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McFadyens Fight For Children With MPS VI
The following is part two of an interview with Andrew McFadyen, the father of eight-year-old Isaac who has the rare disease MPS VI. Wednesday’s story featured Isaac’s continued battle with the disease. Today’s story focuses on the McFadyen family’s advocacy for other children diagnosed with the disease. Friday’s story will announce their lofty new fundraising project.
CAMPBELLFORD — First, back in 2006, it was for his own son Isaac. Then, last year, it was for another Ontario boy, two-year-old Jasper More. Now, it’s for a three-year-old Saskatchewan girl, Violet Revet.
All three young children (although Isaac is now eight) were diagnosed with the rare enzyme deficiency disease MPS VI (Maroteaux Lamy Syndrome). Two of them — Isaac and Jasper — were initially denied funding from their provincial government for the life-sustaining treatment that is available to help to help stave off the devastating symptoms of the disease. Violet, the most recent case, and her parents — Jennifer Yanke and Larry Revet — are still waiting to find out if she is approved or denied. She is only the ninth confirmed case of MPS VI in Canada. There are about 1,100 worldwide.
Andrew McFadyen has lobbied governments to ensure the children get the treatment they so desperately need.
Make no mistake, McFadyen pours his heart into every lobby effort, whether that’s for his own son or someone else’s child. He just wishes he didn’t have to. One child denied, one lobby effort is all one too many, as far as he’s concerned.
“This is now the third big advocacy push that we’ve had to look after and that’s a big problem to me,” he said in an interview earlier this week. “You would think after the first one that precedent has been set and people can go forward and change public policy for the better and ensure there’s an active review process for this type of treatment.”
When Isaac McFadyen was diagnosed with MPS VI, the news was devastating to his parents Andrew and Ellen. Symptoms of MPS VI include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span.
Somehow, they had to find the strength to fight for funding from the government. Treatment, called Naglazyme, provided hope. It’s an Enzyme Replacement Therapy designed to provide patients with a synthetic version of the enzyme they are lacking.
Naglazyme is only available to Canadian patients through the federal government’s Special Access Program. The treatment comes with a hefty price tag of a million dollars a year, to be paid by provincial governments. From the McFadyens perspective as parents, though, you can’t put a price tag on your children.
“We know that this (treatment) is not a cure. I never tell anybody Isaac needs this, or Jasper needs this, or Violet needs this because it’s going to cure the disease,” McFadyen said. “We’ve always said it’s a lifeboat. It’s something that we can provide our children with to slow the progression of the disease and in some cases halt the progression of the disease until we can find that cure. It allows children to have a more normal and healthy life.
“From a parent’s perspective, that’s what you want for your children, to give them the best opportunity at a normal, healthy life,” he continued. “From a government’s perspective, I know they look at this for dollars and cents. They don’t look at the cost of the disease for them if it goes untreated. They don’t look at the cost of corneal transplants, double hip replacements, heart and airway disease…they don’t take that into account. They just look at that cost of one million a year and balk at it. It makes me angry, it makes me upset, and as a parent it really makes me question the importance of children with rare diseases across this country.”
Back when the McFadyens were lobbying for funding, they were strongly considering a move to England, where the treatment is approved. The country was willing to take the family and pay for Isaac’s treatments.
“It’s a priority there, because they have an Orphan Drug Plan, they have policies in place to ensure this is dealt with at an early stage and the best interest of the child is looked at,” McFadyen said. “Kids diagnosed with the disease start treatment within weeks.”
Funding for Isaac’s treatment was eventually approved by the Ontario government. The McFadyens received help from former Conservative MPP Elizabeth Witmer, who was the opposition’s health critic at the time. She invited the Campbellford family twice to the Ontario Legislature. In July 2006, the family received word from then-minister of health George Smitherman that the provincial government would fund Isaac’s ERT treatments.
There couldn’t have been better news at that time. Surely it inspired the McFadyens to help others. That same year they established The Isaac Foundation to help raise awareness of the MPS VI and also money to help fund innovative research projects.
Having The Isaac Foundation has also provided a connection to other families who have had their children diagnosed with MPS VI.
In August of 2011, Darren and Pam More of Palmerston, reached out to the McFadyens when their son Jasper was diagnosed with the disease. Six weeks after the diagnosis, the Mores learned that Jasper had been denied funding to receive the treatment.
That was a shock to McFadyen, that a second child had been denied in this province.
“We were really surprised and obviously really angry,” he said.
With the story spreading through news coverage and on Twitter, led by McFadyen’s efforts, within days the More family received word that the decision had been overturned and Jasper would get funding for the treatment after all.
“When we finally got approval and knew that (Jasper) was going to be covered, that should have been in it and the rest of the provinces should have been on board,” McFadyen said.
