NBC10- Luann Cahn
Parents say an experimental drug that works miracles for their children has been taken away after a study suddenly ended this month. Now, those parents are desperately petitioning lawmakers and drug companies to help raise awareness and funds for this study. NBC10's Luann Cahn reports.
Mike and Lynn Berman say an experimental drug turned out to be life-changing for their daughter, who suffers from a debilitating genetic disorder called Fragile X.
Her parents say the disorder causes their 13-year-old daughter Hannah to be isolated, locked in her head and unable to socialize or do school work.
So when a small company called Seaside Therapeutics offered Hannah and dozens of other children the opportunity to try an experimental drug called arbaclofen, her parents agreed.
They say the medicine worked miracles for their daughter. "It's almost a cure for us, this medication. It's our ticket to the true Hannah," said Lynn Berman.
Dr. Shivkumar Hatti, of Suburban Research Associates, participated in the drug study. "It seemed like the longer they took the medicine the better result we got," said Dr. Hatti.
But this month, the study suddenly came to an end. The Berman's were one of many couples to receive a letter in the mail from Seaside Therapeutics informing them they would no longer be able to receive the drug for their daughter.
"It is due to resource limitations at Seaside, and it is not due to any safety issue. The studies we have completed do not provide sufficient evidence for FDA approval," the letter read in part.
All the parents did sign consent forms that said this could happen, but it didn't make the news any easier to handle.
"You just lose the child that you had," said Mike Berman.
The Vice President of Seaside Pharmaceuticals tells NBC10 they need additional resources to continue their work. They are hoping to get funding for more testing and approval from the FDA to market the drug.