MJFF Scientific Advisory Board member Dave Weiner holds a unique perspective into the world of Parkinsonís disease drug development: He works in both the early discovery phases that take place in the lab, and in a clinical setting. He hopes to leverage this broad experience into finding new drugs for PD, faster. ††
MJFF spoke with Dave in the latest of our monthly feature, Three Questions for a Researcher. Read on to get to know him and his work a little better.
MJFF: What is the biggest challenge you face in your research today?
DW: The biggest hurdle we face in that development of novel therapies for PD is trying to model or predict potential efficacy in humans in the pre-clinical setting.†The costs of developing molecules to enter the clinic is high, and clinical development costs are exponentially higher, factors that limit the number of assets any given organization, no matter what size, can progress forward.†Prioritization decisions need to be made, and this is often based on data derived from pre-clinical models of Parkinsonís disease.†
Unfortunately, these models have proven to be extremely poor predictors of eventual clinical efficacy (with the exception of treatments that improve motor function).†This creates a situation where two highly damaging sets of decisions are made, progressing a molecule with positive pre-clinical model data forward without any real (only perceived) confidence that it has a lower risk to fail, and conversely, stopping work on potentially promising treatments because they do not appear to work well in pre-clinical models.
MJFF: What is one thing people would be surprised to know about your daily work with PD?
DW: That it encompasses both pre-clinical and clinical activities in drug development.† Most scientists in drug development usually work within one area or the other. But I have been able to leverage my experience in both discovery and early clinical development to provide hopefully meaningful input in Parkinsonís therapeutic development to the discovery programs we are working on at Proteostasis, as well as to the various discovery and clinical programs that I am exposed to through my work with the Fox Foundation.
MJFF: How do you unwind after work?
DW: One of my passions is live music, and I try to catch as many performances as I can, in both Boston, and in my adopted hometown of New Orleans.†Many of my friends there are musicians, most are profoundly talented but relatively unknown (for now).†I also swim regularly, and try to snowboard as often as I can.