Friday, February 1, 2019

Taking A Chance On Grace

Last Sunday afternoon, Kyle, Grace and I embarked on a new adventure on this journey, a trip to Morristown, New Jersey, to take the first step in participating in a clinical trial for a drug called RT0001 with a California-based company called Retrotope. 

Grace took part in the first-ever clinical trial for INAD.
It's the first-ever potential treatment for INAD using a D-PUFA (deuterated polyunsaturated fatty acid) that could help to prevent against lipid peroxidation damage and help to restore cellular function. In case you're wondering, I got that from the Retrotope website, and I know about as much as it means as you do.

Grace was so brave during three days of testing.
But in some layman terms, INAD causes a lipid to build up on the end of Grace's nerves in toxic levels. That causes the nerves not to function, and is why Grace can't use her arms or legs. It cause the atrophy of all her nerves, including the optic nerve, which is why she struggles with her vision as well.

We made our home away from home in our favorite Hampton Inn.
The drug, RT0001, is supposed to help break down that build up on the end of Grace's nerves. And as far as we know, doesn't have any harmful side effects.

So, after months and months of discussion, Kyle and I decided to let Grace be part of the trial in hopes it would help slow the regression of INAD. 

Charlotte spent the week with Grandma and Grandpa.
So, that Sunday, we packed the van and loaded up for then nearly 900-mile drive. The first visit would include six testing sessions in various locations, so it was much easier for us to have our van and be able to bring the things Grace would need to be part of the trial. It was definitely a long couple of days, but we are lucky that Grace is such a good traveler. And I'm always grateful that Kyle does most of the driving.

We enjoyed FaceTiming with Charlotte while we were gone.
Our first day in Morristown included some baseline assessment testing, done on video. We provided all of Grace's medical history, including what ages she lost certain skills. I had to get out her baby book to remember a lot of it! But the assessments included things like, could Grace sit up with support, could she try to roll over, could she raise her arms? It was documented on video because they are hopeful that this drug may improve some of those skills. That's not saying that being on this drug will help Grace to roll over, but the drug company can show small results too, things like Grace trying to roll over is an improvement, even if it's only a tiny improvement. 

We loved meeting other members of the INAD community.
They also spent a lot of time going over what would be required of us in the coming year as part of the trial. It's going to be tough. There's a daily food journal, video updates and check-ins, as well as additional visits to the site for a repeat of these assessments at six months and at one year.

INAD moms have a special bond.
The other testing included a nerve conduction test, a hearing screening, an EEG, blood work and an eye exam. All of the tests looked at Grace's nerve function. Grace had never had many of these tests done before, and none of them were invasive or hurt her. Kyle and I asked lots of questions throughout about each of the exams. Those exams will all be repeated in six months to see if the nerve function has improved any.

One of the fun places we ate was a hibachi restaurant, and Kyle got sushi.
All of the people helping with the trial have been wonderful to work with. They helped us navigate a tough week, and we will work with them over the course of the next year.

I think the hardest part of the whole experience was confronting the fact that Grace's nerve function is so poor. I mean, it's not like we didn't already know that Grace had lost most of these skills. But the hearing test showed there was no response from nerves in her ears to her brain stem. The same thing was found with the nerves in her arms and legs. 
Most days, that's not something I think about. Most days, we wake up, do the things we need to do to take care of Grace and try to provide the best life for her. But we don't stop to think about all that's been taken away from her. And for that, I'm grateful. Most days, we simply love Grace and don't worry about INAD, or the fact that it continues to take it's toll on her beautiful body.

Home sweet home.
In the end, I think that's why we agreed to be part of the study, despite the monumental amount of work required to participate in the trial. We want to give Grace every chance there is. And we hope and pray it helps her and all the INAD kids in some way.

But the trip wasn't all work any no play. We managed to stop at some really fun restaurants along the route. Kyle is always partial to the Pennsylvania-based Primanti Brothers chain, and one night, we visited a Japanese hibachi restaurant for sushi that was so much fun.

We also had a chance to meet other members of the INAD community, as we had lunch with the Panwala family. Their oldest daughter, Ariya, also has INAD like Grace. Although we are part of an INAD Support Group on Facebook, we've never actually met any other children or parents. It was fun to connect, and we enjoyed the special visit. Also joining us was Barbara, whose grandson, Leo, is affected by INAD. Although it's definitely not a journey anyone would pick, we truly have met some very special people along the way. And for that, we will always be grateful.

As we crawled into our nice and cozy bed this evening, we are very happy to be home, and excited for this new opportunity. We will take Grace back to New Jersey in February, where she will have a chance to try the drug as well. We're not sure what type of results she will see, but we're so glad to take a chance on Grace. 


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