Fragile X drug trials to start for Luke
About 9 or 10 years ago (Paul and I were still married), we went to a Fraxa meeting that brought families together with Fraxa researchers. I was talking to one of the scientists about his work and he basically said, "Well, Luke will have to play catch-up, but then he'll be normal." Now I had half finished my first glass of wine, so I wasn't sure if I had heard correctly.
"Could you repeat that? I thought I heard you say 'normal'". "I did." And he repeated, "Fragile X is not brain damage. He will need to play catch-up, but this drug will help him function normally."
"WHEN CAN WE START???!!!"
And every year since then, we have been told that the drug trials for adolescents would be a year or two away.
In November, 2011, we learned that the Institute for Basic Research (IBR) in Staten Island was undergoing a review from it's Independent Review Board (IRB) to be a lab that offered the promising AFQ056 (mGLur5) drug trials to adolescents with Fragile X. My contact at the IBR, Dr Angelo Porto, promised that by Dec of 2011, or at the latest, Jan 2012, Luke would be enrolled in the study.
December came and went. And January, And all of 2012….WHEN will this happen???
Finally, the IBR got approval and Luke has his first appointment on Feb 28 at 9 am. The trial will last 4 months and is a double-blind study. At some point, for 4 weeks, he will get a placebo. And for the remaining 12 weeks, he will get either 25%, 50% or 100% of the drug AFQ056.
WE have to visit the lab 6 times over 4 months. Blood must be drawn and ECGs must be performed. But after the 4 months, an extended study guarantees him the drug for 2 years, for free.
Already 27 sites have recruited adolescents for Phase II of this promising drug. And word has it that some young men (and women) are behaving in neurotypical ways they never expressed before. Less anxiety. Learning. Improved learning and focus. And talking. Talking….what is Luke going to say? What has he been thinking for 14 years? What miracle could make my beautiful, tacit child express his feelings, thoughts, opinions.
Feb 28 – I can hardly believe 12 years of waiting is finally going to be over……….