Jet-Setting Clinical Trial

Wow. I cannot believe it has been OVER ONE YEAR since my blog post. I think of posting so very often, and now that I am actually sitting down to do it there is so much to say.

The big thing going on in Rayleigh's life, in our lives, is a clinical trial. This entire post will be focused on just the trial so I get everyone caught up on that first. I'll start you off with the way we learned about the study...

In December 2016 a fellow CDKL5 mom reached out to me and told me that there is a clinical trial happening now for children with a nonsense mutation of CDKL5 or Dravet syndrome. Not all children with CDKL5 have a nonsense mutation, but Rayleigh does. THE DAY that I read her message we were on our way to a neurologist checkup appointment so I brought it up to Rayleigh's doctor. The neurologist looked it up right there during the appointment and we decided that it sounded promising and could be worth a try. The timing of that mom's message to me couldn't have been better.

Here are some key points:

• The medicine is called Ataluren
• Ataluren works unlike most seizure medications which calm or numb the brain pathways, because Ataluren is meant to fix why the seizures happen in the first place
• A genetic mutation of CDKl5 affects the ability to make the proteins that are essential for the brain - the doctors are hoping that Ataluren can allow the proteins through properly, picture a bridge over the broken mutation
• It is not a QUICK FIX medication, it is recommended we give it time to do it's job in her brain and her body
• The clinical trial is in New York City at the NYU Comprehensive Epilepsy Center under Dr. Devinsky and his amazing team
• We would be required to go to NYC every 4-6 weeks for the first 7 visits, then the 8th is 6 months after the 7th visit
• The trial is done in 2 sessions; Session 1 is drug or placebo, then a 4 week washout of neither, then Session 2 is drug or placebo WHATEVER we didn't get during session 1 (So if we got placebo during Session 1 then we would have drug Session 2. We are guaranteed to have placebo for one 3-month session, and drug for the other, we just won't know which is which)

You can learn more about CDKL5 here and all the information on the Ataluren trial here.

To simplify this novel I will just say that I called the coordinator, Rayleigh qualified (yay!) and there were just a couple of not-so-minor details to work out.

The first was a decision to be made.  Rayleigh cannot be on any form of cannabis to participate in the trial. Michael and I talked about this for a few days, but in the end decided to stop the CBD oil and go for the trial. In my mind, we wouldn't be looking at trying this new medicine if CBD was doing everything we hoped anyway, right? 

The second was financial. The foundation that was originally on board to cover all travel expenses for the CDKL5 and Dravet families participating from out of state dropped out. The trial requires at least 8 trips to New York City for the checkups, labs, medicine, forms, etc. There is an amazing foundation we learned of through the NYU Comp. Epilepsy Center called FACES. The FACES organization will provide the hotel lodging for every one of the trips necessary - and in a hotel very near the epilepsy center!

But even with the lodging covered, there was NO WAY Michael and I could afford for the 3 of us to fly to NYC that many times in a year. It just isn't feasible. I looked online for foundations and grants and was overwhelmed, but nothing that seemed to provide what we needed. I reached out to Rayleigh's neurologist, desperate for advice, and she reminded me of the Isaiah Stone Foundation. I emailed them and filled out a grant application for the travel expenses for each trip and it was fully accepted!! Because of them we were able to accept Rayleigh's spot in the Ataluren clinical trial!!!

Whew. At this point, September 2017, we are more than halfway through the trial - we have 5 trips and appointments behind us.

Session 1 started in May and ran until July. Then there was a 4 week washout period in which we stopped the trial medicine and only gave her her usual medicines that she's been taking for years. Then about 3 weeks ago we went back to NYC and Rayleigh started Session 2. I won't go into speculation of what I think she's on now vs. Session 1 here on the blog because everyone is having different results and I don't want to set things in other trial participants minds. What I will tell you is that Session 2 ends in November and then we get to come home with FOR SURE Ataluren and administer that for 6 months knowing what she is getting. I am really ready for that section of this trial, you guys!

This has been an exciting, nerve-wracking, tearful, joyful, hopeful journey. My girl is so very brave and stronger than words can describe. 

Each trip we try to squeeze in a little fun for Rayleigh. Some trips have been more seizure-filled than others so we keep those days low key. We just every moment with Rayleigh to be special for her. She has seen Times Square, loves Central Park, she has enjoyed shopping, and on our last visit we took her to the American Museum of Natural History and she really liked that! Our girl loves going for neighborhood walks or walks around the parks and these trips are like that for her - but on steroids! We walk everywhere and there is so much for her to see. 

