For as long as I’ve directed the research grants program at CurePSP, I’ve been a champion of the investigator-initiated approach. Let the experts decide what’s scientifically ripe and feasible for them, I say. I’ve seen too many RFAs produce opportunistic applications that are a stretch for an investigator both intellectually and practically.
But I thought it was best to compromise that principle by creating this “Road Map to a Cure.” It presents a general approach to developing neuroprotective treatment for PSP that builds on recent advances and hot ideas, interacts with the private sector, and is feasible with present technology. Perhaps best of all, its definitions are flexible enough to accommodate a wide variety of investigator-initiated ideas.
In developing the Road Map, I collaborated with Yvette Bordelon, a movement disorders specialist at UCLA and Chair of the Research Committee of CurePSP’s Board of Directors; and Jeff Friedman, a pediatrician/biochemist at Friedman Bioventures and Scripps and a member of CurePSP’s Board.
As you can see, the Road Map chooses three general hypotheses: a genetic etiologic component, a prion-like (the preferred term is “templating”) mechanism, and a problem with protein folding and management. It also identifies two broad categories of experimental tools that need improvement: animal (or cellular) models and human biomarkers. The Road Map then seeks to develop treatments based on one or more of the three hypotheses and using one or both of the two tools. Then comes the familiar the cascade of treatment development, from target identification to in-vitro or cellular screens to more complex model organisms to early-phase humans trials to late-phase trials.
Of course, some treatments may be ready for testing in animals or humans right now without the preliminary steps, and that would be great. Will CurePSP triage out any applications lying outside of this development model? Not at all. The Road Map is formulated more as a general guide and we recognize that there are other paths.
We also recognize that the Road Map ignores important things like symptomatic or surgical treatments, studies of exogenous risk factors, descriptive and nosologic studies and, perhaps most important, completely new and risky ideas.
The Road Map, as you’ve guessed, is designed partly as a fundraising tool that allows potential donors to understand how scientists would spend their money. The donors deserve to know that and are quite capable of understanding it if we scientists just take the trouble to explain it to them.