Irish child with killer brain disease denied life saving treatment in New York

Heffernan family must raise an additional $250,000 to enter parallel trial

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Saorise Heffernan (5) suffers from Battens disease

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Five-year-old Saoirse Heffernan from Co. Kerry has had her invitation to participate in a medical trial at Cornell University Hospital withdrawn after failing to meet surgical criteria -- but if the family can raise an additional $250,000 in the next few weeks she is guaranteed a place on a parallel trial at the end of the year.

Saoirse, who has a rare and fatal neurological disease called Late Infantile Batten Disease, flew from Ireland last week with her family to participate in a phase-one medical trial at Cornell that was believed to prolong if not save her life.

However, after undergoing a series of physical and neurological examinations at Cornell on Thursday, October 7, the news was bad.
Saoirse is no longer strong enough to participate in a scheduled surgery due to take place this week.  The young Kerry girl had qualified for the trial last May but has since deteriorated.

Her parents Tony and Mary Heffernan were distraught by the bad news.

“We were beyond devastated to learn that Saoirse would not qualify for the trial,” Tony told the Irish Voice from New York on Monday.

“She narrowly missed the federal criteria that had to be met, and we had all our hopes pinned on the trial which were immediately shattered with the news.”

The Heffernans were informed by Dr. Ronald Crystal, a professor of medicine at Cornell Medical College who is regarded as one of the world’s leading authorities on Batten Disease, that Saoirse had progressed too far for the trial.

“Mary and I were all geared up for Saoirse to begin the trial so it was very upsetting, but I guess now we just have to get on with it and hope for the best,” said Tony, trying to remain optimistic. 

However, Crystal offered alternative hope to the family, but at a cost.

The doctor told the Heffernans that Saoirse does meet the criteria for a parallel treatment trial due to start in November/December of this year.

“We’ve been given a glimmer of hope with this parallel trial, it’s the same treatment, the same guy carrying it out and in the same location. It will just be administered differently with less time in the theater,” explained Tony.

Although happy that their daughter has some hope, the family are now faced with trying to raise an additional $250,000 in a matter of weeks to pay for the phase two treatment trial.

In total the cost of the trial will be $750,000, $250,000 more than they had originally been advised.  To date they have raised $500,000.

“If we don’t raise the rest of the money Saoirse will become another statistic, and we want to avoid that,” said Tony.

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