Nine weeks after burying their only daughter, Tony and Mary Heffernan from Co. Kerry have welcomed the news that their two-year-old son, Liam, has been invited to participate in pioneering brain surgery in New York that may save his life.
Liam, from Keel, Co. Kerry, was diagnosed with Late Infantile Batten Disease a year ago after his sister Saoirse, five when she died in January, had been suffering from the fatal disorder for a few months.
Liam will become the world’s youngest child to participate in the pioneering second generation human medical trial which has recently commenced at the prestigious Weill Cornell University Hospital in New York. A date has yet to be set.
The invitation for Liam came exactly nine weeks after his sister Saoirse passed away in her parents’ arms from the same rare, fatal disorder. The family, who just returned to Ireland last week following an assessment at Cornell, received the good news by phone on Monday evening.
“They told us Liam had successfully met the strict criteria to allow his participation in the trial,” said his dad Tony.
Favorable medical examinations and testing at Temple Street Children’s University Hospital in Dublin and at Great Ormond Street in London gave Cornell the go ahead to invite the toddler for testing in the U.S. last month.
The brain surgery involves drilling six holes in Liam’s skull to allow the gene transfer treatment to be administered to 12 locations in his brain.
Since the trial began in August 2010, only three children worldwide, all of whom are American, have received the gene transfer.
The trial coordinators have recently gained permission from U.S. federal authorities to treat Liam following their application to reduce the participation age below the three-year-old minimum age requirement.
Last year Saoirse was invited to participate in the same assessment, but due to a rapid deterioration in her health over the summer the invitation was retracted.
The Heffernans were beyond heartbroken, and after returning to Ireland armed with another plan Saoirse grew sicker until finally nothing could be done for her medically.
After an emotional appearance and plea on Ireland’s longest running talk show, The Late Late Show last year, Tony and Mary founded Bee for Battens in an effort to raise the necessary funds to send their children to the U.S. for the trial. The money collected also helps the other families in Ireland who have children with the disease. Since that time the foundation has raised hundreds of thousands of dollars.
There are only approximately 750-1,000 sufferers of Late Infantile Batten Disease worldwide, with only a handful in Ireland. Bee for Battens, which recently celebrated its first anniversary, has been very active in raising awareness of Batten Disease in Ireland and abroad.
It will continue its fundraising campaign to allow the participation of other Irish children members in medical treatment trials, to fund research, and to provide support to other Irish sufferers and their families.
In New York a charity has also been set up on the back of a fundraiser held in the Kerry Hall in Yonkers, New York, last September where over $120,000 was raised for Saoirse and other Irish children with the disease.
To donate, checks can be mailed to the Saoirse Foundation, Kerrymen's Association, 307 McLean Avenue, Yonkers, New York 10705-4488.
The charity’s website www.beeforbattens.org also accepts donations and works to increase the awareness of this cruel disease.