We feel so fortunate to live in a day that science continues to push back against these terrible seizure disorders, working to find treatment. And we are just thankful for this opportunity to give Rayleigh a better quality of life. 

I'll leave you here some of my favorite pictures from our clinical trial adventures in New York City!

Trip #1 April 2017 - Rayleigh LOVES the flower shops on the sidewalks

View from the plane on trip #2

Inside Grand Central Station

Some appointments are long, administer meds wait for reaction, draw blood, etc, so we bring her portable DVD player along, visit #3 she watched Moana between blood draws

Rayleigh loved the penguins at the Central Park Zoo!

She loves when the seatbelt light comes off and she can lay on our laps on the plane!

Your love, prayers and good vibes are always, always appreciated! And I want to give a HUGE shout out to all of our family and friends that have stepped in and stepped up to help us make this happen. No deed has been too small or unnoticed - from providing Rayleigh's in-flight entertainment to caring for our 2 puppers and the house during each trip we make. We love you.

Lastly, please consider making a donation to any of the foundations I have mentioned here, the CDKL5 foundation, FACES, or the Isaiah Stone Foundation! We are proof that these foundations really make a difference to so many lives!! 

Disney Disney Disney

Those of you who know me well know that I do not like to dwell on the sad things in life. I like to stay hopeful, optimistic, and most of all, HAPPY! And the thing that makes me most happy is when Rayleigh Bug is happy!!! And we are so fortunate that she has had so many happy moments these past couple of months!

Michael and I saved up and got tickets for Disney on Ice! We went to the box office to buy the tickets so that we could see where the seats would be and the wheelchair accessibility. I am SO glad we did! Rayleigh's (and her parents') seats were in the second row! We bought the tickets right behind the VIP section so that Rayleigh could easily keep up with the motions and make out the characters. We were telling Rayleigh all about what the show would be like and even found some video previews to show her. I think she knew something exciting was coming up! 

The day of the show she had occupational therapy in the morning and then took a nap and then she was ready to go!! We opted for the evening showing because we knew we'd have the best chance of her being alert at that time - and for the first time in forever, things finally went her way! {See what I did there with the Frozen music? hehe!}

Frozen Clothes from Bow to Toes!

Disney on Ice is during our State Fair so we spent a little time seeing the sights before the show. Fair Selfie!

She got her face painted at the fair to really really get ready for the show!

The show was about 2 hours long. She was TOTALLY ENGAGED the entire time!! She did not have a single seizure during the show! She was actually wiggling and doing her amazing, excited squeal!! I'm not going to lie, I totally teared up a couple of times when I saw how stinking happy she was! It is the most heart-warming memory I can think of. That Rayleigh. She loved Disney on Ice: Frozen!

So totally into it!

Rayleigh loved when the snow fell! And we were close enough that some flurries even landed on her!

Which takes me right to her Make a Wish. Make a Wish is a wonderful organization makes wishes come true for children with life-threatening medical conditions. Our sweet Bug has been approved to have her wish granted! We went over a list of things she might like or greatly benefit from; like a bedroom makeover, backyard play area, iPad and accessories, and of course, Disney. We just kept going back to a trip to Disney World. Rayleigh loves princesses, Mickey Mouse Clubhouse, Disney movies and she also loves being outdoors, walking around, and swinging, spinning, and bouncing. 

After seeing how much Rayleigh enjoyed Disney on Ice: Frozen, we KNEW Disney World is what Bug wants!! 

We recently met Rayleigh Bug's AMAZING Wish Sponsors, McClayn and Nicole. I'm not exaggerating when I say amazing, you guys! They have such big hearts and want to make this Wish as best as it can be for our girl! We are soo happy to have them! 

Rayleigh with McClayn & Nicole - they brought her those Minnie Mouse ears!

When we met with them, they said that we could answer 4 Wish questions to best determine Bug's Wish. Obviously, Disney was already a major possibility. You fill in the blank for: "I Wish to Be...", "I Wish to Meet...", "I Wish to Go...", and "I Wish to Have..."
Here are Rayleigh's answers:
1.) I Wish to Be a Princess
2.) I Wish to Meet a Princess
3.) I Wish to Go to Disney World
4.) I Wish to Have an iPad


Well, Michael and I decided that we can save up for a little while and eventually get her an older generation iPad - but we could never ever do Disney World the way that Make a Wish can do it for Rayleigh. So there you have it. The Cains will soon be taking their girl to Disney World with a huge thanks to McClayn, Nicole, and the team at Make a Wish Oklahoma!!! YAY!!!