British Columbia and Quebec were already on board following Isaac’s case when children in those provinces were diagnosed.
Saskatchewan, which has the newest case, was a different story when Violet was diagnosed.
“To have another province go through this exact same thing, for us it’s astounding that we can still be in this position — that we can’t look after our children that are suffering from these rare diseases,” McFadyen said. “It really goes to show that a lack of an Orphan Drug Plan here in Canada does impact our kids in a negative way.”
McFadyen is also concerned that it took 18 months for Violet to receive the proper diagnosis that she was suffering from MPS VI. Worse, he says, is that Violet has since been waiting an additional seven months waiting for the approval of treatment.
“The timeline between symptoms appearing, diagnosis and then beginning treatment is really detrimental to Violet and her health and it’s actually embarrassing for our health care system to have those statistics out there because we aren’t looking after the needs of these kids suffering and I really feel for the family and I really feel for Violet,” McFadyen said.
McFadyen noted the Saskatchewan government says they are looking into the safety of the treatment. He said that shouldn’t be a concern since it’s approved in the United States and England and is already being used in three other Canadian provinces through the Special Access Program.
“It’s just not approved here in Canada because of the limited number of children that are affected by this disease,” he said. “What they’re really doing is looking at the cost on a year-by-year basis. I understand that it’s not easy to commit a million dollars in funding for one child per year for a government, but it seems to be that we have a great health care system until you get really sick or in need of something that’s really expensive. Then there’s questions on whether they’re going to treat you or help you.”
Due to how rare these cases of MPS VI are, providing funding, McFadyen suggests, isn’t going to bankrupt a government.
In addition to the advocacy work McFadyen has been doing behind the scenes, he said a social media campaign evolved for this case. The Twitter and Facebook pages for Saskatchewan Premier Brad Wall were flooded with public comments calling to approve the funding for Violet.
At this point, McFadyen has the experience of knowing what governments need to ensure these files move quickly. He can also provide any safety data they require and the benchmark information they need.
“My issue was the timeline involved here,” he said. “It doesn’t seem to be the highest priority. When I take to Twitter to express that fact or when I put my press release out, sometimes it’s surprising how many people pick that up and are interested and engaged and start that snowball effect of tweeting the premier and hitting his Facebook page.
“We’re getting responses from the premier and responses from the people involved and they are signaling that they’re listening to us and they hear us and work is moving forward in a timely fashion. Now it’s my role to ensure that timely fashion is within the timelines that Violet needs and that we as Canadians expect the people making our decisions on our behalf.”
McFadyen said a decision could be announced any time now.
While Isaac has been on the treatment for several years now and benefitted from it, even little Jasper More has made great strides a year later.
“Jasper is doing so well,” McFadyen said. “He’s had no ill effects from the treatment that he’s receiving, the disease seems to have slowed or even halted in him, and that’s really positive because we know if children get on this treatment really, really early, the outlook for them is spectacular.
“There’s all the hope in the world that he won’t have to suffer any of the symptoms that this disease can lead to. The family is doing okay. I think they’ve come around to their new circumstances in life and are enjoying every moment that they have together. We see them often. They come to all of our Isaac Foundation events, and they’ve joined our board and the charity and they’re looking after a lot of the fundraising with us and they’re really engaged to find a cure. To see Jasper running around with the big smile he has on his face is really rewarding.”
Now, he wishes the same for Violet.
“Ideally, we win this one last battle and public policy can be set for the rest of the country and maybe it will spark Canada to come up with an Orphan Drug Plan so this never has to happen again for any child affected with any type of rare disease,” McFadyen said.
UPDATE: Violet’s family has received word that the funding has been approved and she will get the treatment she needs.
jeff.gard@sunmedia.ca
twitter.com/NT_jgard
Drug Approved For Saskatchewan Girl With Rare Disease
Naglazyme will extend the little girl’s life, was not previously available in the provinceReported by News Talk Radio staff
First Posted: Aug 8, 2012 7:25pm
Violet Revet, a three-year-old girl from Langenburg, is one of only nine people in Canada who has Mucopolysaccharidosis VI (MPS-VI) and now she will receive treatment she desperately needs.
MSP-VI has no cure and the longer she goes without treatment, the shorter she could live.
Andrew McFayden’s son, Isaac, was diagnosed with the disease in 2005.
“It’s a very devastating condition. Essentially kids that are diagnosed with MPS lack an enzyme in their blood that breaks down all the complex sugars. Because those complex sugars that occur naturally in the body don’t break down they need to find a place to go and so they end up being stored in the bones, tissues, organs, and muscles and it leads to many progressive symptoms: heart and airway disease, progressive stiffening of the joints, corneal clouding, spinal cord compression.”
“Essentially every muscle, tissue, and organ in the body is affected by this disease with the exception of the brain.”
