Read more - Irish child with killer brain disease OK'd for New York gene therapy
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Heffernan from Co. Kerry has had her invitation to participate in a medical trial at Cornell University Hospital withdrawn after failing to meet surgical criteria -- but if the family can raise an additional $250,000 in the next few weeks she is guaranteed a place on a parallel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.
“We don’t want to lose our child to Batten Disease. It’s simply not an option.”
He added, “The $750,000 is only for the actual trial. We will need more if Saoirse has to spend extra nights in the hospital and things like that.”
Additional funding for travel and accommodation expenses are also needed.
The trial is scheduled to last for 18 months, all of which time Saoirse and her family will be based in New York.
Every penny of the money must be paid upfront before Saoirse can be guaranteed a place on the trial.
“It’s like this, as soon as we get all the money together, the sooner we can get back here and get Saoirse on her way to treatment,” said Tony.
“We will spend the next few weeks raising the money in some way or another to get Saoirse back here to New York.”
“I’ll go door to door if I have to for the next few weeks -- anything to save my daughter’s life,” he said.
Saoirse is currently the only child well enough to qualify for the parallel trial, and does not need to undergo anymore qualifying tests for participation.
“She is in once we can raise the money, and that needs to be done sooner rather than later,” he said.
Saoirse’s younger brother, Liam, two, will also be accepted for a trial beginning next summer.
Liam was also diagnosed with Batten Disease earlier this year, and although in great spirits at the moment he is deteriorating at a much more rapid rate than Saoirse did at his age.
Since Saoirse’s diagnosis last year, the Heffernans have spent every waking moment raising funds to pay for potential trials, and so far they have successfully secured $500,000, including the $120,000 raised by the Irish community in New York a few weeks ago.
The Heffernan’s founded the all-Ireland Batten Disease charity, Bee for Battens, in March to support sufferers, fund research and participation in medical treatment trials. The family is due back in Ireland on Wednesday, where they are scheduled to go straight to Temple Street Hospital.
“As soon as we get off the plane Saoirse will be admitted to Temple Street for a full head to toe checkup, and hopefully adjustments to her medication to help her through till November/December,” explained Tony.
There are less than a handful of sufferers in Ireland, with only 750-1,000 sufferers worldwide.
To date Late Infantile Batten Disease has always been fatal, and these trials are taking place to attempt to devise a cure. The Cornell trials have been a success so far.
Tony told the Irish Voice about twins from England who were both Batten sufferers. The little girl made the trial (the parents could only afford to send one child). She is still alive. Her twin brother died.
The charity’s website is www.beeforbattens.org.
Checks may also be sent to the Saoirse Foundation, the Kerry Hall, 305 McLean Avenue, Yonkers, New York, 10705.
Contact April Drew at april@irishvoice.com or 212-684-3366, ext. 116 with fundraising ideas.
Read more - Irish child with killer brain disease OK'd for New York gene therapy
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Switch to the desktop site to post a comment.illhelp | Dec 09, 2010, 02:29 PM EST
She deserves this and her brother an awful lot. PLEEEEEEEEEEEEAAAAAAAAAAASSSSSSSSE HELP...
illhelp | Dec 09, 2010, 02:27 PM EST
Its unfair shes too young. Money doesnt matter people should get together and raise money. A childs life comes first. People raise money fast and stop being selfish. This child will die fast without your,ours help. Please agree. xxx
ShamrockMiss | Oct 15, 2010, 10:07 AM EDT
Dublinjas - I agree with you, but I would slightly reword it and say that the US is a world leader in medical research and the development of cutting-edge therapies. There is no question about that. However, the US does not lead the world in terms of delivering high quality care to the average American. Just consider that the hospital bed alone at Cornell's hospital probably costs about $1,600 per day. At least, that's what the hospital will bill Saoirse's parents for her stay. That's $48,000 for one month of inpatient care. People without health insurance who don't qualify for medical assistance through the state (you have to be pretty poor to qualify) and can't pay the bill on their own simply won't get admitted to a hospital. They won't get the procedure they need, so will stay at home and cope on their own, or perhaps try to manage their illness with a cheaper medical option. Sometimes they simply die. That's just the reality of the situation here. I mean, if Cornell really wanted to do the procedure, they could donate the room for her, and look how much closer her parent's would be to having enough money. There are ways to make this kind of thing happen if people just can muster up the will to do it.
Dublinjas | Oct 15, 2010, 05:52 AM EDT
I don't think the US. should be considered 'Just a Nation' when it comes to things Medical, I think we can all agree that it is a World leader in the field of medicine and as such it has rightly become a Mecca for those who seek treatments that are out of the box as well of course as hi tech medicine at the cutting edge, But it beggars belief how the fee of $750,000 was calculated and if this stands then I guess there comes a point where the treatment is just not accessible to this family sad to say. otherwise where do you draw the line. I hope I am wrong and I hope these children get into the program but it does not look promising.
