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Thursday, November 20, 2014 - 6:49pm

Trial drug for children with rare genetic disorder discontinued: Local family concerned

Photo provided by staff.
Friday, May 24, 2013 - 10:58am

One young boy from Baton Rouge with a genetic disorder called Fragile X has another hardship to face. After his parents got him on a clinical trial with a drug proven to be successful; his condition was drastically starting to improve. But now, the trial is canceled.

“I felt like a rug had been pulled out from underneath me. I felt like I had been punched in the stomach," said Patricia Kisamore.

Kisamore heard her son's medicine would soon be discontinued only a few days ago. It's medicine that's changed his life.

“All I could think of is 'oh my God, what are we going to do now,'" noted Kisamore.

A little over one year ago, she did some research and started her son Logan in a clinical trial for children with Fragile X syndrome. Before the trial, Logan struggled with speech, school and even social interaction.

"With us, it's been language. We went from just a few words to maybe 150 words,” said Kisamore. ”Not full sentences, but we are at 2 and 3 word utterances."

Even more than the language acquisition, Logan was starting to act like any other 9 year old.

“The biggest change we saw was in school. He actually developed friendships with the kids in his class.”

But now the drug that's helped Logan grow will no longer be available, which raises many questions for his family.

"Because he’s come so far and that’s all I could think of. I am going to lose all the words, I am going to lose all the progress that he’s made and I don’t want to lose it."

But no matter what Patricia says, she knows Logan is not the only one in this trial.

“If there is any reassuring thing in it is that we aren’t alone,” said Kisamore.

Patricia and her husband don't know what will happen after he is taken off the drug. They don't know whether he will get worse, improve or stay the same. All they can do is live in the present and hope something better comes along.

“I try not to concentrate on the future right now because I don’t want to miss the moments that we have now," stated Kisamore.

The drug developers, Seaside Therapeutics, released a statement about the canceled trial.

"The study termination is due to resource limitations at seaside, and is not due to any safety issue associated with STX209. We know that this termination will be disruptive and disappointing for many. We are planning to complete phase 3 and the results of this study should be available late summer. If the results are positive, seaside will discuss with FDA the required next steps for approval of STX209.

"This means the drug could potentially become available in the future but there is still nothing guaranteed."

Patricia says other kids who have stopped taking the drug also stopped improving but she says she'll continue to keep an eye out for further studies and just hope Logan continues to improve and beats all odds.
 

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