So Many Changes in a Few Short Months

Our family has been very busy keeping up with lots of moving parts in this journey we are on! Since my post in July Rayleigh has started school, is completely off Banzel, now gets real food through her feeding tube instead of formula, is on a slightly increased dose of Charlotte's Web, had family photos done, went to Disney on Ice, has in-home nursing, went on her first field trip, and is getting a wish granted by Make a Wish of Oklahoma! 

Whoa.

Real quick, here are some of my favorites from this year's family photo session!

Family photos for us are bittersweet. We love love love capturing our beautiful girl in pictures to make memories that will last forever. On the other hand, it is one of those things that makes us painfully aware of her disabilities. Obviously, no one is more aware of her disabilities than Michael and I, but we are so involved every single day that it has just become life. A beautiful, meaningful life that we love belonging to. But several things in life make us think, "Dang, that was hard". For family photos, it was because we couldn't just sit her on the ground and tell her to smile, or pick any pose we like. Every single pose had to be thought through: "How will we hold Rayleigh?", "Can't do that, she doesn't have enough head control", "She's too big for that kind of pose but not able to do the older-kid kind of poses"... 
Luckily, we had a wonderful and very patient photographer who would allow us to get in poses we typically do with Rayleigh or ones I found online that I thought we could do. Some worked out great, others... not so much. Nevertheless, I am so pleased with this year's batch of family photos and am so grateful to have these memories! 


Now to the update: Poor little Rayleigh's body is still adjusting to the medicine wean of Banzel. It has been about 1 month since her last dose. We weaned very slowly and the wean went really well. We saw very little withdrawal, and when seizures would increase from withdrawal she would stabilize and get to her baseline after only a couple of days. She is still very tired after seizures and even when she only has a few a day, the post-seizure-naps can wreck up a day. We tried to take her to the science museum on Monday but as soon as we checked in she had her first big seizure of the day so we turned around and came home so she could get a good nap. This is not abnormal for our life.

After the final dose of Banzel we saw withdrawal symptoms come and go for several weeks. At this point, she has had 4 good days in a row so we are hopeful that she is fully stabilizing (knock on wood!). She is having less than 5 seizures on a good day, but still up to 10 of her bigger 3 minute complex partial seizures on a bad day. Two weeks ago we increased her Charlotte's Web Hemp Oil by a tiny amount, Rayleigh starts to show changes from the hemp oil at around 3 weeks so I'm excited to see what comes about - because, again, she has had 4 good days in row ;) 

School is going well! Buggie currently goes half days as we don't think she could tolerate a full day of activities right now. She loves the music, art, circle time, and of course being around people! 

First Day of School - Yes, she was asleep when we left home!

But she woke up when class was about to begin!

Her first field trip was last week and I think she really enjoyed it! It was a windy day at a Pumpkin Patch farm. Rayleigh got to pet different animals, pick out pumpkins and go on a hayride with her school friends!

I'll do a separate post on Disney on Ice and Rayleigh's Make a Wish. They are too much fun to squeeze into an update-post! I'll just leave you with this sneak peek picture from the day we met Rayleigh's sponsors! 

The Perfect Summer Day

Yesterday was the Fourth of July; our flag was proudly flying in the wind in front of our house, the sun was shining, and the heat was on! 

Rayleigh's dad had to work a little in the morning, so Bug and I lounged in bed and watched cartoons for a little bit and then went to the backyard to read and enjoy the beginning of what would be a wonderful day! 

Mommy & Bug Backyard Selfie :) 

Next, because of a seizure, Rayleigh took a nice nap in the cool air conditioning. When she woke up we headed to the mall for an afternoon movie! This was sort of spur of the moment, but our girl was in a great mood and I've been wanting to try the movie theater again. We've only taken Bug to the theater once and it was a disaster! We took her to a very early showtime of Despicable Me 2 for a sensory show - and being in withdrawal from weaning a medicine she was cranky and miserable and just wanted to be home, so we left about 30 minutes into the movie. But that was a couple of years ago and she has been having some really good days so we thought LET'S TRY! 

