A Challenge Accepted! ;)

OLYMPUS DIGITAL CAMERAIt hasn’t happened before. I was given a MISSION, if I chose to accept it, to explain some new developments in regards to Sarizotan (Newron) and Anavex 2-73 (Anavex) receiving FDA Rare Pediatric Disease Designation: “Mel Lancaster have you posted about these announcements or am I just behind on things? Have you deciphered it for parents who are new and may not get all of this yet?” Let me just say I was quite flattered! So, never one to step away from a challenge… Here we go.

One of the first hurdles for these companies and drugs to get past is Orphan Drug Designation. “The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.” (From the FDA website)

A “Rare Pediatric Disease”, in contrast, is a rare disease “and one where the disease is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals from age zero to 18.” (From the FDA website). Also, if a rare disease begins in childhood but doesn’t become life threatening until adulthood, it doesn’t qualify. If a disease is rare, begins in childhood, but isn’t life-threatening, it also does not qualify.

So, this is a subtle difference, but important. Now, Orphan Drug status and Rare Pediatric Disease may seem synonymous to a degree, however they are not, nor are the incentives for the companies. And this difference in incentives is the BIGGEST reason this new designation for these companies is so important.

In order to understand what is such a big deal about this, one must have a basic understanding of the different designation/statuses that these drugs/companies have or may already have. And before anyone asks, yes Trofinetide/Neuren has most if not all of the following.

 

From the FDA website:

Fast Track status: Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

  • More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
  • More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
  • Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
  • Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA

Priority Review: A Priority Review designation means FDA’s goal is to take action on an application within 6 months. (instead of 10 months) *This is the BIG DEAL about Rare Disease Designation*

Orphan Drug Status: An orphan drug is a drug meant for a rare disease or condition. With this designation, the company receives incentives to continue to develop this drug. Some of the incentives include:

• 7-year marketing exclusivity

• Tax credits (up to 50% of clinical development costs)

• Exemption/Waiver of application (filing) fees

• OOPD assistance during the development process

Rare Pediatric Disease Designation: “The Rare Pediatric Disease designation provides incentives to advance the development of rare disease drugs and biologics. Additionally, the FDA’s Rare Pediatric Disease Priority Review Voucher Program states that a sponsor with a Rare Pediatric Disease designation who receives marketing approval for a rare pediatric disease may be eligible for a voucher that can be redeemed to obtain priority review for any subsequent marketing application.” (From Business Wire)

And, therein lies the biggest part of this news. Having drugs be given Rare Pediatric Disease Designation makes it POSSIBLE to apply for the priority review. A priority review means the FDA must take action on a MARKETING application within 6 months instead of 10.  IF a company already received a priority review through another process (i.e. Fast Track Status- which is then specific for THAT drug only), this is theirs to do, in particular, one of two things. 1. They can keep it and use it for another drug, even if it is not rare or even for a pediatric disease, OR 2. they can sell it for tens of MILLIONS of dollars and more. If they DON’T have it already and get it through this program, then they can use it to speed up the time from the application submission to when they can begin to market the drug.

Priority Review is important to US, because it gets a drug, if approved, to our children approximately 4 months faster (give or take). But, if a drug already has priority review and then it gets ANOTHER through the Rare Pediatric Disease (RPD) Designation Program, it’s theirs to keep or sell. For the company/ies this is a tremendous monetary advantage .

I congratulate both Newron and Anavex on this accomplishment.

 

 

 

 

 

Posted in Acadia, Anavex, Anavex 2-73, FDA, Neuren, Neuren Pharmaceuticals, Newron, Orpan Drug Status, sarizotan, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | Leave a comment

A Day with ACADIA- Part 2: Assurance

acadia2

Some of the ACADIA employees at the Rett syndrome Awareness event.

Katelin and I had the great pleasure of being invited to ACADIA a couple of weeks ago for Rett syndrome Awareness Month. Last week I posted Part 1: Awareness; I had PLANNED to follow that closely with Part 2, but a little thing called the “Official initiation of Phase III” interrupted that plan! But, this part is especially important now that the trial has officially started.

 

I have held ACADIA and Bill Keller, Executive Director; Patient Advocacy and Industry Relations, in high esteem since I first met them over a year ago. I was struck by their commitment to the families- how can we make this process easier for families. I was reassured by how important they believed US to be in this process. They interviewed multiple families, had an in-person roundtable with families in Princeton, and reached out to people and organizations that could give insight as to how to best run the trial in a way that supported families. I was reassured then, now I was given assurance.

