Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Researchers explore gene treatment to obliterate HIV before it does damage

26.05.2004


Volunteers now sought for Stanford trial



Doctors may someday have a new way to combat AIDS by going straight to the source: destroying the virus before it has a chance to wreak havoc on a patient’s immune system.

Thomas Merigan, MD, the George and Lucy Becker Professor of Medicine in infectious diseases at Stanford University School of Medicine, is seeking volunteers for a study to test a possible method of empowering an infected person’s own cells to destroy HIV as it enters the cell. The process involves removing the patient’s stem cells - the ones in the bloodstream that form the different immune system cell types that HIV infects, such as T cells and macrophages - and inserting a gene that produces an HIV-obliterating enzyme.


"This is a broad-spectrum treatment that could integrate well with other therapies as the disease progresses, as it will in all patients eventually," said Merigan, who is collaborating with other researchers at UCLA and St. Vincent’s Hospital in Sydney, Australia, under the sponsorship of Johnson & Johnson Research. "The goal is to genetically engineer the cells and make them resistant to infection."

The strategy developed by Merigan and his colleagues relies on the fact that the genetic information of HIV is encoded in RNA rather than DNA. Enzymes called ribozymes can chew up RNA at very specific sites, rendering it inactive. If a ribozyme specific to HIV RNA were present inside the cells that the virus infects, then it could constitute a first-line defense against the invasion. Even if HIV did make it into the cell and replicated, the ribozyme could potentially cleave the HIV RNA at various steps during the virus’ life cycle. With this unique approach to warding off HIV infection, the enzyme produced by a person’s own vulnerable cells could demolish the critical genetic instructions of HIV while not affecting his or her RNA.

"With this technique, we’d like to be able to offer an additional approach for patients who in their own minds appear to be doing quite well," said Merigan. "We know there is a sufficient risk that patients will eventually need more than the 19 drugs currently available."

A total of 70 patients will be enrolled at three sites for this stage of the testing. The participants will be randomly assigned to receive either the ribozyme gene therapy or a dummy gene. Johnson & Johnson Research produces the reagents used for introducing ribozyme into the blood stem cells.

In the field of AIDS treatment, there is recognition that an approach like the one Merigan is testing would be a huge development in the ability to combat HIV. David Evans, an information and advocacy associate for Project Inform, a San Francisco-based national nonprofit working to end the AIDS epidemic, said, "Experiments of this type fall perfectly in line with Project Inform’s agenda to refocus researchers toward a cure for HIV rather than indefinite treatment with costly antiviral drugs that consistently lead to drug resistance and long-term toxicities."

Martin Delaney, founding director of Project Inform, said he applauds the researchers for their persistence and continued interest in developing new treatments for HIV/AIDS, but added, "It is too early to comment on the potential of this particular experiment and, even if successful, it will be several years before the results can be turned into a widely accessible therapy."

Merigan said that similar studies testing the strategy of using gene therapy in more than 1,200 patients have proven the technique to be safe. The safety studies for the current trial looked at a total of 14 patients and found no safety concerns.

Merigan discussed a scare a few years ago when two babies undergoing gene therapy developed leukemia, which was successfully treated. He noted that the HIV trial had been delayed for two years while the trial’s safety design was examined by the National Institutes of Health, the U.S. Food and Drug Administration and local review boards. He said the verdict was that the immune system of adults with HIV differs greatly from that of babies, whose immune systems are not fully formed, and that they should not face the same risk.

The criteria for potential study participants are strict at this stage: men and women with HIV who are between ages 18 and 45, on only the first or second round of anti-retroviral therapy, a relatively low amount of virus in the blood (less than 50 particles per milliliter of blood) and a reasonably high level of T cells (300 cells per microliter of blood or greater). Pregnant women or those with any AIDS-related illnesses will not be included in this trial. Those who are interested should call (650) 723-6231.


Others in the study team in the division of infectious diseases at Stanford include Andrew Zolopa, MD, associate professor of medicine; Michael Harbour, MD, clinical instructor; Debbie Slamowitz, study coordinator for clinical trials; Jane Norris and Sandy Valle, physician assistants; Patricia Cain and Sylvia Stoudt, staff nurses; and Mark Winters, research assistant.

