An American hospital is offering a potentially life-saving hand to one of two Irish siblings suffering from a fatal disease.
Saoirse Heffernan, 4, suffers from late Infantile batten disease, and so does her 22-month-old brother, Liam. Saoirse and Liam’s parents have been told that their children will not survive this very rare neurological condition.
However, hope has come from Weill Cornell University Hospital which has invited Saoirse to New York to assess if she is strong enough to undergo a gene transfer trial that could save her life.
“We got the call last Thursday from one of the coordinators of the trial to say they were pleased to invite Saoirse over for an assessment to see if she would be fit for the trial,” Tony Heffernan, Saoirse’s father, told the Irish Voice on Monday.
Although Tony and his wife Mary had registered both of their children with Cornell for trials, they were unsure anything would come from it.
Receiving the call, said Heffernan, sent joy and excitement rippling through their home in Keel, Co. Kerry last Thursday.
“We were just overjoyed that our persistence paid off and the fact that people out there want to help our children, it really is just wonderful,” said Heffernan, 38.
The Heffernans are now hoping that Saoirse, who travels to New York next week to undergo the baseline assessment of her present condition, will be strong enough to make it through to the clinical trials later this year.
“She will be in for tests for about three days,” explained Heffernan, a ships captain by trade who currently works with Höegh & Co, an international shipping company.
Following medical examinations and testing at Temple Street Children’s University Hospital in Dublin, Saoirse’s results were looked on positively by Cornell’s medial team.
On Thursday, May 27, Saoirse, who has lost 70% of her eyesight from the disease and struggles day to day, will attend Cornell, where her future will be determined.
If she is accepted to the trial, Saoirse will undergo gene therapy, which involves injecting a harmless gene-bearing virus into the brain. It has been found to significantly slow the progression of the disease.
Saoirse’s parents have spent the past few months fundraising to collect as much money as possible to get both children on the clinical trial.
Based on the cost incurred by U.S. families who had children on this trial, Heffernan estimates that up to $500,000 could be spent per child on the trials.
If all goes well next week and Saoirse is strong enough, she will then be put on a list with other children suffering from Batten Disease. If she makes the final cut then a substantial amount of the hospital trials will be paid for.
The Heffernans will, however, be responsible for everything else, including living expenses for 18 months -- the duration of the trial -- in New York.
However, if Saoirse doesn’t make the first cut she may be allowed on a “compassionate use” trial, a program that allows its treatment to be administered to seriously ill patients that are not enrolled in clinical trial. The Heffernans would have to incur the cost of this program.
“We want to be ready. If we get accepted we want to have the funds there to save our kids’ lives,” said Heffernan.
Tony, originally from Cork, and his wife Mary, from Kerry launched a new charity website, www.beeforBattens.org to raise funds for the children, and to increase the awareness of this cruel disease.
Although the Heffernan children are the only two Batten sufferers in Ireland at the moment, the charity will continue to raise money and be available for future Irish children who may suffer form the disease.
“It’s also creating awareness about the disease and that’s important,” said Heffernan.
After Saoirse and Liam’s story ran in the Irish Voice two weeks ago and subsequently on our sister website, IrishCentral.com, funds have come pouring in from the U.S.
“Thanks to all the fundraisers here and the kind hearted donations from people in America we have now raised close to *****150, 000,” Heffernan said.
“There is even a woman with children who suffer from Batten that wants to set up a U.S. version of Bees for Batten,” he added.
The only other sufferer of this disease in the Irish Republic is Saoirse’s younger brother, Liam, and the family is hoping that he will follow his sister to the U.S. later this year or early into 2011.
“Liam is still too young for trials, but we are hoping that by the end of the year he will get a call for an assessment like Saoirse,” explained Heffernan. Initially diagnosed with epilepsy, Saoirse has been in and out of hospital since she was young with aggressive seizures, until finally last September it was discovered that she had a disease that would end her life.
Just before Christmas last year, Saoirse was given two to three years of life expectancy.
Losing their child wasn’t an option. Immediately after diagnosis, Heffernan began searching for cures or programs worldwide that could give his little girl a chance in life.
While the Heffernan’s were frantically trying to find help for their eldest child, another blow came their way in March -- Liam too was confirmed to have the fatal disease.
Looking back on their willingness to save their children’s lives, Heffernan said, “If we had sat back and didn’t go about finding out information about the possibilities of saving them and creating a charity we would be suffering and in a very bad position today.
“I’m just glad we have the foresight to drive ahead when we needed to and now it’s paying off with the news of the assessment in New York,” he added.
Heffernan is very grateful to the Kerryman’s Association of New York which has taken on the responsibility of looking after Tony, Mary and Saoirse (Liam will remain at home with family) while they are in New York.
“Bill O’Sullivan and the Kerryman’s Association and the Kerry and Cork GAA teams have been superb to us, and we are looking forward to meeting them all when we get there,” he said.
To donate to the Heffernan’s effort to save their children’s lives and to help others in the future who suffer from the fatal disease log onto www.beeforBattens.org.
Jackie Kennedy’s granddaughter has uncannily similar looks