One thing Bill Keller said that really resonated with me because of the sincerity and sense of duty that he said it with- (paraphrasing here) “I met Steve Kaminsky and he told me that since this drug (Trofinetide) existed it was our responsibility to get it to families.”

And that sense of responsibility is clear in everything ACADIA does-from listening to their employees’ desire to learn more about Rett syndrome from families to setting up a website weeks ago that put families in touch with sites. Despite all that, the one thing that sealed the deal for me was this-

ACADIA wants to hear from US, the Rett community-

  • Can you please let me know, so that I can share with ACADIA, the resources and support that you value most in caring for your Rett child by way of getting new information?
  • We also talked about community support programs such as Strollathons etc. but it would be great to know, if ACADIA attended these events, what information would you value the most. Keeping in mind they aren’t able to divulge anything about how the trial is going, but should they be a presence at these events ACADIA wants to make sure that they are providing something of value and not information we already know, which would cause frustration.

Let’s pull together and give them options to choose from. This is not a company that is saying… we’re the scientists, you’re just going to have to wait until it’s all over and deal with it… they’re saying, “We are in this together.” Something along the lines of “It takes a village” to do this Phase III and all its aspects right.

What assurances did we get?:

That they are systematically having sites inspected and rolling them out as soon as possible.

That all issues with manufacturing-such as multidose storage etc. have been resolved.

The mere fact that the trial is a go means they have enough Trofinetide in four different tiered doses for every child in the trial and for Lilac 2-an extension to (hopefully) cover from the end of the trial to approval.

That ACADIA has a plan in place to assist families with the costs of trial participation.

That ALL the families matter, those in the trial and those that don’t qualify because we’re all going to get it if (when) it gets approved.

I will forward suggestions to ACADIA in a couple of weeks, giving YOU-parents/grandparents/caregivers a chance to participate in a real way. ACADIA feels that trial participants are not the only ones who can make a difference. Here is a chance for those with children/adults that don’t qualify for the trial itself to contribute in a positive and meaningful way.

You can leave a comment here, on Facebook or email me at m3ml@hotmail.com. 

We’ve got this. We’re in good hands with ACADIA and Neuren.

 

 

Posted in Acadia, Dame Margaret Brimble, Education, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment

Hot off the Presses- The Horses are Outta the Gate!

racehorse-1-1377009Well yesterday’s post WAS going to be the last one this month but then I got an email from Acadia- “Hot off the Presses” it said and then this announcement- ACADIA Pharmaceuticals Initiates Phase 3 Pivotal Study of Trofinetide in Rett Syndrome, a Rare Neurodevelopmental Congenital CNS Disorder  The tears started around the word “initiates” and by the time I got to “Trofinetide” I was a blubbery mess, snot and all. Within minutes of posting it to Facebook, I got a call from another mom and there we were the two of us crying, swearing, cheering, making no sense and so much sense all at the same time. I count that as one of the precious memories I hope to carry all the way to my grave.

Following quickly on the heels of that was this announcement from Acadia- Trofinetide Phase-3 Clinical Study Update There’s a lot of pertinent information in there, so please take a look. The thing that jumped out at me are three sites- Phoenix, AZ; Houston, TX; Chicago, IL- have already started screening. Now, I KNOW some parents are getting responses along the lines of… we already have so many interested families… do not be discouraged, reach out to sites further down the line and also remember unfortunately not every child screened will pass the process, so we NEED more interested families than spots in order for them all to get filled.

And lest we forget where it all began- Neuren released its own announcement . So, in case you all couldn’t guess, this is BIG, BIG NEWS.

I know that as we each have read these announcements the possibilities that come to us are all different. In a moment the “never gonna’s” have become “maybe will’s”, “could it really be possible’s” and while I am totally on the side of Trofinetide making a difference, I can’t possibly guess if all girls will be affected eventually in the same way, but I believe with all my heart and soul that at the very least some of your “never gonna’s” are going to go away. I believe you will hear the lilt of your child’s voice in some way that everyone will hear and understand. I believe hands will do things that may be very small or huge and it will seem like a miracle.

