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First Impressions

June 20, 2016

My first three months at RSRT have been a whirlwind of immersion in the Rett community. I’ve traveled extensively meeting with clinicians, scientists, and pharmaceutical and biotechnology company executives. I’ve attended a number of RSRT meetings that have gathered together the labs attempting reactivation of MECP2, the MECP2 Consortium, and the Gene Therapy Consortium. I’ve also had the honor of meeting some families and their beautiful girls.

This intense immersion will continue into the coming months as I become thoroughly acquainted with RSRT’s extensive research portfolio and more broadly the entire Rett research landscape.

Frankly, there is more activity than I expected. Activity on all fronts, from basic science to clinical research and translational medicine. We’ve all seen the many press releases and announcements from companies pursuing or exploring Rett programs. Although some of the press releases are public relations driven it is encouraging nevertheless to see industry interest. I fully expect this interest to increase.

The reality, however, is that the majority of novel therapeutics fail to demonstrate meaningful efficacy in clinical trials. Although, some of these failures will be due to lack of efficacy on the part of the drug, ineffective outcome measures may also be to blame. Specifically, trial participants improve in meaningful ways, but these improvements are not captured on the efficacy measures.

It’s absolutely critical that we improve the effectiveness and efficiency of future clinical trials, and that’s why a primary focus of mine during these first three months has been to launch the Outcome Measures and Biomarker Development consortium. This is a key effort if we are to improve the translation of lab discoveries into medications our children can benefit from. In doing so we reduce drug development risks and encourage investment from industry. This is a major goal at RSRT.

I have been impressed with the passion that I have seen on the part of many of the people I have met. Although a lot of hard work remains I am confident that we will make significant strides. Please know that I share your sense of urgency. I look forward to keeping you apprised of RSRT’s research efforts and hopefully to meeting many of you in the future.