In 2004 a clinical trail was launched at Cornell to study a gene therapy method for treatment of the signs and symptoms of Batten’s Disease. An experimental drug works by delivering a gene transfer to the brain. In 2008 the procedure was deemed safe.
The Cornell trial is to begin at the end of the year. According to Heffernan, they usually only accept eight to 10 children.
Both Saoirse and Liam are registered for the trial, but neither has been confirmed and won’t be for another few months.
“The children have to be very strong for this trial, and thankfully Liam is at the minute, and although Saoirse is not so strong she is a real little fighter and we hope she will make it onto the program too,” he said.
According to Heffernan, there are only three children in Ireland alive with Batten’s Disease.
“That’s our two plus one other child. I’ve already been in contact with at least six parents who have buried their children from this,” he said agonizingly. “We don’t want this to be us too.”
Now the Heffernans are putting all their focus into raising as much money as possible to get their children to the U.S.
“It costs about $500,000 for each child to attend the trials,” he said. “We’re not even one-tenth there for one child but we will keep going.”
“People have been fantastic here in Ireland, especially in Cork and Kerry holding several fundraisers raising great money,” added Heffernan.
Heffernan also praises Billy O’Sullivan from the Kerrymen’s Association in New York who contacted the family to offer their support.
“We are not accepting death. We are going to push hard to do this for our children. We have to it’s as simple as that,” he said.
In the meantime the Heffernans are doing their best to give their children a normal life. Although Saoirse can’t go outside much -- problems with her eyes makes her sensitive to light -- the family work and play close together and try to maintain as much normality and stability as they can.
In their spare time, the Heffernans have set up a charity called the Saoirse Foundation to help raise the necessary funds to get their children to the clinical trials.
For more information on Batten’s Disease and how to donate money log onto www.beeforbattens.org.
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