There are still things we need to know- is there a cap on improvement, meaning eventually is there a place where our children can’t improve anymore when we see there is still room. Are there side effects with long term use. Each and every family that partakes are heroes to all of us; even those who don’t end up qualifying, you tried and that’s everything in a world where trying can be so hard and defeating, it’s so very brave to take that chance.

The race has begun, the horses are out of the gate; I’m betting on ours, the one with the purple-headed jockey; I’m betting on us.

 

 

Posted in Acadia, Dame Margaret Brimble, hand clasping in Rett syndrome, margaret brimble, Neuren, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, Trail to a Texas Trial, Trofinetide Phase III, Uncategorized | Leave a comment

A Day with Acadia- Part 1: Awareness

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This is my last post of Rett syndrome Awareness month. I had hoped to do more, but Rett syndrome made sure a good amount of awareness was happening right at home, but this was going to be the last either way.

 

I like to end this month on an up note and that presented itself when I was invited to speak at Acadia in San Diego. Bill Keller, Executive Director; Patient Advocacy and Industry Relations, of Acadia wanted to do a Rett syndrome Awareness event for the employees of Acadia… because the EMPLOYEES wanted to know more about it and to get a sense of the families and children they are working so hard to help.

Now, had Bill told me the scope of the audience I still would have showed up but probably with a liiittttle bit more trepidation. And, I wouldn’t bring it up except that I want you, the reader, to understand the interest and to know that this was a voluntary event. There were so many people who wanted to be there that our interview had to be broadcast to another room, there weren’t enough chairs for everyone and people were standing AND it was being broadcast as a webinar to Princeton.

Katelin, Flynn and I were met warmly by so many of the executive staff and made to feel so very welcome. Bill had asked what kind of food Katelin liked and damn! did they get it right. My girl feasted on Chicken Alfredo and then feasted on my Chicken Alfredo, lol. and, get this… Katelin’s wheelchair was in need of some minor repair but it was making it difficult to use and Bill promptly called the maintenance dept and it was zoomed away and came back as good as new!  Thank you, Juan! There was a photographer snapping away. I’ve been sent four pictures, two of them have Flynn in them. I have to wonder if the other two are the only ones that didn’t have Flynn, lol, she was VERY taken with our boy. Needless to say, we were treated like very honored guests.

It was a big deal. Probably one of the biggest deals I’ve ever participated in. There was a briefing, a debriefing, sound checks, I almost felt like I was on a News talk show. I had a portable microphone. It was an experience I can hardly believe Katelin and I were invited to. I can only hope that I represented our community as well as it deserves.

The first thing they showed was a video of Katelin of her initial journey with Rett syndrome that I made as a NNZ-2566 trial fundraising video (I edited the very end out that was asking for donations 😉 ) Luckily, Bill had forwarded the questions to me in advance so I was prepared and I will share some of them with you.

  • Can you share with the ACADIA team some of the activities that are going on in the local community around Rett Syndrome? :

I, of course, brought up the strollathons, and golf fundraisers, the LA Feast, but other events that I feel are important for the community are events like the Paint a Purple Pumpkin, and the Paint Your Nails Purple and post a picture. These are important for our community to feel connected to each other. One of my FAVORITE events is Blue Sky Day and the message it sends- so symbolic of our climb as parents to help our children, find a cure/treatment and symbolic for our children- the effort to achieve every little advancement in their own personal lives.

I recently participated in a podcast series and shared the link with Bill- To listen to the episodes, please visit anchor.fm/remarkablepodcast or search for “Remarkable Rett” wherever you listen to your favourite podcasts.

  • What about other clinical trials and areas of research?

I mentioned GP2C here. They did a great article about the different trials going on this year. The ones specifically I mentioned were the Sarizotan trial; Anavex 2-73 we discussed the Epidiolex trial in another question. I know there are two that I missed- the Ketamine trial and Triheptanoin UX007. The Ketamine trial is something I’m excited about. I haven’t yet researched Triheptanoin UX007.

  • I know that you have been following the Rett Syndrome research space for years. What is going on that excites you about future developments?

Well, of course, I said the Trofinetide Phase III excites me, lol. But, there are other developments as well. Anavex 2-73 is an exciting possibility. One area that is intriguing is the use of stem cells and one recent practice is feeding a fresh placenta to children, you can find out more about it here- Placenta Preservation. But, what excites me the most is the sheer number of trials in some stage of development or another.

