New drug technology produces marked improvement in hepatitis C therapy in animals

December 03, 2009

In a dramatic finding, a new drug for hepatitis C virus (HCV) infections that targets liver cells produced a substantial drop in blood levels of the virus in animals and continued to work up to several months after treatment, say scientists at the Southwest Foundation for Biomedical Research (SFBR) in San Antonio.

The drug, SPC3649, was developed by the biopharmaceutical firm Santaris Pharma A/S in Denmark using their proprietary nucleic acid chemistry called "locked nucleic acid" or LNA. SPC3649 is a nucleic acid, or DNA-based drug, that captures a small RNA molecule in the liver, called microRNA122, that is required for HCV replication.

"Our collaboration with Santaris Pharma proved that the drug worked exceptionally well in treating HCV infections in chimpanzees," said SFBR's Robert Lanford, Ph.D., the lead author on the study appearing in the December 3, 2009 issue of Science Express, which provides rapid electronic publication of research papers ahead of print in the journal Science.

SPC3649 also showed a high barrier to resistance, a major problem with therapies that directly target the virus. Moreover, this proof of concept study suggests that the technology might also prove useful in treating many other diseases such as HIV, cancer, and inflammatory diseases.

HCV infections affect 170 million people worldwide and progress over years to end stage liver disease including cirrhosis and liver cancer. In the United States, 4 percent of the adult population is chronically infected with HCV. The only U.S. Food and Drug Administration approved therapy is interferon and ribavirin, which is very toxic, requires 48 weeks of treatment and works in less than half of patients. Scientists believe that a cocktail of three drugs will be needed to prevent antiviral resistance and replace interferon.

The new therapy could potentially replace interferon in future cocktails, since it provides a high barrier to resistance. "This antiviral could be used alone to treat disease progression and there are indications that it can convert interferon non-responders to responders, so that non-responders to the current therapy could be treated with the combination of this drug with interferon," Lanford said. It may also be a good therapy to use after transplant, since it may help suppress HCV in the new liver. It has no toxic or adverse reactions and this is critical in the transplant setting.

In the study, four HCV chronically infected chimpanzees were treated with the new antiviral. The two animals that received the higher dose had a drop in viral levels in the blood and liver of 2.5 orders of magnitude or approximately 350 fold. The surprising findings were the lack of antiviral resistant mutants and the fact that the therapy continued to work for several months after dosing stopped.

In one advance, the new study was a critical proof of concept that the LNA technology could work for HCV. It proved that miR-122 was truly required for HCV replication in an animal infected with HCV. Previously the role of miR-122 in HCV replication had only been shown in tissue culture. A second advance was the finding that LNA therapy could work against an important disease model, suggesting that the new technology can be applied to other diseases.
Lanford can be reached through Joe Carey, SFBR's Vice President for Public Affairs at 210-258-9437.

SFBR is one of the world's leading independent biomedical research institutions dedicated to advancing health worldwide through innovative biomedical research. Located on a 200-acre campus on the northwest side of San Antonio, Texas, SFBR partners with hundreds of researchers and institutions around the world, targeting advances in the fight against cardiovascular disease, diabetes, obesity, cancer, psychiatric disorders, problems of pregnancy, AIDS, hepatitis, malaria, parasitic infections and a host of other infectious diseases. For more information on SFBR, go to

Texas Biomedical Research Institute

Related Virus Articles from Brightsurf:

Researchers develop virus live stream to study virus infection
Researchers from the Hubrecht Institute and Utrecht University developed an advanced technique that makes it possible to monitor a virus infection live.

Will the COVID-19 virus become endemic?
A new article in the journal Science by Columbia Mailman School researchers Jeffrey Shaman and Marta Galanti explores the potential for the COVID-19 virus to become endemic, a regular feature producing recurring outbreaks in humans.

Smart virus
HSE University researchers have found microRNA molecules that are potentially capable of repressing the replication of human coronaviruses, including SARS-CoV-2.

COVID-19 - The virus and the vasculature
In severe cases of COVID-19, the infection can lead to obstruction of the blood vessels in the lung, heart and kidneys.

Lab-made virus mimics COVID-19 virus
Researchers at Washington University School of Medicine in St. Louis have created a virus in the lab that infects cells and interacts with antibodies just like the COVID-19 virus, but lacks the ability to cause severe disease.

Virus prevalence associated with habitat
Levels of virus infection in lobsters seem to be related to habitat and other species, new studies of Caribbean marine protected areas have shown.

Herpes virus decoded
The genome of the herpes simplex virus 1 was decoded using new methods.

A new biosensor for the COVID-19 virus
A team of researchers from Empa, ETH Zurich and Zurich University Hospital has succeeded in developing a novel sensor for detecting the new coronavirus.

How at risk are you of getting a virus on an airplane?
New 'CALM' model on passenger movement developed using Frontera supercomputer.

Virus multiplication in 3D
Vaccinia viruses serve as a vaccine against human smallpox and as the basis of new cancer therapies.

Read More: Virus News and Virus Current Events is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to