Issue link: http://www.e-digitaleditions.com/i/399268
Meet the Founder, cont... There were many contributions and refinements thereafter to what became known as the UHDRS. After several pilot studies, we started in 2003 to apply the UHDRS in HSG clinical trials. So that was an important ten years between the talking and the doing. HD INSIGHTS: What was the biggest success you experienced during your time leading the HSG? SHOULSON: I think the biggest success or satisfaction from my point of view is how sustainable the HSG has been. Like the PSG, once people were involved, such as investigators, coordinators, social workers, neuropsychologists, and scientists, the HSG built commitment, cooperation, momentum and sustainability. Our other early accomplishments were PHAROS, the observational study that was started in 1999, then the landmark "Neurobiological Predictors of Huntington's Disease" (PREDICT-HD) (see HD Insights, Vol. 7, p. 8) and "Cooperative Huntington's Observational Research Trial" (COHORT) studies, 3 followed in short order by the CARE-HD trial of Coenzyme Q10 in HD. 4 Our most tangible accomplishment was planning, conducting and reporting the TETRA-HD HSG trial that led to the approval of tetrabenazine to treat chorea associated with HD. 5 Another benefit of approval of tetrabenazine was that it signaled to innovators in industry that HD should not be neglected, but rather be thought of as an important unmet need and a market. HD INSIGHTS: What was your biggest disappointment or failure? SHOULSON: Well, we never had a strategic plan, which in retrospect, I think would have been useful. In some fashion, we were just kind of flying by the seat of our pants. Eventually, in 2012, we made the transition to an independent entity, HSG, Ltd. That was an important milestone, moving from childhood into adolescence. I just wish we had gotten through that childhood a little bit faster. HD INSIGHTS: You recently accepted a position on the board of directors of Prana Biotechnology. Can you tell us why? SHOULSON: Prana Biotechnology was developing the experimental drug PBT2, which had novel mechanisms and showed safety and signals of cognitive benefit in a European Alzheimer disease trial. 6 Working closely with Dr. Steven Hersch, we developed the clinical research interest in examining PBT2 in HD, and an approach that became the HSG Reach2HD dose- ranging phase II trial under the leadership of Dr. Ray Dorsey, Dr. Julie Stout, Dr. Diana Rosas, and Elise Kayson. As recently reported, Reach2HD showed safety and tolerability and signals of cognitive benefit that may prove to be reproducible and clinically meaningful (see HD Insights, Vol. 7, p. 1). Once my leadership of the HSG concluded in April 2014, and upon an invitation from Prana, I accepted the position as a non-executive director in order to provide clinical research expertise and help ensure that PBT2 was developed to its full potential in HD. Prana has a dedicated group of scientists and developers who have come to appreciate the many unmet needs of HD and the opportunities that exist to relieve some of the burdens. I continue my full-time work in leading the regulatory science program at Georgetown University, which has reviewed and authorized my work with Prana. I am compensated by Prana, both as a member of the board and as a consultant, but I have no equity positions, stock options, or incentive packages. I am focused on fully developing PBT2 for HD, whatever the outcome of future studies might be. HD INSIGHTS: What have you learned working directly for industry that you did not know before? SHOULSON: I learned a bit about business development as a bystander over the years that I was leading the HSG and taking on industry-sponsored research. I now better appreciate the need to integrate clinical research and business development. These endeavors are often undertaken in parallel but separately. That may be a mistake. It is very natural to do them together. The people responsible for business development must appreciate the clinical entity and unmet needs that are the problems that bother our patients and families. Then too, the clinical researchers who know and care for the manifold problems need to know how to be entrepreneurial and capitalize on opportunities. While seemingly awkward, clinical and business development best proceed hand in hand. HD INSIGHTS: What does the future hold for you? SHOULSON: I enjoy what I'm doing now. My day job is at Georgetown University, where I direct the program in regulatory science of medicine. It is an outstanding opportunity. We have a Center of Excellence in Regulatory Science and Innovation (CERSI) that is supported by the FDA, conducts important research in regulatory science, and offers an online Master's program in regulatory science, linked to the Georgetown-Howard Universities Clinical and Translational Science Award (CTSA). Copyright © Huntington Study Group 2014. All rights reserved. "I now better appreciate the need to integrate clinical research and business development… It is very natural to do them together." H D I N S I G H T S HD Insights, Vol. 9 18