  • Can you share your opinion about a topic we hear a lot about in California which is medical marijuana? Does it have a place?

I freely admitted this is not an area that I have expertise in. Mostly of what I shared came from reading accounts of other parents. I said it definitely has a place, whether it’s Charlotte’s Web or the like or other systems of delivery, like Epidiolex. I also brought up other alternative therapies such as essential oils and other naturopathic therapies. But, because this is not an area I can really give in depth insight on, I referred to Syndi Knowlton of Utah Kids.

  • Can you share with the ACADIA team your recent introduction to Dame Margaret Brimble and her role in the development of Trofinetide?

I LOVE this story and memory. Dame Brimble is the inventor of Trofinetide and you can read more about it- A Mother Meets a Child She Will Give New Life To

  • How is Katelin doing today? What are you excited about for her future?
  • What is your greatest hope?

These two go together. First, Katelin is doing very well. She’s healthy. Her spinal rod surgery went as well as could ever be expected- her back is straight, she grew almost 6 inches and she hardly ever chokes in her sleep anymore.

My greatest hope and what I am excited about in her future are intricately linked. Trofinetide is my greatest hope. *disclaimer: her trial has not been unblinded, this is my personal opinion* I’ve seen it at work. My daughter has felt it at work. Katelin has only love and admiration for Trofinetide and the people who work on it; it’s obvious every single time we do something like this, meet people involved in the trials, when we met Margaret, when we meet James Shaw and Dr. Nancy Jones of Neuren. She KNOWS it works and she’s waiting very patiently for it. Sometimes when she’s very upset, I tell her…when Trofinetide comes, this will be alright… and she calms right down.

What am I excited about her future??? College. I fully expect that Trofinetide will enable her to communicate/speak well enough to attend classes. She is going to get to LEARN what SHE wants to learn-space, biology, maybe even writing! (be still my heart 😉 )

After our interview there was a short Q & A; the one question that struck me the most was- We’ve had several parents here now and you all seem to have such joy, why is that? Well, I did joke that we were when we were there! (In my mind I was thinking- we get a great lunch and don’t have to clean up, it’s like a vacation!) However, for me, this is why I think we are able to find joy in the face of everything we deal with- we learn that the little things are the big things and the big things that most people worry about are nothing compared to those little things. You learn that showing your child a butterfly or smelling all the different flowers while you’re out walking keeps you young and reminds you of the miracles all around you. When a butterfly lands on you it can sustain you, because really, isn’t that a small miracle? Simply put we learn to be present in the moment and find the joy.

Recently, there’s been a disconnect in our community and I want to say, this experience proves to me that our hope is dependent upon all the different facets that parents promote: fundraising, research, awareness; even if it doesn’t always look like we think it will.

Without funding there is no research or trials, without trials there is no hope, and awareness isn’t needed just for those things to occur; it’s for people like the employees of Acadia- people who are going to work every day to save our children, who want to know why they should, who deserve to be told that we are grateful; people who deserve to see the children they are going to give a voice to; people who deserve to know that we are committed right alongside them to help our children, that we are not just waiting for others to be our saviors. Neuren and Acadia, among other companies, deserve to be made aware that yes they get a paycheck, but there’s a reward of a lifetime waiting at the end of trials- saving thousands of lives. Not just, hopefully, that they survive better but that they truly get to live.

Watch for: A Day with Acadia- Part 2; Assurance! Coming next week!~

 

 

 

 

Posted in Acadia, Anavex, Anavex 2-73, Communication, Dame Margaret Brimble, fund-raising for rett syndrome, gp2c, hope, ketamine, kyphosis, Neuren, Neuren Pharmaceuticals, Newron, NNZ-2566, NNZ-2566 trial, Remarkable Rett-a podcast series, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, RSRT, sarizotan, service dog, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment

20 Years in Oz

Trail To A Texas Trial

imageI saw a writing contest advertised on Facebook, the very next ad under it was something to do with the Wizard of Oz…and so I was inspired.

I had hoped to get this published, but no takers. However since this is Rett syndrome Awareness month and I won’t have time to update regularly, I would rather share it than let it never get read, especially after all the work Carrie Weaver put into editing. Without her, it would not have said all that needed to be said.

