CAMPBELLFORD – A Campbellford family knows all too well the effects of MPS VI (Maroteaux Lamy Syndrome), a rare enzyme deficiency.
Seven-year-old Isaac McFadyen -the son of Andrew and Ellen and brother of Gabriel -is one of eight confirmed cases in Canada (and 1,100 worldwide) who have the progressive disease. Symptoms include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span.
The McFadyens also know how much treatment has helped Isaac — a treatment he gets only because of provincial funding. Funding they had to lobby to get.
About five years after Isaac’s diagnosis, a Palmerston, Ontario family is going through a similar experience. Darren and Pam More’s two-year-old son, Jasper, was diagnosed with MPS VI six weeks ago. They learned Monday that Jasper was denied funding to receive the treatment.
“It’s shocking in our province right now that this is taking place. We do have precedent set with Isaac and it’s virtually an identical case,” Andrew McFadyen said on Tuesday. “We know through Isaac’s precedent that they are… paying for it at the government level and it’s been very obvious how important that treatment has been to Isaac. For them to be putting the (More) family off right now and telling them no he’s not getting funded… to me it’s a travesty and it’s not representing equal access to health care for everybody.”
However, Health Minister Deb Matthews noted in an interview with QMI Agency on Wednesday the laws in Ontario changed in 2006 to prevent politicians from choosing which drugs do or don’t get covered. Now, the decision rests with an executive officer who relies on a panel of experts that weights medical evidence.
“However, I do want to learn more about this and I need to understand the basis of the decision,” Matthews said.
The McFadyens established The Isaac Foundation in 2006 to help raise awareness and also raise money to help fund innovative research projects (more than $240,000 has been given out) which aim to find a cure for MPS VI.
There is no known cure yet for MPS VI, but there is the life-sustaining treatment available called Naglazyme, which is an Enzyme Replacement Therapy designed to provide patients with a synthetic version of the enzyme they are lacking. Small doses are infused into the patient’s bloodstream.
“Studies are showing right now that kids that get on this treatment at a young age, basically where Jasper is, it gives them a better opportunity to prevent some of the side effects that come with the disease,” McFadyen said. “You can slow the progression of the disease, but it’s very, very difficult to reverse any of the symptoms that you’ve already seen, so in Isaac’s case he did start early but there was still a lot of disease in him beforehand and he had to have the spinal cord decompression surgery.
“Children diagnosed before age of three are thought to have a severe case and need to be treated immediately in order to prevent a lot of difficult things disease can inhibit in children,” he said.
Naglazyme is only available to Canadian patients through the federal government’s Special Access Program. The only provinces funding the treatment are Ontario, British Columbia and Quebec.
Isaac McFadyen has been receiving the treatment once a week for five years at the Hospital for Sick Children in Toronto. The cost of the treatment, produced by Biomarin, can range from $300,000 for a small individual to $1 million for a young adult each year.
It took lobbying to get Isaac’s treatment funded. The Isaac Foundation also works nationally and internationally to advocate for and support families dealing with MPS VI.
That’s why the McFadyens want to help the Mores. They’ve become accustomed to connecting with families internationally, but this is only the second time they’ve helped advocate for a family in Ontario.
It’s also “the first time that it’s someone as young as Isaac was when we first began this process,” McFadyen noted. “That’s why we created this charity and why we feel it’s important to have around. Not only to advocate, but we’ve been speaking with the family to kind of walk them through the stages of what to expect with this disease and what’s to come in the coming months. Also trying to give them hope that there is a treatment there and if we can get Jasper on it, that he has every opportunity of a wonderful quality of life and you know for him it will be a lifeboat until we can find a cure for this disease.”
Told of the More family’s circumstances, Ontario Premier Dalton McGuinty took a moment from his tour of the Port Hope Community Health Centre on Tuesday to say this spoke to him on a higher level than that of premier -rather, that of a father.
McGuinty said his heart goes out to the family in their difficulties, but he had no firsthand knowledge of the case and could not comment. He did suggest getting the Minister of Health, Deb Matthews, involved.
Matthews echoed the premier’s sentiments.
“I certainly certainly understand why this doesn’t sit right with Ontario families. I am following up to learn more about this drug and this particular situation,” she told QMI Agency on Wednesday. “I do know how important this is, and we want to do the right thing.”
Matthews said she will urge the ministry to examine the latest evidence on naglazyme.
“Maybe Lou knows something about it,” McGuinty added on Tuesday, referring to his host.
Northumberland-Quinte West MPP Lou Rinaldi nodded in the affirmative.
McFadyen confirmed he had updated Rinaldi on the events.
Rinaldi said yesterday that besides being aware of the case, all he knows is that the More’s local MPP John Wilkinson from the Perth-Wellington riding was working closely with the Ministry of Health.
This story about Jasper More gained some attention on Twitter during the past few days. McFadyen got a response from Matthews on Monday when he mentioned her Twitter handle, @Deb_Matthews in a tweet.
“I am looking into this. Thank you for bringing it to my attention,” she wrote.
McFadyen said Tuesday he was having no luck getting through to Matthews’s office, and found it difficult enough just to get someone to leave a message for her.
“I was told yesterday they’re looking into it and now I can’t get a hold of anybody today,” he said.
“What I do know is that there has been a very, very high call volume to Minister Matthews’s office today from people all across Ontario who are very concerned with this,” McFadyen adding, while also saying people told him they not only call the minister’s office, but contacted their local MPPs as well.
“There’s a lot of interest in this and it all seems to be very positive, understanding that this is a little boy that desperately needs to be on a treatment and it desperately needs to begin now,” McFadyen said.
After receiving tweets from McFadyen and others interested in the case, Matthews wrote to McFadyen again Tuesday night.
“We are all working hard on this case,” she said. “I know it is difficult, but know we are doing our best to move forward.”
An appeal to the rejection of funding was made on Tuesday.
What the More family is dealing with now, including lobbying for funding, takes the McFadyens back to when Isaac was diagnosed.
“It’s so, so hard to go through this process for a family and I know that they’re lost. You’re dealing with the news that your son has been diagnosed with this disease, and all the questions going on in your mind and adjusting to a new reality of life. It’s terribly overwhelming and I really feel for them right now,” McFadyen said.
“(Getting the funding) is something that we believe in, and if it were anybody else’s children, they would only expect the same. They would expect to have everything done in order to look after the well-being of their children. The fact that’s not being done right now is actually shocking and disappointing.”
jgard@northumberlandtoday.com
twitter.com/NT_jgard
– With files from Sheena Goodyear
