UCSF researchers have determined that two drugs currently approved to treat either malaria or certain psychotic illnesses are effective in treating mouse cells infected with the infectious protein known as the prion (PREE-on). Prions cause new variant Creutzfeldt-Jakob disease, the human equivalent of "mad cow disease," as well as numerous other rare neurodegenerative diseases in animals and humans.
Because the drugs have long been used, are known to cross the difficult-to-penetrate "blood-brain barrier," and caused a dramatic response in the cells, the UCSF researchers advocate the immediate establishment of clinical trials to investigate the efficacy of the drugs in patients dying of prion diseases. Prion diseases are relentless and uniformly fatal.
The researchers report their findings and their recommendation for clinical trials in the August 14 issue of Proceedings of the National Academy of Science (PNAS).
"It's a big leap from findings in cell culture to those in humans, and we do not know if we will see a favorable response in humans. But the results we saw, in a cell model we consider valid, make this lead worth pursuing immediately," said the lead author of the study, Carsten Korth, MD. Korth is a postdoctoral scholar in the UCSF laboratory of senior author Stanley B. Prusiner(1.), MD, UCSF professor of neurology and biochemistry and director of the UCSF Institute for Neurodegenerative Diseases.
UCSF neurologists, in collaboration with the researchers, are in the final stages of developing a clinical trial(2.) to test the efficacy of the two drugs in the treatment of Creutzfeldt-Jakob disease (CJD) and other prion diseases. The drugs -- quinacrine and chlorpromazine - will be tested separately and in combination. The researchers hope to begin enrolling patients later this year.
The trial will be the first to test the effectiveness of drugs to treat human prion diseases(3.), which can aris
Contact: Jennifer O'Brien
University of California - San Francisco