We’re proud to announce a Microryza crowdfunding campaign to support our first therapeutic trial.

Anle138b, unveiled earlier this year [Wagner 2013] currently ranks as the most promising compound for treating prion diseases.  It was originally tested in mice infected with RML, a well-studied strain of rodent prions, and it delayed terminal disease by 30% even when it was administered fairly late in the game, after about 2/3 of the incubation time had passed and symptoms had already started to emerge.  It also interfered with the formation of human prion strains in a test tube, but it’s never been tested against a human prion strain in vivo.

That’s why we think another test of anle138b, against another prion strain relevant to human disease, is of critical importance.  Our project will fund the testing of anle138b in Dr. James Mastrianni‘s model of the genetic prion disease Gerstmann-Straussler-Scheinker syndrome (GSS) [Yang 2009].  These mice carry the mouse equivalent of the human A117V mutation in the prion protein gene.

We’ll test at two timepoints.  An early group of mice will receive the drug starting from weaning, while a late group of mice will receive the drug at the onset of symptoms of GSS.  Together these experiments will tell us how useful anle138b could be prophylactically and how useful it could be in symptomatic patients.

The compound’s inventor, Dr. Armin Giese, has ambitions to move the drug to clinical trials, perhaps as soon as within 2 years.  If our GSS trial is successful, the results will help to advance anle138b towards those clinical trials.

It’s possible that we’ll learn that anle138b doesn’t work against GSS – many anti-prion compounds have proven strain-specific.  But so far, anle138b appears to be more universal.  It worked against the aggregation of alpha-synuclein, the protein involved in Parkinson’s disease, in vitro and also had therapeutic effects in two different mouse models of Parkinson’s disease.  If it does work, there’s a possibility that anle138b’s reach will end up being much larger than prion disease.

We think this is an important project, and we hope you agree.  Visit our Microryza project page to contribute today!