

It is a breakthrough that virologists Dr. Gunter Kraus and Dr. James Hnatatyszyn call the “scissors effect,” a term that describes a process inhibiting the virus’s ability to infect and replicate.

There is no known cure for HIV or AIDS, the later stages of the infection.

“HIV’s most well-known trait is its high mutation rate which generates new variants of the virus that are resistant to current drug therapies,” said Dr. Kraus, who has been working on this project for 20 years.

This newly-developed technology has the “ability to target and stop certain molecular functions, [making] a future application for this process clearly a vaccine development on the cellular level,” Kraus said.

This new vaccine is not a cure for AIDS, Kraus said, but it is a major breakthrough in potentially stopping the spread of the virus through new infection.

“Our discovery presents a major victory in the war against HIV,” Kraus said. His team’s recently-published findings in the January 2002 issue of Gene Therapy support Kraus’s claim.

The promise of their new research will likely take several more years to develop into anything like a vaccine for public consumption, and it is likely to be a sort of therapy treatment that must be administered over a course of time, Kraus said.

But there is hope.

One crucial step for this new technology to progress further is for UM to take action to secure the patents on the research so that Kraus and his associates can effectively seek out sources of funding from large pharmaceutical companies.

Private sector investors must first have a patent to secure financial interests before they will commit funding and researchers to a project.

Kraus also said that “a biochemist needs to make a stable, synthetic form of the EGS 560” -before this can become a viable form of treatment – which, “fortunately, [the EGS 560 molecule] is small, and should therefore be easily replicated.”

If a patent were to be secured quickly, then pharmaceutical companies could begin work on making this a readily available preventative mechanism, Kraus said.

This entire predicament takes on a real seriousness when one considers the statistic that over 14,000 people daily are infected by the HIV virus.

If UM does not pursue the patents within the next year, the patent rights will fall to the researchers, who hopefully will privately pursue them, bringing this new valuable medical breakthrough to as many people as possible, as soon as possible.

While the research team waits for their patents to clear, they are continuing research that tests EGS 560 against different, more complicated clades, or types of the HIV virus. Their results are very promising, but more research and funding is needed to advance the process further.

Funding is crucial for widespread testing and development, as expensive technologies are needed to store and utilize samples, and pure concentrated forms of the HIV virus are used – therefore, the team has to use a Bio-Safety Level 3 laboratory.

The basic premise behind the research is simple.

Within a cell is RNA, the message component that makes cells act and form uniquely-essentially providing the information to manufacture proteins.

RNA is also the first place that the HIV virus infects the body.

After infecting the body’s RNA, the virus then proceeds to invade the DNA, the very cellular code that maps out the creation of further cells.

Once the DNA gets infected by HIV, then all of the newly-formed cells are infected too, spreading the virus throughout the body-a viral attack that the body cannot fight because the HIV virus attacks the CD4 T cells that are the source of the human immune response.

Human CD4 T cells are the cells that allow the body to fight disease, and the destruction of the body’s CD4 T cells by HIV is what cripples the immune system.

Once AIDS sets in, a patient’s ruined immune system is unable to fight any sort of disease or infection.

Kraus and Hnatyszyn used a natural cellular mechanism, called RNase P, that cleaves RNA, preventing HIV transmission to the DNA.

RNase P has an interesting external guide sequence (EGS), which can be targeted to recognize and interfere with almost any cellular process.

The researchers designed a specific type of RNase P – EGS 560 – which targets the body’s CD4 T cells.

The cells treated with EGS 560 block HIV infection by blocking the virus’ admission into the RNA.

If that process is interrupted, HIV will not progress to AIDS.

In their research tests, “the CD4 T cells treated with the EGS 560 were unable to be infected and remained healthy for more than 30 days,” Kraus said, and “after years of development we are optimistic that our discovery will provide a potent weapon in the fight against HIV.”

RNase P’s special cleavage properties were first discovered in 1989 by Drs. Sidney Altman and Thomas Cech, who were awarded the 1989 Nobel Prize for Chemistry.

Dr. Kraus points out that not only does RNase P hold promise for HIV prevention in high-risk populations, but “in addition, since it is a relatively simple molecular process, it may also have the potential to eradicate cancer cells, and perhaps produce vaccines to combat pathogenic viruses other than HIV.”

This news suggests that the creation of EGS 560 may just be the tip of the iceberg in an exciting new realm of medical technology in disease prevention.

“We hope this research will have a snowball effect on research in this area,” Kraus said.