ShamrockMiss | Oct 14, 2010, 06:37 PM EDT
The medical industry is just that - an industry. Cornell could probably find a way to do this procedure for $500,000 if they really wanted to. Someone who knows should look at how that $750,000 total is calculated. I bet there's a lot of profit and some pretty high salaries in there.
Buffalobrave | Oct 14, 2010, 09:40 AM EDT
So much for Capitalism. One's life and health should never depend on how much money you can spend. International and national health care should be a basic human right and NOT determined by the size of your bank account. The world is a disgrace!
DennisQ | Oct 14, 2010, 07:39 AM EDT
The Batten's Disease Research and Support Association (BDSRA.org) has an easy to understand online brochure about the disease. BDRSA's video, called "What is Batten's Diseases?" is availabe on YouTube.
Animals with disorders similar to Batten's have improved with therapies originating from stem cell research. The ban on human stem cell research was liften in early 2009 when Obama took office. Human embryos are a product of in vitro fertilization procedures, and are routinely discarded. These stem cells might have been used in the search for a cure for Batten's, but the delay is now behind us.
The Obama administration's economic stimulus program is now funding research into Batten's, raising the possiblility that families in the future won't go through the same ordeal as the Heffernans.
LiamDavid | Oct 14, 2010, 04:12 AM EDT
May all of our prayers be with Saoirse and her brother Liam, as well as their parents. I too, am upset at the staggering cost and seemingly insurmountable barriers that lay between these children and their badly needed treatments for what currently is an ultimately fatal disease. Also, I wanted to comment on the remarks made here by “baileyBD”, wherein the writer states that “Obamacare is about to take hold and ruin it” when referring to the current state of American Health Care! If the writer and all the rest of those who delight in bashing President Obama with uninformed comments about “socialized medicine, and how Insurance companies are suffering or doctors rethinking their career choices and companies dropping new retirees from medical packages”. This is nothing more than fear mongering and throwing around lies and misstated facts so as to scare and upset their fellow Americans due to the coming elections in November. The truth of the matter is that “baileyBD” and the Tea Partyers along with Republicans (which is really redundant), either have not read the law or they don’t understand it? Insurance companies are still racking in the big bucks, and are far from closing anything! This “Obamacare” as baileyBD referred to, will now allow millions more of Americans to have health care! Tea Party members also blame the President with trying to raise taxes. They try and make it sound as if all Americans will get a tax increase, but the truth is it’s a tax for the wealthiest 3% of Americans who earn more than $250,000 per year. So, how about we all step away from the politics and return to our concerns for these children! The only money we should be talking about is donations for Saoirse and Liam. Slan
Temerity | Oct 13, 2010, 06:51 PM EDT
Mmnnnn. very interesting.Very sad.
ShamrockMiss | Oct 13, 2010, 02:06 PM EDT
baileyBD: Unfortunately, we do not have the best medical care in the world. Considering the entire service system and patients in all income levels, the US ranks #37 on the WHO list of countries in terms of quality of care. When one only considers the care available to the wealthiest Americans, then the US would be #1, but just for the population groups who can afford to pay out of pocket. Just because the best healthcare "exists" in the US doesn't mean people can actually receive it. That takes the kind of money most people just don't have. BTW - which country ranks #1? France.
Searlit | Oct 13, 2010, 02:03 PM EDT
baileyBD, see the last page of this article for where to send donations. You sound like a medical professional, maybe you can help.
baileyBD | Oct 13, 2010, 01:19 PM EDT
I am an American and can verify that we DO indeed have the best medical care in the world. Free market practices have created this fact. You do NOT need to be very wealthy or very poor to receive care. At least for the time being, as Obamacare is about to take hold and ruin it. Just the threat/promise of socialized medicine has many insurance companies already closing up shop, doctors rethinking their career choices and companies dropping new retirees from their medical benefit package. That being said, please remember that Saoirse was part of a study or experimental program, not seeking "normal" care. Plus, the majority of us here did not even know about Saoirse. How can one contribute to the fund to make sure she is able to participate in the less stressful/physically demanding treatment that she does qualify for?
sharon domon | Oct 13, 2010, 01:01 PM EDT
I think that the United States lets other children come to the states for treatment free of cost. Why would they deny her the treatment she needs? Its appalling and cruel. She is stuck in the middle of not being rich enough and not being poor enough. Its insane..I will pray that clearer heads will prevail and she will soon be here to receive her treatment.
Searlit | Oct 13, 2010, 11:57 AM EDT
That's why people say America has the best medical care in the world - except one can't access it unless you're very wealthy or very poor. I'm sorry to hear that Saoirse must wait again.
kilgara | Oct 13, 2010, 11:25 AM EDT
If people knew the amount of wasted money in medical research it would be a bigger scandal than Watergate.
spitfire | Oct 13, 2010, 10:49 AM EDT
i personally think it is a disgrace they will take children from third world countries without blinking an eye but they will not take this child -- but only for the extra monies it will cost which I think is so so wrong