McFayden was told Isaac would only have seven or eight years to live. That was before doctors realized a treatment was making the rounds in the UK and the US, a synthetic enzyme known as naglazyme that acts as a replacement in the body.
Isaac could get access to it under a special access program but the drug wasn’t covered in Ontario. It took McFayden eight months of lobbying before the provincial government agreed to pay for it.
Violet’s family asked Saskatchewan’s health ministry for funding so she could get the same treatments.
Today, the government drug review process has approved naglazyme and her doctor will begin administering the treatment.
With files from News Talk Radio’s Patrick Book
Expensive Medicine Approved For Saskatchewan Girl
(Note:CBC does not endorse and is not responsible for the content of external links.)
A youngster with a rare medical condition has been approved for coverage of a costly drug, the only known therapy for MPS VI, also known as Maroteaux-Lamy Syndrome.
Patients with the condition lack a key enzyme in their systems.
A therapy is available but costs around $250,000 to $300,000 per year for a patient.
The family of 3-year-old Violet Revet recently appealed to provincial politicians to help them through the approval process.
A decision, which was announced Wednesday, means the treatment involving infusions of a replacement enzyme can begin.
According to officials, only nine people in Canada have the disease.
Revet’s situation caught the attention of a national foundation which mounted an awareness campaign, using social media such as Facebook and Twitter, to encourage providing coverage of the treatment.
On Wednesday, the Isaac Foundation said news of the approval was welcomed by the girl’s family.
“Its a relief to finally get the ball rolling on her treatments after waiting seven long months,” Larry Revet, Violet’s father, said in a news release from the foundation. “We can now move ahead with the next step to ensure Violet has a stronger, healthier life ahead of her. This is great news.”
“My daughter has been given a chance for a bright future,” Jennifer Revet, the youngster’s mother, added. “For this I am truly thankful.”
The foundation’s director, Andrew McFadyen, noted the approval process highlights the need for Canada to improve policies related to what are known as orphan drugs, or medications for extremely rare conditions.
“Families dealing with rare disease shouldn’t have to have put their children on the front page of newspapers to receive the treatment they deserve,” he said.
Violet Revet, Saskatchewan Girl With Rare Disease, Will Have Her Treatment Paid For By Province
CP | By The Canadian Press Posted: 08/08/2012 9:40 pm Updated: 08/08/2012 10:23 pm
Violet Revet of Langenburg is one of only nine people in Canada who has Mucopolysaccharidosis VI. ALAMY
REGINA – The Saskatchewan government has agreed to pay for treatment for a three-year-old girl with a rare disease.
Violet Revet of Langenburg is one of only nine people in Canada who has Mucopolysaccharidosis VI.
The disease has no cure and the longer she goes without treatment, the shorter her life span will be.
Andrew McFayden’s son, Isaac, was diagnosed with the disease in 2005.
McFayden, of Campbellford, Ont., was told Isaac would only have seven or eight years to live.
That was before doctors realized a treatment was making the rounds in the United Kingdom and the United States — a synthetic enzyme known as naglazyme that acts as a replacement in the body.
Isaac could get access to it under a special access program but the drug wasn’t covered in Ontario.
It took McFayden eight months of lobbying before the provincial government agreed to pay for it at a cost of about half a million dollars a year.
“It’s a very devastating condition,” said McFadyen, whose eight-year-old son goes to Toronto for regular treatments.
“Essentially, kids that are diagnosed with (it) lack an enzyme in their blood that breaks down all the complex sugars. Because those complex sugars that occur naturally in the body don’t break down they need to find a place to go and so they end up being stored in the bones, tissues, organs, and muscles and it leads to many progressive symptoms: heart and airway disease, progressive stiffening of the joints, corneal clouding, spinal cord compression.”
McFadyen has said that while the treatment is not a cure, it will keep the children alive while efforts to find a cure are made.
Violet’s case had prompted many supporters to flood Premier Brad Wall’s Twitter account with pleas for the funding, and Wall asked the ministry to look into the matter.
On Wednesday the approval came through.
(CJME)
Every Moment is a Gift for the McFadyen Family
The following is part one of an interview with Andrew McFadyen, the father of eight-year-old Isaac who has the rare disease MPS VI. Part two will feature the McFadyen family’s advocacy for other children diagnosed with the disease. The third part will announce their lofty new fundraising project.
CAMPBELLFORD – A year ago this time, Andrew McFadyen would have said his oldest son, Isaac, was the healthiest he had ever been.
Even in October, the McFadyen family from Campbellford was in part celebrating that success at The Isaac Foundation’s second annual Gala For A Cure in Cobourg.