Stanford University Medical Center integrates research, medical education and patient care at its three institutions - Stanford University School of Medicine, Stanford Hospital & Clinics and Lucile Packard Children’s Hospital at Stanford. For more information, please visit the Web site of the medical center’s Office of Communication & Public Affairs at http://mednews.stanford.edu.

PRINT MEDIA CONTACT: Amy Adams at (650) 723-3900 (amyadams@stanford.edu)
BROADCAST MEDIA CONTACT: M.A. Malone at (650) 723-6912 (mamalone@stanford.edu)

Amy Adams | EurekAlert!
Further information:
http://mednews.stanford.edu

More articles from Health and Medicine:

nachricht Penn vet research identifies new target for taming Ebola
12.01.2017 | University of Pennsylvania

nachricht The strange double life of Dab2
10.01.2017 | University of Miami Miller School of Medicine

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Interfacial Superconductivity: Magnetic and superconducting order revealed simultaneously

Researchers from the University of Hamburg in Germany, in collaboration with colleagues from the University of Aarhus in Denmark, have synthesized a new superconducting material by growing a few layers of an antiferromagnetic transition-metal chalcogenide on a bismuth-based topological insulator, both being non-superconducting materials.

While superconductivity and magnetism are generally believed to be mutually exclusive, surprisingly, in this new material, superconducting correlations...

Im Focus: Studying fundamental particles in materials

Laser-driving of semimetals allows creating novel quasiparticle states within condensed matter systems and switching between different states on ultrafast time scales

Studying properties of fundamental particles in condensed matter systems is a promising approach to quantum field theory. Quasiparticles offer the opportunity...

Im Focus: Designing Architecture with Solar Building Envelopes

Among the general public, solar thermal energy is currently associated with dark blue, rectangular collectors on building roofs. Technologies are needed for aesthetically high quality architecture which offer the architect more room for manoeuvre when it comes to low- and plus-energy buildings. With the “ArKol” project, researchers at Fraunhofer ISE together with partners are currently developing two façade collectors for solar thermal energy generation, which permit a high degree of design flexibility: a strip collector for opaque façade sections and a solar thermal blind for transparent sections. The current state of the two developments will be presented at the BAU 2017 trade fair.

As part of the “ArKol – development of architecturally highly integrated façade collectors with heat pipes” project, Fraunhofer ISE together with its partners...

Im Focus: How to inflate a hardened concrete shell with a weight of 80 t

At TU Wien, an alternative for resource intensive formwork for the construction of concrete domes was developed. It is now used in a test dome for the Austrian Federal Railways Infrastructure (ÖBB Infrastruktur).

Concrete shells are efficient structures, but not very resource efficient. The formwork for the construction of concrete domes alone requires a high amount of...

Im Focus: Bacterial Pac Man molecule snaps at sugar

Many pathogens use certain sugar compounds from their host to help conceal themselves against the immune system. Scientists at the University of Bonn have now, in cooperation with researchers at the University of York in the United Kingdom, analyzed the dynamics of a bacterial molecule that is involved in this process. They demonstrate that the protein grabs onto the sugar molecule with a Pac Man-like chewing motion and holds it until it can be used. Their results could help design therapeutics that could make the protein poorer at grabbing and holding and hence compromise the pathogen in the host. The study has now been published in “Biophysical Journal”.

The cells of the mouth, nose and intestinal mucosa produce large quantities of a chemical called sialic acid. Many bacteria possess a special transport system...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

12V, 48V, high-voltage – trends in E/E automotive architecture

10.01.2017 | Event News

2nd Conference on Non-Textual Information on 10 and 11 May 2017 in Hannover

09.01.2017 | Event News

Nothing will happen without batteries making it happen!

05.01.2017 | Event News

 
Latest News

Water - as the underlying driver of the Earth’s carbon cycle

17.01.2017 | Earth Sciences

Interfacial Superconductivity: Magnetic and superconducting order revealed simultaneously

17.01.2017 | Materials Sciences

Smart homes will “LISTEN” to your voice

17.01.2017 | Architecture and Construction

VideoLinks
B2B-VideoLinks
More VideoLinks >>>