Yesterday, I was contacted by a Neuren Rep wondering if I had heard of the Pediatric Trial with NNZ (Trofinetide) and would I mind promoting it. I had an inward chuckle at that 🙂 and she sent me a blurb, just a few short lines. Not as short as Ernest Hemingway’s famous 6 word story “For sale: baby shoes, never worn.” but in both cases you can extrapolate the larger story. So from this approved blurb, let me give you a longer story….
“Neuren Pharmaceuticals is currently conducting a Phase II clinical trial of a potential new treatment for pediatric Rett syndrome (females aged 5-15 years). This is taking place at various locations across the US and reimbursement is available for reasonable travel costs incurred during participation. Please visit clinicaltrials.gov or http://www.rettstudy.com for study locations and further information about the trial. Latest updates can also be found on the rettsyndrome.org Facebook page. ”
That’s the short story, I could make it so much longer, chapters really… we could start with chapter 1
Once upon a time in New Zealand, a woman named Margaret Brimble and her team (see Wikipedia page, no sloucher, the mother of Trofinetide https://en.wikipedia.org/wiki/Margaret_Brimble ) created NNZ-2566….
The US Army is at war and soldiers are surviving horrific injuries, thanks to advancement in medicine and surgery, but this means long term disability for many and long term care for Traumatic Brain Injury victims. Wouldn’t it be great if something, anything, alleviated just even some of the symptoms??? Well, it would, so in comes Intrepid-2566, a long range, 10 year study (results due out any day now.)
Hmmm, says the brain detectives, wonder what else might be helped by NNZ-2566, what about Autism??? and associated disorders like Rett syndrome and Fragile X (cue in movie…ok, seminar) http://www.bing.com/videos/search?q=professor+snape+autism+and+NNZ&view=detail&mid=DD8677D5857F59C50A11DD8677D5857F59C50A11&FORM=VIRE
and so it really begins…
This is a personal chapter, this whole blog really. One child’s journey through the Phase II NNZ-2566 trial. This chapter is a hopeful one, but not without it’s tears. Wrought with outstanding generosity and a mom’s happy tears and one young woman’s journey as she regains skills lost in infancy only to be lost again, all within 40 days. It’s about her strength and commitment, seen every day as she never fights all the equipment they attach her to. The smile she gets when she’s told she’s going to help the younger girls, she’s their hero. And even when she regresses again and cries for three days, she comes out of it strong as ever. It’s about her clapping when Trofinetide gets Orphan Drug Status, asking for a phone if she’s able to talk at the end, yes, she’ll get one. And moving right on to “Car?” So optimistic is my girl (but no, she’s not getting a car!” (Disclaimer, Katelin’s participation has not been unblinded, meaning I do not know definitively if she received NNZ)
This is a retrospective chapter of the HUGE impact fundraising has had on getting us to that little blurb, the tireless dedication of people like Monica Coenraad and so many others who dig deep and deeper still to find the funds needed. To which we all owe a great deal and probably don’t ever get thanked enough.
The results of two successful trials, Rett syndrome and Fragile X, you can find their announcements here: Rett syndrome results and Fragile X, hell, just look at all of Neuren’s announcements...one success after another. And make sure to look at the first one “First subject commences Neuren’s Rett syndrome Phase 2 trial.”
Now we enter Chapter VI
I am certain there are many, many chapters that could be added in, that I am missing; interesting necessary chapters full of behind the scenes names that should be known and remembered for which I apologize not knowing, but am grateful for all the same.
The Pediatric Trial Commences.
I implore everyone in the US whose children fit the criteria and who have the means (note: all reasonable travel expenses will be reimbursed) to make this an exciting chapter long in substance, but short in the writing. Check out Rettstudy.com to find out if a site is opening near you. This is your chance to stomp on Rett syndrome. Knock it down. It’s a great feeling, it really is.
That will be full of waiting for the results and the start of the major Phase III, including children and adults. I hope for Katie to be in that trial. We need a cure for our children, and it’s our children, with their bravery, that will get it.
I don’t know how many chapters are left until the end, and I don’t know how many times they will be edited or wrinkled up and thrown away, only to be dug out and flattened and taken another look at, but I am certain, beyond doubt, that the ending words will be something like this…. A child developed Rett syndrome. In one hand the doctor held the results, in the other a prescription for a treatment that would prevent it’s symptoms. And that child went on to live a happy, healthy life. And for Rett parents there were no more short life stories.