Caleb Welin (Cambridge, MA)

In April of 2006, my son was diagnosed with Fragile X Syndrome at the age of 22 months. Caleb is an amazing boy who is full of life and love but he struggles to share that with the world sometimes because of the effects of fragile X.

For the last seven years my husband and I have been waiting for the cure we were told that we would have in our lifetime. Last fall we took steps to participate in a drug study for STX 209, a drug being tested by Seaside Therapeutics, Inc. to treat the core symptoms of fragile X.

We were uncertain about participating in the trial initially but we heard such promising stories from other members of the fragile X community that we decided to take the chance. My husband drove Caleb to the study location many times over the course of the trial, sometimes we had to take Caleb out of school early for the visits. We decided some missed school was less important that what we could gain from taking part.

When we completed the double blind placebo controlled Phase III study we enrolled in the open label extension with no second thoughts. We have seen amazing progress for Caleb since that time. His language has improved, he is more social, he is able to do things, like go to the circus that he could never do previously.

And then I learned that the extended trial of STX 209 is over. We had heard rumors of money troubles but we all hoped for a solution and some advance notice. We didn’t get either.

I woke up the next morning and stood crying at the counter while making Caleb’s breakfast, which is always accompanied by a spoon of applesauce and his morning meds, knowing that we were one dose closer to the end.

I’m not even sure where I should to begin. I’m wracking my brain trying to find the answer. I know nothing in this business is guaranteed, there’s a lot of money pharmaceuticals because it’s expensive work. Changing the world takes lots and lots of money.

I refuse to believe that this is as far as the story goes. I’ve never expected a fairy tale ending, but this defies logic. We are almost there. In the face of incredible odds, Seaside has gotten us to the point where we can see the goal line. Now we need that one person to stand up and either clear the way or give them a nice big push from behind.

One person, that’s it. Please help me find that person.