20 Years in Oz
By Melinda Lancaster


Seen almost exclusively in females, Rett syndrome typically represents between 6-18 months of age. Characterized by  developed microcephaly, stereo-typical hand movements and a regression of skills, Rett syndrome effectively wipes away all hand-use, verbal language, and mobility, leaving children trapped within their own bodies.  Many children develop seizures, breathing abnormalities, swallowing difficulties, abnormal weight loss, GI…

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A Trip of a Lifetime

road tripI recently took a trip with Katelin. A long, boring, exhausting trip- more than worth it for the end result, but the getting there and getting back were tough. Katelin threw up on the way back and that was the least of the difficulties that came up. On the plus side, I had hours upon hours to think about the upcoming Trofinetide trial.

 

Usually when I think about the trial, it’s all about the benefit to Katelin. This time it came to me all the benefits to the FAMILY this would/could mean. Think about it…

If your child can speak/communicate and are calm-

  • PEOPLE WILL BABYSIT. Think about that. A babysitter. OMG. Husbands and wives going on DATES. Single parents doing the same. You won’t have to turn down invitations because you can’t find a sitter. (of course, unless you want to.) 😉
  • People can WORK, possibly full-time. All those parents who’ve only become a caregiver can have an identity outside the home.
  • You can go on family vacations!
  • Think of all the hours of therapy your child may not need and how you can fill them up with normal kid things.
  • If he/she has to go to the bathroom, they can just tell you instead of crying because they’re holding it in because they want to go on the toilet instead of in their diaper/pull-up.
  • You can go to your other kids’ events; there won’t be a worry about overheating, or screaming, or being overstimulated (at least you have a better shot!)
  • Think about this… no more stupid state testing which always comes back “scores below normal” on everything. No more punches in the gut every time report card time comes around and it’s either all sub-standards or all A’s, neither of which our kids deserve.
  • The economic relief of not having to continue to buy all sorts of therapy/safety/personal hygiene supplies/communication devices etc.
  • There are so many positive aspects that we can all come up with, individualized to our circumstances that will make it all seem like the beginning of a new life for everyone in the family, not just the child who suffers from Rett syndrome. There will be decreased stress, for those of us with aggressive children- I believe there will be a great decrease in aggression and self-injurious behavior, I’ve already seen it happen.

Thinking about these possible outcomes, I was not full of excitement and joy, I was filled with relief and a different level of hope than I have just for Katelin. It’s a hope I hardly dared to think of, but I feel a change in this journey; I feel that somewhere along the way the “ok” was sent out and I finally picked up on it.

Yes, we want all the amazing, wonderful things for our kids they deserve and will get, if Trofinetide works half as well as I think it will. But, the aspect of the effect on the family as a whole unit is not something I’ve given much thought to. Wow, a babysitter; family that doesn’t disappear or isn’t welcoming; birthday parties with classmates and cousins. Of course, along with that will probably come… I want THOSE jeans, I don’t want THAT for dinner…Wouldn’t that all be just amazing!

I don’t think it’s a pipedream, especially for the younger ones. I think it’s within reach. What a trip of a lifetime it will be.

 

Posted in Acadia, Communication, cure, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, screaming in rett syndrome, Special needs siblings, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | 2 Comments

A Mother Meets a Child She Will Give New Life To

katelin and margaretIt is no secret that I have high expectations for Trofinetide, I fully expect Katelin will speak; I fully expect her to be able to read and write; I expect that she will be calm, well she is Irish, so calm until she gets her Irish up. 😉 In essence, I expect her to have a brand new life. Maybe not QUITE the life that was stolen from her as a baby, but a life far better than Rett syndrome tried to leave her with. There are many people to thank for that- Acadia Pharmaceuticals for believing this was a gamble worth pursuing; Neuren Pharmaceuticals and the stock-holders who saw the potential and invested SO MUCH time and effort in getting this to Katelin’s trial and for so much more than that- for caring so much about my girl, and all our children. But, it all had to start somewhere, and the place it started with was the lab Margaret Brimble worked in.

Margaret invented Trofinetide nearly 20 years ago. That would’ve been right about the time Katelin finally had the gene testing for Rett syndrome and had it come up positive. I wish I had known, had felt in that moment of “Eureka” there was hope. But, Margaret knew. She tells me quite a story of how one of the investors for NNZ-2566 (initially designed for TBI) had a child with Rett syndrome, not knowing at that time so many years ago, he had just invested in a treatment for the very thing that was affecting his family so significantly.

