Hoping this letter finds everyone enjoying the final days of summer and getting ready to head back to school. Be sure to check out our “back-to-school” sale in the GP2C and Rett University store. We are offering a 20% discount (Code=BACKTOSCHOOL) for all GP2C merchandise and Rett U online courses, so this is a great time to get your teachers, therapists and caretakers a little “continuing education” before the school year starts! Susan and Kourtney are also booking private Rett U consultations for the remainder of 2019, so reserve your space soon.
From the research world, we know many of you are involved in, or considering, various clinical trials planned for this year. These include the much-anticipated AveXis-Novartis gene therapy (AVXS-201) trial. We recommend you continue to monitor this site for the latest announcements from Rett Syndrome Research Trust and AveXis. Additionally, I highly recommend review of this June 5, 2019 article, Making Sense of it All, which plainly identifies the process (and challenges) for bringing new treatments to fruition, including current status of the AveXis gene therapy project. One quote to pique your interest:
“…The timeline to complete IND (Investigational New Drug)-enabling studies is typically 18 to 24 months with an average cost of $10 to $15 million per lead compound. If the FDA approves the IND it’s off to the races with clinical trials in humans.
The AveXis/Novartis product is currently at this phase of development. The basic science and lead compound development work was funded by RSRT through our Gene Therapy Consortium 1.0. We purposefully recruited Brian Kaspar into the Consortium with the hope that the data generated would be sufficiently encouraging that his company, AveXis, would want to pursue Rett as an indication. A million things could have gone wrong but fortunately the pieces fell into place. AveXis funded the IND-enabling experiments and filed the IND application with the FDA. We are currently awaiting word regarding when the clinical trial will start.”
Many of you are further aware of the RSRT-sponsored Ketamine trial that is in-progress. For those participating, we appreciate your sacrifice and commitment, and hope all is going well for your children. Finally, and just recently announced, recruitment has begun for the long-awaited Phase III trial for Acadia-Neuren’s Trofinetide. For more information, check out this website for guidance on enrollment and participation criteria.
All exciting and encouraging news… and the kind of stuff that keeps us working hard (#HopeInAction) every day at GP2C, on behalf of our children and yours! We never say it enough, but thank you for all of your prayers, support, encouragement and fundraising efforts. We can’t do this without you, and our community is second to none with respect to supporting each other. Please let us know if we can assist you in any of your fundraising efforts, as we all work together to end this challenging journey.
Have a great August!