December 08, 2009

Gene Therapy Success Blocking Virus Replication and Stem Cell Successes

A Danish drug tested on San Antonio chimpanzees is showing promise as a new gene-based therapy that fights hepatitis C and potentially other diseases.

The drug, developed by biopharmaceutical firm Santaris Pharma doesn't directly fight the virus, which is quick to develop drug resistance.

Instead, it targets and locks down a gene in the liver that the virus needs in order to replicate, said Robert Lanford of the Southwest Foundation for Biomedical Research.

The two chimpanzees that received higher doses of SPC3649 had a 350-fold drop in the viral levels in the blood and liver, Lanford said. The animals, which are the only other creatures besides humans that can be infected with HCV, were given one dose a week for 12 weeks. Not only did they not develop resistance to the drug, which usually happens with days, Lanford said, but the virus stayed suppressed for several months after researchers stopped the therapy.

“Not only did we knock down the virus, but we saw an improvement in the liver itself because we could keep the virus down so long,” he said.

It's also a promising platform for gene therapy, a technology that has been frustrating scientists for some time, Lanford said.

An estimated 3.2 million people in the United States have hepatitis C, or HCV, and many don't know it, according to the Centers for Disease Control and Prevention. It can range from a mild illness lasting a few weeks to a lifelong disease that may not become apparent for years, and can lead to cirrhosis or liver cancer.


2. Stroke-damaged brains could be repaired within 5-10 years using adult stem cells from teeth, according to one of Australia's leading stroke physicians who is pioneering new research in this field.

Associate Professor Simon Koblar from the University of Adelaide and The Queen Elizabeth Hospital is leading a research project that shows dental pulp stem cells extracted from teeth may prove far more beneficial for brain repair than other types of stem cells.




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