“Three weeks after that we got word that his heart wasn’t doing all that well,” Andrew McFadyen said during an interview earlier this week. “Then in early March, he had some sort of brain event that we don’t really know what it was. There were indications that it could have been a stroke. So we’ve been spending a lot more time than normal at the hospital. We spend a lot of time there anyway but we were doubling and tripling up on our trips and having a lot of emergency scans and emergency tests.”
Isaac McFadyen, who is now eight years old, has lived with MPS VI (Maroteaux Lamy Syndrome) since 2005. He is one of only nine confirmed cases in Canada — and about 1,100 worldwide — of the disease which is caused by a rare enzyme deficiency. Andrew McFadyen and his wife, Ellen, established The Isaac Foundation in 2006 to help raise awareness of the disease and money to help fund innovative research projects.
Having watched his oldest child live with the progressive disease since 2005, McFadyen said every day is a blessing for himself and Ellen, who also have a younger son, Gabriel.
“When we were told he might not live past the age of seven, every moment with him is a gift and we recognize that and take it all in and it’s really special for us,” he said.
This story about the McFadyen family isn’t new, but rather more of a reintroduction.
For those who don’t know, symptoms of MPS VI include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span.
“Children who have this condition lack an enzyme in their blood that breaks down all of the complex sugars,” McFadyen explained. “We have the complex sugars that build up naturally in our body, it has nothing to do with the foods that we’re eating or the diet that we’re on. They just build up naturally in our body and are broken down by all the enzymes in our blood. Children that suffer from MPS lack one of the enzymes responsible for breaking down those complex sugars, so instead of breaking down, they find a place to go and store. They store up in the bones, the tissues, the organs, the muscles…very progressively throughout the course of the affected individual’s lifespan.”
As devastating as the news of the diagnosis was all of those years ago, there was still a rollercoaster of emotions to follow.
First, there was hope when they found out a treatment was available to slow the progression of the symptoms, but at a price tag of a million dollars a year that the Ontario government would have to agree to pay for.
“Parents have to endure the devastating news that their child has been diagnosed with this condition. I was sick for weeks and lost 40 pounds. It’s not news I would wish on any parent ever,” McFadyen said. “We were told at the time that Isaac wouldn’t make it past his early teens and that even by seven years old he might be gone from us. That was really, really hard for both my wife and I to take in and handle.
“Then this idea that we found there’s a treatment that can stave off the disease, slow it down…that’s incredibly hopeful and you want to do everything in your power to ensure your child can get this treatment. But then there’s this hook that yes you can bring it to Canada so long as the Ontario government says they’ll pick up the price tag of a million dollars.”
While not a cure for MPS VI, the life-sustaining treatment available is 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.
Naglazyme is only available to Canadian patients through the federal government’s Special Access Program.
Initially, the McFadyens request to the provincial government was denied.
“Being told no from your government, no we’re not going to pay to save your child’s life is devastating, it’s heartbreaking, it’s worrisome…it makes you question the priorities of the people in charge.
“That was really hard, in one sense trying to cope and trying to just come to terms with your new circumstance in life and then to turn around and not have your government look after you was disappointing and discouraging for us.”
They didn’t give up, though, and continued to lobby the government. With the help of former Conservative MPP Elizabeth Witmer, who was the opposition’s health critic at the time, they were ultimately successful (more on the initial rejection and subsequent approval in part two of this interview tomorrow).
Isaac heads to Sick Kids in Toronto a weekly basis and the treatment has altered his life.
“Had he not been on this treatment, he would be suffering from a lot of very, very devastating symptoms,” McFadyen said.
Despite the setbacks during the past year, McFadyen said he believes, and hopes, that Isaac’s condition is stable again.
“We always have hope that’s the case, but we know that we have to work really hard and find a cure and other treatments because as we’ve known from the start this treatment isn’t a cure,” he remarked. “You can only stave off the disease, or slow it down for so long. There’s always going to be this disease building up in his body and so I guess this past year has really just re-motivated us and re-energized us to ensure that we can find a cure for him and everyone who has this disease.”
McFadyen said there’s a “tricky balance” of developing a sense of normalcy when things are going well.
“It just becomes an everyday part of your life — our weekly trips to Toronto, his receiving this very expensive medication that seems to be doing really well — and then you’re blind-sided with what you think is a routine heart appointment. You’re blind-sided when his schools calls and says he’s suffering this incredible headache and can’t see out of his eye,” he said. “That blind-sided news takes you right back to the start again. That’s what we’ve been going through this past year. We keep telling ourselves we’ll never let our guard down again, but sometimes it’s okay that we do because that means things are going really well and he’s doing okay.”
Isaac’s parents want him to live as normal a life as he possibly can.
“We don’t want Isaac ever to just be identified as the sick little boy,” McFadyen said. “We want Isaac to be identified for who he is and what he does and the fact that he just happens to have this rare disease.”
jeff.gard@sunmedia.ca
twitter.com/NT_jgard