Published November 25, 2019
Paul DeCaen, Ph.D. | Northwestern University
PKD Foundation (PKDF): How did you first get involved in PKD research?
Paul: I got involved late, but the timing couldn’t have been better. My training was directed towards studying the molecular structure and function of ion channels, which move ions in and out of cells. For about twenty years, we’ve known that variants in polycystin genes cause ADPKD and that they likely form ion channels. However, the field was stuck — we lacked a robust method for measuring their activity and the proposed channels lacked molecular context. About five years ago while training in David E. Clapham’s laboratory at Harvard Medical School, I established methods for measuring polycystin channel function in the primary cilia — a tiny organelle found on cells in the kidney-collecting duct. Around this same time, we reported with Erhu Cau’s team from the University of Utah the first high-resolution structure of the channel. Ultimately, these two game-changing advances coalesced to form the framework for understanding ADPKD at the molecular level.
PKDF: What are you working on currently?
Paul: We continue to make new discoveries in the polycystin molecular structure and define how these channels work, in molecular detail. We are also determining how variants in polycystins alter their activity in ADPKD — my hope is that this knowledge will set the foundation for rational drug design for this disease. Since there are numerous variants in the ADPKD population, it is likely they will affect the channel in different ways. In other words, some gene changes may cause the channel to be turned off. If so, can we design drugs to re-activate it? By defining unique prototypic drug mechanisms, we will have a premise for “personalized” PKD treatment based on the patient’s specific gene mutation.
PKDF: What would you like the patient community to know about your research?
Paul: The PKD foundation has provided critical seed-funding for my research program. We have put these funds to good use by acquiring cutting edge instruments and hiring a dedicated group of scientists. Thanks to your contributions and the Foundation, my lab has obtained additional funds to carry our scientific program forward in the years to come.
PKDF: Do you have a personal connection to PKD?
Paul: I’m often reminded by friends that they or someone in their family are impacted by this devastating disease. The reality of PKD has also become more vivid for me since joining the PKD Foundation and hearing people’s stories at PKD Foundation-sponsored events. If you recognize me out on the PKD walk, don’t be afraid to say hi and ask me what’s new in the lab.
PKDF: What excites you most about this research?
Paul: Given the frequency and distribution of PKD, I can’t think of a more meaningful and worthwhile scientific endeavor. We have an optimistic and brilliant team applying the most cutting-edge technologies, which will continue to transform our understanding of PKD at the molecular level, one discovery at a time. The excitement in the lab is contagious and the pace of our work can be very intense. I love it.
PKDF: What are some of your personal interests outside of research?
Paul: I love playing volleyball and watching my 1-year old daughter eat chocolate pudding.
PKDF: Anything else you’d like to share?
Paul: We are hiring postdocs and technicians. Come join our team that aspires to conduct transformative PKD research and we will provide you with the skills set to address tomorrow’s challenges in medicine.
PKD Foundation is the largest private funder of PKD research in the U.S. Since 1982, we’ve invested close to $50 million in more than 1,300 research, clinical and scientific grants, fellowships and scientific meetings. Each year, The Foundation identifies and supports the work of scientists and researchers from around the world who look for ways to treat and eventually cure PKD.
Our vision is to end PKD. Donations help fund necessary research that leads to more effective treatments and ultimately a cure for PKD.