One of the two siblings suffering from a fatal disease in Ireland spent two days at Cornell University Hospital in New York last week to assess if she is strong enough to undergo a gene transfer trial that could save her life.
Saoirse Heffernan, four, 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 condition.
However, hope came from Weill Cornell University Hospital in New York a few weeks ago when an invitation was extended to Saoirse (Liam is still too young) for an assessment to see if she would be fit for their ongoing trails.
Although Tony, originally from Cork, and his wife Mary, from Kerry, had registered both of their children with Cornell for trials, they were unsure anything would come from it.
Tony, 38, told the Irish Voice on Tuesday that the assessment went well, but they won’t know if Saoirse will fit the criteria for trials for another few weeks.
“Some parts of it were a bit overwhelming to her because of tiredness and jetlag, but she did well. We hope to have news of her results in the next month or so,” Heffernan said.
Saoirse spent last Thursday and Friday in the hospital and was in good spirits.
“Overall she was in great form,” said Heffernan, a ships captain by trade.
The Heffernans remain positive.
“The good news for us is that Saoirse was the seventh child to be screened (out of hundreds) so we are hopeful.”
Heffernan added, “Plus we had some positive news from individual doctors, some saying that Saoirse had one or two attributes that were stronger than other candidates so that news was very well received by us, but of course we are not hanging our hat on it.”
If 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.
While in New York the three Heffernans (Liam stayed at home with family) were looked after very well by families from Kerry and Cork.
“We are very grateful to the wonderful hospitality we were shown by Billy O’Sullivan and family, Tom Kennedy and family and Mary’s neighbor Paidie Hanley who all fed us, gave us a bed, and looked after us very well,” said Heffernan.
“It’s wonderful to know we have so much support across the Atlantic.”
Saoirse, who has lost 70% of her eyesight from the disease, fell in love with the O’Sullivan’s dog Molly, and for her fifth birthday on Friday her parents are buying her a Molly-like dog.
“She absolutely loved Molly so we told her we would get her her very own Molly for her birthday and she is over the moon,” Heffernan said.
Saoirse’s parents have spent the past few months fundraising 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 Saoirse makes the final cut for the trials 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.
The Heffernans launched a new charity website www.beeforbattens.org to raise funds for the children, and to increase the awareness of this cruel disease.
A committee has been set up in the Bronx/Yonkers area to organize an extensive fundraiser for Sunday, September 26 at the Kerry Hall in Yonkers. Details of the fundraiser will be released in a few weeks.
To donate to the Heffernans’ 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.