Andrew McFadyen
Sarah Harmer To Perform For Charity Gala In Cobourg
Sarah Harmer/Isaac Foundation Gala
Submitted photo
Formed in 2006 by Andrew McFayden and Ellen Buck-McFayden after their son, Isaac, was diagnosed with mucopolysaccharidosis VI (MPS VI), the foundation funds innovative research projects that aim to find a cure for children’s MPS VI.
MPS VI is caused by the lack of an enzyme in the blood that breaks down complex sugars which then build up in the tissue, organs, and bones and leads to, among other things, hydrocephalus, heart and airway disease, spinal cord compression, stiffening joints and stunted growth.
Isaac was diagnosed with the disease when he was 18 months old. At the time, he was only the third child in Canada diagnosed with MPS VI and there was no approved treatment. Luckily, approval came shortly after Isaac’s diagnosis and he began treatment that he continues to this day.
“It’s not a cure but it’s his life-boat until a cure is found,” said Mr. McFayden. “And Isaac has that future back that he never had without treatment.”
Since the foundation formed, it has raised $240,000 for research and by the end of the year Mr. McFayden plans to make it $300,000. With most of their major fundraising events happening outside their home-base area, Mr. McFayden said they had always wanted to have a gala and thought it was the perfect event to have close to home.
“This is the second year we’re having the gala,” he said. “Last year was a great success, Ron Sexsmith performed and we were able to raise $20,000. This year we’re hoping to raise $40,000.”
A great deal of international support and recognition has come from big names such as musician John Mayer and Major League Baseball star Roy Halladay, who both have become close friends of Isaac and the foundation.
John Mayer, who Mr. McFayden said often lends a hand to raise awareness of the organization during his concerts and on his website, will be the recipient of this year’s Foundation ‘Lifetime Impact Award’, an award presented to a person who has made a positive impact on and increased Isaac’s quality of life as he fights MPS VI.
Last year’s recipient was Kitchener-Waterloo MPP Elizabeth Witmer, who tirelessly advocated to secure funding from the Ontario government to fund the life-sustaining treatment that Isaac receives at The Hospital For Sick Children on a weekly basis, said Mr. McFayden.
“We’re always so humbled with people and their desire to help,” said Mr. McFayden. “People seem to read about Isaac’s story and they want to help, we’re really very fortunate and couldn’t ask for more.”
The Isaac Foundation’s 2nd Annual ‘Gala For A Cure’ will be held Saturday, Oct. 1, at the Best Western Ballroom in Cobourg. Tickets for the event, which include a wine tasting by Huff Estates Winery, hors d’oeuvres, dinner, Ms. Harmer’s performance, a silent auction, and dancing, are on sale now and can be purchased by contacting Ellen Buck-McFadyen at 705-395-8854 or ellen@theisaacfoundation.com.
For more information about The Isaac Foundation, Isaac McFadyen, or MPS VI, visithttps://www.theisaacfoundation.com.
Isaac Foundation To Hold Second Gala For A Cure
NORTHUMBERLAND – Government funding for live-sustaining treatment for MPS VI (Maroteaux Lamy Syndrome) is already extremely important. The only thing better would be finding a cure for the rare enzyme deficiency disease.
That was only reinforced to the McFadyen family of Campbellford last month when they helped another Ontario family lobby the provincial government for the same funding their own son receives to help battle MPS VI.
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 established The Isaac Foundation in 2006.
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.
Isaac 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. The McFadyens lobbied the provincial government to get funding for the treatment and were ultimately successful.
In August, they helped a Palmerston family do the same. Darren and Pam More’s two-year-old son, Jasper, was diagnosed with MPS VI six weeks ago. Within days of being notified they were denied funding for the treatment, the provincial government reversed its decision.
Both families are grateful for the funding as they aim to find a cure.
The Mores will be attending the second annual Gala For A Cure, presented by The Isaac Foundation, Saturday, Oct. 1 at the Best Western in Cobourg.
Tickets are $100 (receive a $50 tax receipt) and will feature a special intimate musical performance by Juno Award winner Sarah Harmer, as well as wine tasting, dinner and a silent auction.
“Knowing that we desperately need to find a cure for these children makes these events that much more important and that much more special for everyone,” Andrew McFadyen said. “Knowing that every dollar goes directly into research is very important as well.
It’s also important to raise awareness of the disease.
“The last five years of us raising awareness was very crucial in helping Jasper get treatment,” McFadyen said.
Last year’s inaugural gala raised $20,000 and went towards research on behalf of The Isaac Foundation. The success was from those who bought tickets to the event as well as donated items to the silent auction.
“For me it was the community feeling that was there,” McFadyen said. “This sense of caring and wanting to help out. With it being such an intimate evening, I felt it was really special for everyone.”
Singer/songwriter Ron Sexsmith helped make last year’s gala a special evening, McFadyen noted, and he expects Harmer will help create a similar atmosphere. The McFadyens are thrilled to have her be a part of the event. They are friends with The Tragically Hip, who are friends with Harmer, and they also received help from Sexsmith’s wife in bring Harmer to this year’s gala.
“There’s a lot of connections that Sarah and we already had, we just needed to connect the dots in order to make it work,” McFadyen said. “When she looked at the website and learned about The Isaac Foundation, she was more than happy to come and pitch in and be a part of this. We were really touched when we got the call saying yes she’s going to be there and perform, and she’s going to spend the whole night with us.”