I “met” Margaret on-line years ago after I did a blog post “What’s in a Name”. I received the following comment: “I am the mother of trofinetide -we made it for Neuren in our lab at Auckland university; I was brought to tears when I read this article.
I am so glad I did medicinal chemistry for a career, better than being a clinician- we get to try to make magic treatments for dreadful diseases like Rett syndrome.”

Since that day, we’ve kept up a correspondence and FINALLY, after years and awards that have piled up for her, including recently being made a Dame and on August 27, 2019, being inducted into the Medicinal Chemistry Hall of Fame in San Diego, we were to meet!

I had it all planned out! HA HA Otherwise known as “the best laid plans of mice and men”, we were to arrive at the hotel around 4pm, get Katelin dressed, meet her husband, Mark, and have a leisurely few minutes before going into the ceremony. Well due to a nearly 5 hour drive, instead of 2, to San Diego, needing to stop to feed a very loud, hungry child, none of that happened. We missed the ceremony, Katelin was not nearly as neat and clean as I wished, but I did throw a clean shirt over her for our picture, lol. If that doesn’t sum up the life of a special needs family, I don’t know what does!

From there on out, though, it was surreal. I have been wishing to meet Margaret for so long, I have often wondered what kind of fool I would make of myself. How many times can you say “thank you”? I even had my stepdaughter, Andrea, give me eyelash extensions so if I cried, mascara would not run down my face. Yet, when I met her, it was like two old friends meeting after a long parting. I held the hug a little long, I’m sure, and I had the urge to hold her hand, as if that would keep her there just a little longer; my heart surely knew I would miss her the moment she was gone. It was JUST like friends catching up- funny stories, what the kids are up to, what’s next. There was not one moment of awkwardness, not even when Katelin decided the night had gone on long enough and I had to jump up and get her out of the restaurant. What a gift- to spend the night with people who, never having met us in person, made no judgement whatsoever when it came to whatever had to be done to make Katelin feel comfortable and happy. There was so little time and so much to say, so much unsaid, but PERFECT in every way-a night I will remember for all my life.

Since we missed the presentation, she gave us a quick overview of her slides and there was Katelin wearing a Trofinetide necklace. When Katelin saw herself, she clapped her hands. She knew this was a big moment for both of us and was as good as gold right up until she was ready to go to bed! And, since Katelin had been in Margaret’s presentation, person after person came up to meet her- scientists, Hall of Famers, students, a microcosm of the scientific community that influences the lives of our children in ways we cannot imagine. One surprise that left me star struck was meeting Bruce Maryanoff, the inventor of Topomax. I have to admit, I think he was slightly taken aback when I hugged him and thanked him for helping so many of our children.

I learned so much about the twisting, turning path that has brought Trofinetide to our doorstep and it has been quite a journey. One of the things that Margaret shared was a photo of healthy volunteers lining up to get NNZ-2566, the first people EVER. How brave they were.

I also learned that her husband, Mark Brimble, is a bonafide genius, writing the software that allows computers to translate the different languages around the world and allow the connections that make the World Wide Web actually worldwide. (Knowing this makes me feel better about not understanding a word of a seminar I attempted to watch)

It’s taken a couple of days to really reflect on this whole experience and I will try and put into words what I’ve learned or rather had reinforced in a way I could not have imagined.

Every drug, piece of equipment, method of therapy our children use began with a single person or team. While it’s easy to focus on the company that promotes them, behind each of these is a name and a face that will largely go unrecognized or unknown. The world owes a great debt to these people. I feel beyond fortunate to have met some of these people, to hear their stories and to come to know that they are, in many ways, just like us; they gather to celebrate those that deserve recognition, hang around and toast to successes and are generous and so kind to make one child feel like they are a superstar and among a room full of giants in science made Katelin out to be a giant among them. It was a meeting worth 4 years in the making and 5 hours on the road.

hall of fame

Thank you, Margaret, the Rett community at large is also grateful for your contribution from the bottom of our hearts.

I want to thank the American Chemical Society-Medicinal Chemistry Division for making allowances in an effort to make sure we could be present and for being so welcoming and kind to Katelin.

PS: they all loved Flynn, too. 😉

Posted in Acadia, ACS Medicinal Chemistry Division, American Chemical Society, Dame Margaret Brimble, hope, Mark Brimble, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Rett Syndrome, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , | 7 Comments