Today is the last day of October. Another year gone by from one Awareness Month to the next and still no treatment. But this is not a humdrum post BECAUSE treatment is now within sight. Each year in October superhero parents put on their capes and causes and go out into the world and educate, fund-raise, sell raffle tickets galore. These parents astound me. Every time I see a gala, golf tournament, comedy show etc. I’m simply flabbergasted at the effort, truly awed. I’m not one of them. I write and hope somewhere, somehow it reaches people and moves them to research Rett syndrome and maybe donate, because the donations are working!
There is an incredible amount of research going on in this field and multiple studies either in the works, recruiting, on-going or just completed. THIS is what Rett parents and caregivers need to be aware of- the research is happening, donations are coming in… but without people signing their children up for clinical trials none of it will matter.
Newron (not to be confused with Neuren) is conducting the Sarazotan trial for children with breathing difficulties. They are having a great deal of difficulty filling the necessary spots. Click HERE to see where active trial sites are or for more information from the clinicaltrial.gov site-Click HERE and the RettSTARS website. This is one of the few trials that is occurring in countries other than just the United States-Australia, United Kingdom, Italy, India
One of the most anticipated trials coming up is the Phase III pivotal trial of Trofinetide, to be conducted by Neuren. This trial will begin recruiting in the next few months, and in my opinion represents the best chance right now for addressing the symptoms of Rett syndrome and has been shown to have the possibility of modifying the course of the disorder. Anyone can go to neurenpharma.com to review all their announcements and trial results.
Anavex has developed a compound Anavex 2-73 which has been shown to be tolerated in human studies. They are beginning to reach out with a preliminary questionnaire for interested parents, which you can access- HERE There is a short information video as well.
The Natural History Study is an ongoing, years long, collection of data. Parents are encouraged to enroll their children in this study, not only is it imperative to collect data in the long term, but it is possible that future trial participants will be taken from those enrolled, as this will have the most up to date data on each participant, in addition to a well documented progression of the disorder.
A quick search on clinicaltrials.gov shows 42 trials in some phase of completion, ranging from “not yet recruiting” to “completed” or “terminated”. I hate that one, “terminated”, meaning for some reason or other it was stopped prior to actual completion. But the exciting thing is the number of trials-42! and on such a rare disorder, too.
So, this is a time to be hopeful and to be aware that there are people out there, hidden behind masks and scrubs, that are doing their damnedest to help our children. I know when parents/caregivers see another trial come to an end and still nothing is approved it is a rollercoaster of emotion, we get weary of hoping at times; sometimes we are cynical; sometimes we just can’t bother to even care- who can blame us when we know so many children for whom a treatment/cure will never come. But then, I look at Katie who was so brave during her trial, my superhero, and I know if she can be that brave, so can I; I know that our community is chock full of superheroes in every shape, size and gender. We are formidable and we will win in the end.