Today RSRT announced $9 million of research awarded in 2015. What sets these awards apart from previous years is not just the significantly increased dollar amount but also the emphasis on clinical research. This focus reflects the critical need to conduct efficient and effective clinical trials with specific measures that are objective and sensitive to changes over short periods of time (months not years). We don’t have those measures yet and they must be urgently developed in parallel to the basic science we are funding. When a medication (like a drug that activates MECP2) or a biological (e.g. gene therapy) shows promising results in animal models we must be prepared to test its safety and efficacy in the best possibly designed trial. To address the current knowledge gap RSRT has awarded $4.5 million to finance a unique collaboration among four clinicians (Alan Percy, Aleksandra Djukic, Daniel Tarquinio, Tim Benke) who will explore a number of novel outcome measures.
This consortium is designed to be nimble, allowing the pursuit of outcomes that look encouraging while quickly discarding those that don’t. RSRT will manage the infrastructure to properly curate this ambitious project including a cutting edge data management system that will make anonymized data available to clinicians, scientists, industry and investors, thereby maximizing its potential use. This project is critical for assuring that lab science translates to having an impact on our loved ones struggling with Rett Syndrome and related disorders. It is very exciting that we have reached the stage with Rett research where we must develop clear measures for the impact we expect to have.
As always at RSRT, we are tremendously grateful to all the families that hold events and raise funds, to our generous donors, and to Rett organizations in the U.S. and around the world. None of what RSRT does would be possible without this support.