Moving the Needle

I have always enjoyed the creative process. In my younger days, I was fond of cooking, which was not always sanctioned as an appropriate activity for a young boy in that era of our society. As I moved forward with my high school education, I had an intense interest in science. In fact, when an AP chemistry class was going to be denied us due to lack of enrollment, the dozen or so of us who did sign up for the class marched into the principal’s office and demanded that the class be offered. I am sure you can imagine the ruckus that caused. However, we succeeded in getting the class. I have made a career of swimming upstream and proving dogma wrong, even at the risk of my job at times. Some might even say tilting with windmills. I think that spark of fighting against the odds is what provides a link between the Rett family community and me. I can relate in a small way to Rett families due to the devastating chronic challenges of mental illness in my own family, both birth and adopted members. These are however infinitesimally small challenges compared to what Rett individuals and their families face on a daily basis. I am totally sincere when I say with great admiration that what Monica Coenraads and the whole RSRT team has accomplished in ten years is nothing short of miraculous. The stage is now set to make extraordinary advances.

There is an alignment of planets right now for new technologies that need the expertise of folks such as Randy Carpenter, Jana Von Hehn and myself to translate them to actual products that can move to the clinic in a few years. I am excited about the early trials in gene therapy with the AveXis/Novartis team. My decades in drug development, however, have taught me that it is an iterative process. Rarely does the first round of a new technology work to the level that will satisfy the entire Rett community. I am therefore committed to moving as many interventions to the clinic as make technical and medical sense. I personally would like to see 4 to 5 interventions move to the clinic in the next 3 to 5 years. They include a second-generation gene therapy, DNA editing, RNA editing, protein augmentation, and possibly a read through or MECP2-reactivation approach.

This vision is extraordinarily aggressive, but that is the only way I see that drug development should and can move forward. I have moved the needle before in my drug development career. My team worked with our major pharma partner to move Adynovate from concept inception all the way to the market. We also developed an opioid that does not cause addiction from invention to successful phase 3 and now an NDA submitted to the FDA. I want to move that needle again in a big way with Rett Syndrome.

I hope to meet as many of you as possible in the months to come. In the meantime if you have any questions I can help with don’t hesitate to contact me. timriley@rsrt.org