But devoted parents, Tony and Mary Heffernan, were devastated at the weekend after American doctors told them, Saoirse, five, who has the rare neurological condition Batten’s Disease, is not suitable for an experimental medical trial subsidised by the US federal government.
The parents, from Co Kerry, are in a race against time to raise another $250,000 (€180,000) to secure Saoirse’s place on a US hospital-run parallel trial later this year.
“We fear it’s Saoirse’s last glimmer of hope,” an emotional Tony said from New York last night.
“We’re devastated – still coming to terms with the news. It’s really a matter of life and death now,” he said.
Saoirse and her little brother, Liam, two, are just a handful of Irish people with Batten’s Disease – a rare and fatal neurological disorder.
After months of fundraising, the family took up an invitation last Monday to attend New York’s Weill Cornell University Hospital for tests to determine Saoirse’s suitability for participation in an experimental medical trial due to start tomorrow.
She underwent a barrage of physical and neurological tests but world-renowned Batten’s disease expert, Dr Ronald Crystal, told Tony and Mary that her condition has progressed too far.
She no longer meets the strict verification criteria for the phase-one treatment trials, he said.
“We came all the way out here in the hope that Saoirse still met the criteria since her last test in May,” Tony said.
“But she has obviously slipped back. We know it’s a progressive condition but she’s deteriorated faster than we thought.”
But Dr Crystal has given the family some hope. He said Saoirse meets the criteria for a parallel treatment trial due to start at the hospital in late November or early December.
But Tony and Mary, who have already raised $500,000 in just six months for Saoirse’s treatment, must raise another $250,000 for this trial.
“If I don’t get my daughter on this trial, I’m going to bury her,” Tony said.
“Someone has put a price on her head. We managed to raise $500,000 within six months and we had the public behind us. We now have to go back and ask for more. Our backs are to the wall, guns to our heads. I’m going to knock on every single door to get the money.”
Liam has already been accepted for the subsidised trial and is due to start his treatment next summer.
The family is due back in Ireland on Wednesday when Saoirse will return to Temple Street Children’s Hospital for a full check-up and adjustments to her medication.
Batten’s Disease is an extremely rare neurological condition.
There are less than a handful of sufferers in Ireland, with only 750-1,000 sufferers worldwide.
To date, it has always been fatal and the US trials are designed to find a cure.
* Donations to: www.beeforbattens.org; any AIB bank, a/c no: 06040095, sort code: 93-63-24; or by cheque to BeeForBattens, Castledrum, Keel, Castlemaine, Co Kerry.