And we are SO GLAD we did!! She loved it, y'all! We went to see Inside Out! The theater we chose has the wide recliner seats and we were able to get 2 spots and wheelchair spot. Rayleigh watched the first bit in her wheelchair and then snuggled up with her blanket on my lap and we raised the armrest so she could be close to her dada, too! We did her afternoon tube feeding right there, easy peasy. Goodness, I'm still so proud of her, and so happy she was able to enjoy the movie!!! 

Movie Theater Selfie!

We went over to her grandparents' house after the movie for dinner and some swimming! 

We sent Daddy for sunglasses after her snapped a picture! hehe!

Then, of course, fireworks!! We went to a park in a nearby city where loads of people go to pop off their own fireworks! Rayleigh managed to stay awake long enough to watch her dad and Gpa shoot off some pretty ones, but then she fell asleep snuggled on my lap. Her awesome Lovie sat in the air conditioned van with her to finish watching the boys. 
Sorry I didn't take more pictures, I notice at the end of a fun day that I don't take very many pictures of those moments we had - I was so busy living life and enjoying the moment that I forgot to whip out my phone and snap a quick picture to remind me of the fun we had! I'll work on getting better, haha!

I am just soo thankful that Rayleigh got to enjoy the holiday! Today, she is making up for all of that spent energy with a big long nap (hence the time for this update!). 

Home Sweet Homa

Hmmm... Where to start? I guess I'll just dive right in. After months and months of Michael trying to transfer his job to Colorado we thought, "Well, maybe there's a reason this isn't falling in to place despite our efforts." 

That's the small thought that led to a grand discussion and ultimately, the decision to stay in Oklahoma. At least for the foreseeable future. 

Our decision was not made lightly but once it was spoken aloud, there was a wave of calm that went through me. It felt like I had finally released a breath that I had been holding in for a very long time. 

Rayleigh is the most important thing in my life. Do I think it would be beneficial for her to have different options in CBD oils and access to THC oil to use as a supplement to the CBD oils? Sure. Do I think the answer is to uproot and move to Colorado for their supply? Not right now. We have seen some great stuff from high CBD oil use. In fact, Rayleigh is currently taking Charlotte's Web Hemp Oil. She is still having 6-10 complex partial seizures a day, they are still wearing out her little body, BUT she is having random days of only 2 or 3 of these seizures. And, no joking, her eyes are so much more alert. The girl is AWAKE when she is AWAKE! We just recently increased the dose (you have to titrate your way up to an optimal dose slowly) and we have seen a little bit of movement coming back, mostly wiggling her arms or moving her head! 

So, I'm not sure if high CBD oil is going to be our answer for seizure control or not.
But I'm going to keep on trying until I have reason to stop. 

To everyone that donated toward our Colorado move: THANK YOU! You have such loving hearts. That money was set aside and has been/is being used to buy the Charlotte's Web Hemp Oil. Each bottle is $250 plus shipping, so your help is soo appreciated! 

Oklahoma recently passed a bill that allows only high CBD oils to patients under 18 with intractable epilepsy. Rayleigh fits the criteria (duh.) and has her neurologist's blessing. I've got some issues with the new law, the biggest being: How the heck are the patients to get the oil when it's still federally illegal for the providers to ship any form of medical cannabis outside of their state? 

{Charlotte's Web was classified as Hemp Oil this past year and is currently shipping to all 50 states under that pretense. All other high CBD medical cannabis oils that I know of are NOT shipping nor classified as Hemp.}

After Oklahoma passed the bill OU Medical Center announced that they will begin the Epidiolex trial. This is for a big pharma version of medical cannabis, it is very high in CBD and extremely low in THC. I have heard CDKL5 success stories from the clinical trials of Epidiolex in other states so I am going to contact the coordinator and inquire about getting Rayleigh into the study. I will not put her in the study if it is controlled with a placebo. Why would I take her off a for sure high CBD hemp oil to put her in a study with the possibility that she gets the placebo?

In closure, we have a plan with her neurologist to wean her Banzel (one of her seizure medicines). Once Banzel is out of her system we will start either Fycompa or Sabril. She's been on Sabril before with good seizure control but some side effects, she's never been on Fycompa and while it's only been available for about a year I'm hearing good things. Decisions. Hopefully the medicine that we decide on and the CWHO will give Rayleigh enough seizure control that we can get back to weaning Onfi (the monster seizure medicine). 

Thank you for your patience and love while we were in limbo about the move. 
Oklahoma, you have a strong hold on this family!

And now, some pictures from the past year!