Tuesday, February 19, 2019

A New Beginning

Just before we left, the director of the first-ever clinical trial said she thought we should take a picture with Grace of the momentous occasion that she tried her first does of RT001. And of course, that was right up our alley, so we grabbed Flat Gracie and posed for a photo.

Getting ready to fly to New Jersey.
In some ways, it marked the end of what seemed like very long journey. We first learned about Retrotope and their experimental drug, RT001, more than a year ago, when they published a press release online that there were two INAD patients taking the drug as part of a compassionate use program. The drug company has claimed that the drug is able to stop or at least slow the progression of INAD and can also help to reacquire lost skills. We immediately contacted the company, and discovered they were hoping to do a clinical trial for the drug in INAD patients, and that Grace would likely be a candidate.

At the time, they told us the trial could start as early as March of next year. We learned that things like this often times take much longer than we would like. When it didn't start in March, we heard July or August, then October, and we got a call after the first of this year that Grace would be able to travel to New Jersey and participate in the clinical trial.

Grace shortly after taking her first dose of RT001
Kyle and I have spent months and months debating whether or not to enroll Grace in the trial. At seven and a half years old, she's the oldest patient they have right now, and the cut-off age for participation is ten. We know this drug is not a cure, and that the positive effects we see may be minimal. But you never, ever know how something is going to play out, and we wanted Grace to have a chance to try this new drug. We also wanted to be able to further INAD research, and since we didn't feel the drug would have any harmful side effects to her, we finally decided to go for it.

In January, we made a week long trek to New Jersey, driving the 13 hours and taking Grace to three days of baseline appointments. It was a long week for sure, but we learned a lot, and we are so grateful that Grace is a good traveler and we met lots of nice people.

I think the hardest part of participating in the trial is that we had to return to New Jersey in two weeks to 45 days after those initial tests so Grace could have another baseline assessment and officially start the use of RT001. This visit was likely to last only part of a morning, so instead of driving the 900 miles, we opted to fly and do the trip in two days.

Checking out the New York City skyline!
We haven't flown with Grace in nearly a year and a half, and both of us were a little worried about how it would play out.   Since Grace can't sit up or hold her head up, it makes it tricky to sit her in her own seat buckled up for the take off, and the last time we flew with her, the airline broke her medical stroller.

But we pushed those worries aside (mostly), and decided it was the best decision since our trip was so short. And we couldn't be more pleased with the way everyone took care of us. When I texted that to Kyle's mom on the way home, she wrote back that Grace just seems to bring that out in people, and I couldn't agree more.  We flew Southwest, where everyone took amazing care of us. From checking us in and setting us up for pre-boarding to making sure we had everything we needed, we were very comfortable on both flights. Grace was even able to sleep across my lap.  We rented a minivan from Enterprise, which was also a wonderful experience and drove it to our favorite Hampton Inn in Denville, where the night clerk offered to say a special prayer for Grace during his morning devotional. We are continually blessed by so many that happen across our paths on this Grace-Filled Journey.

This morning, Grace had another baseline assessment, and they showed us how we can administer the drug to her each day. It's a little overwhelming. Clinical trials require a lot of data and record-keeping (and I can't say those things fall in my strong suit). But we are excited that she finally had a chance to try the drug that has helped some INAD kiddos.

A view of Lady Liberty.
I am SO proud of Grace for being part of this initial clinical trial. She has been so strong and brave through each and every exam, each and every assessment and even trying the medicine this morning. She never complains and always has a smile for everyone we encounter. She reminds us that life is always to be celebrated and to life each and every moment to the fullest. Oh, and that a smile can brighten everyone's day.

Before we headed for home, we had a little time to kill before we got to the airport. As he was driving, Kyle saw a sign for Liberty State Park with viewings of Ellis Island and the Statue of Liberty. How could we pass up an opportunity to take in such great sights? Although it was too cold to take Grace on a ferry ride to see the statue up close, we walked around and looked at the New York City skyline and stopped to see Lady Liberty. What better place to start a new adventure than at a place that marked one of the beginnings of our country.

Ellis Island and the Statue of Liberty.
So, in a way, Grace getting to try the drug marked the end of a long journey, it also marked a new beginning. Over the course of the next year, we will continue to record when she takes the drug and any progress she makes along the way. We also have two more trips to New Jersey at the six-month mark and the one-year mark to assess any improvements or changes.  Next time we go, we hope to take Charley with us and do a little sight-seeing in the area.

We won't know for awhile if RT001 will make any difference for Grace. Maybe it helps her a little, and maybe it doesn't. But we know that it's a chance we took to give Grace every single chance there is. And we are SO grateful to each and every person who helped us along the way on this amazing Grace-Filled Journey.


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