A team of researchers from UCSF and UC San Diego has identified an approved arthritis drug that is effective against amoebas in lab and animal studies, suggesting it could offer a low-dose, low cost treatment for the amoebic infections that cause human dysentery throughout the world.
Based on these results, the team has received Orphan Drug Status for the drug, known as auranofin, from the U.S. Food and Drug Administration, and has applied for approval to start clinical trials to treat both amebiasis and the parasite Giardia in humans.
The findings, which showed that auranofin inhibited growth of the parasite Entamoeba histolytica in lab tests as well as two rodent models of the disease, highlight the importance of screening existing drugs for new purposes, especially for neglected diseases, the researchers said. Findings will be reported in the June 2012 issue of Nature Medicine and were selected for advance online publication on the Nature web site, at www.nature.com.
The combination of an off-patent drug and decades of clinical safety data offers the possibility of providing a lower-cost solution worldwide with fewer side effects or risks of bacterial resistance than the current therapy, according to co-senior author James McKerrow, MD, PhD, a professor of pathology in the UCSF Sandler Center for Drug Discovery.
"When we're looking for new treatments for the developing world, we start with drugs that have already been approved," said McKerrow, who co-authored the paper with Sharon Reed, MD, of UC San Diego and first author Anjan Debnath, PhD, of UCSF. "If we can find an approved drug that happens to kill these organisms, we've leapfrogged the development process that goes into assessing whether they are safe, which also makes them affordable throughout the world."
Each year, 50 million people worldwide contract amebiasis through contaminated food or water,
|Contact: Kristen Bole|
University of California - San Francisco