Here's what's next

Therapeutic studies in models of fatal familial insomnia and other genetic prion diseases.  We’ve now successfully funded three such studies of anle138b, which we consider to be the farthest along of the current drug candidates for prion disease.  But any one drug alone is unlikely to be a solution – we expect that prion diseases will ultimately be treated by drug cocktails much like HIV.  For this reason we are keeping tabs on the other potential treatments currently in the preclinical phase.  We want to step in and provide validation of the most promising candidates in genetic models, in order to speed their progress towards clinical trials.

Early stage drug development.  It took years of work to bring anle138b to the point where it was ready for preclinical studies like the type we’re doing today.  We were lucky to find that the drug’s inventors, Dr. Armin Giese and Dr. Christian Griesinger, had already completed most of this work and had a strong drug candidate in hand.  This made it easy for us to step in and test the drug’s effects on genetic prion diseases.  But dozens of other potential treatments are still in the early stages, with researchers working on understanding how the drugs work, how to make them more effective, and how to make them as non-toxic and long-lasting as possible.  We would like to help more potential drugs move past these initial stages.  Not all will be successful – it is important to have many horses in the race.

Optimizing gene silencing.  While a few different drugs show promise of delaying disease, the only approach that has ever reversed prion disease after the onset of symptoms is to turn off the prion protein gene altogether.  This can be done therapeutically with RNA interference or “gene silencing” approaches, but it’s a long road ahead.  An important first step is to have the best possible small RNA molecules to target the prion protein gene and suppress it.  To this end, we want to screen thousands of RNA sequences to determine which candidates to move forward with.

Making treatments universal.  Some anti-prion treatments that have seemed promising in the past have turned out to only work against one or two very specific subtypes or strains of prion.  We want to understand why this is the case and develop new methods of searching for drugs that will be universal against all prion diseases.