Teaching old drugs new tricks at the Small Molecule Discovery Center: arthritis drug could treat dysentery

The UCSF School of Pharmacy’s Small Molecule Discovery Center (SMDC) provided a key assist to researchers seeking to repurpose existing drugs to treat the worldwide scourge of amoebic dysentery.

In the June, 2012 issue of Nature Medicine (and online in advance on Nature’s web site), researchers from seven labs including the UCSF Sandler Center for Drug Discovery and the SMDC (which is administered by the Department of Pharmaceutical Chemistry) report that an inexpensive off-patent drug for rheumatoid arthritis, auranofin, is 10 times more potent than the current treatment against a particular amoeba that is a key cause of human dysentery. Such gastrointestinal infections affect up to 50 million people and cause about 70,000 deaths annually, making it the fourth leading cause of mortality from protozoan infections worldwide.

Michelle Arkin, PhD, SMDC associate director for biology and faculty member in the Department of Pharmaceutical Chemistry, and Steven Chen, a specialist at the SMDC, worked with Anjan Debnath, PhD, a UCSF School of Medicine postdoctoral fellow, to develop a high-throughput screen (i.e., fast, simultaneous, automated) of hundreds of potential drug compounds. In particular, they were able to conduct such screening in an oxygen-free (anaerobic) environment that mimicked the natural environment of the disease-causing amoebas. The screening identified auranofin as especially active against the parasite, which was followed by confirmation of its effectiveness in laboratory and animal studies.

Based on their results, the scientists received orphan drug status for auranofin from the U.S. Food and Drug Administration and have applied for approval to start clinical trials testing the parasite treatment in humans.


Lab Tests Show Arthritis Drug Effective Against Global Parasite - UCSF.edu

A high-throughput drug screen for Entamoeba histolytica identifies a new lead and target - Nature Medicine


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About the School: The UCSF School of Pharmacy aims to solve the most pressing health care problems and strives to ensure that each patient receives the safest, most effective treatments. Our discoveries seed the development of novel therapies, and our researchers consistently lead the nation in NIH funding. The School’s doctor of pharmacy (PharmD) degree program, with its unique emphasis on scientific thinking, prepares students to be critical thinkers and leaders in their field.