Anyone wishing to purchase tickets, or donate items for the silent auction, can contact Andrew by phone at 613-328- 9136 and e-mail at mcfadyena@me.com or Ellen at 705-632-0983 and ellen@theisaacfoundation.com.
Tickets are limited.
jgard@northumberlandtoday.com
Province To Fund Palmerston Boy’s Expensive Drug Treatment
By Vik Kirsch, Mercury staff, www.guelphmercury.com, Updated: August 26, 2011 6:30 AMJasper More will begin receiving life-saving but extremely expensive drug treatment within weeks for a rare genetic disorder.
PALMERSTON — Jasper More will begin receiving life-saving but extremely expensive drug treatment within weeks for a rare genetic disorder.
The Ontario Ministry of Health will fund the enzyme replacement therapy, which can cost up to $800,000 a year, for the 2 ½-year-old Palmerston boy who has Maroteaux-Lamy Syndrome (MPS Type VI). He was diagnosed in the spring.
He is the second Ontario child to receive treatment for the rare disease, which afflicts eight Canadians and roughly 1,100 people globally.
His parents, Darren and Pam More, feel enormous relief at news that the drug regimen is to begin as early as September.
“We’re a lot better than we were in April let me tell you,” Darren More said Wednesday. “In April, when we found out, we were just completely wrecked.”
MPS Type VI is the rarest in a family of diseases that fall under the umbrella of mucopolysaccharidosis disease, a metabolic disorder in which enzymes the body needs to break down sugar carbohydrates are missing or malfunctioning, compromising a child’s development as waste material builds up in the body.
There’s no known cure and the prognosis is a shortened lifespan. But with groundbreaking enzyme replacement therapy, patients can live nearly normal lives, advocates say.
Health Minister Deb Matthews said Wednesday the government is covering the cost after an evaluation by the executive officer of the provincial drug program, who looked at a variety of factors such as the therapy itself and assessments by other jurisdictions, and a rare diseases review committee.
Absolute conclusions, Matthews said, are difficult in cases of ultra-rare illnesses such as Jasper’s form of MPS, but she was happy with the funding decision.
“I’m very pleased,” she said. “It’s the right thing to do.”
The Mores have been helped in learning about MPS and appeals to the government by the Canadian MPS Society and the Isaac Foundation, a Campbellford, Ont. research funder founded by the parents of Isaac McFadyen.
Andrew McFadyen said his seven-year-old son, who has received government-supported therapy since 2006, is doing extremely well.
“(He’s) probably the healthiest he’s ever been. Essentially, we’ve stabilized the disease.”
McFadyen termed the treatment a godsend. “It saved his life, for sure.”
The foundation is funding research into treatments with an ultimate goal of a cure. It’s about to release findings on a research program at a New York hospital on MPS Type VI.
“That’s what Jasper and Isaac both have,” McFadyen said, stressing any advances made will have positive implications for all versions of MPS.
Jasper’s April diagnosis was confirmed through further testing in July, said his father, a purchasing manager for an Arthur-area firm. The couple has two other children, Daphnie, 9, and Clayton, 5, who are free of the disease.
More said when both parents are carriers, as they are, a child has a one-in-four chance of getting the genetic disease. “We had absolutely no idea we were carriers at all,” he recalled.
Jasper was born with two hernias and later developed a growth on his spine. Today, the boy has some enlarged organs and bone deformities, but the condition will improve with therapy. “Type VI is treatable. (It’s) not curable, but treatable.”
The enzyme replacement therapy offered by a California firm is tailor made, so it can’t be mass produced. “It has to be specific to the patient,” More said.
Jasper will have to travel each week to a London, Ont. hospital for an intravenous drip.
The treatments will last “as long as he lives, or until they find a cure,” More said.
Province To Fund Palmerston Boy's Expensive Drug Treatment
By Vik Kirsch, Mercury staff, www.guelphmercury.com, Updated: August 26, 2011 6:30 AMJasper More will begin receiving life-saving but extremely expensive drug treatment within weeks for a rare genetic disorder.
PALMERSTON — Jasper More will begin receiving life-saving but extremely expensive drug treatment within weeks for a rare genetic disorder.
The Ontario Ministry of Health will fund the enzyme replacement therapy, which can cost up to $800,000 a year, for the 2 ½-year-old Palmerston boy who has Maroteaux-Lamy Syndrome (MPS Type VI). He was diagnosed in the spring.
He is the second Ontario child to receive treatment for the rare disease, which afflicts eight Canadians and roughly 1,100 people globally.
His parents, Darren and Pam More, feel enormous relief at news that the drug regimen is to begin as early as September.
“We’re a lot better than we were in April let me tell you,” Darren More said Wednesday. “In April, when we found out, we were just completely wrecked.”
MPS Type VI is the rarest in a family of diseases that fall under the umbrella of mucopolysaccharidosis disease, a metabolic disorder in which enzymes the body needs to break down sugar carbohydrates are missing or malfunctioning, compromising a child’s development as waste material builds up in the body.
There’s no known cure and the prognosis is a shortened lifespan. But with groundbreaking enzyme replacement therapy, patients can live nearly normal lives, advocates say.
Health Minister Deb Matthews said Wednesday the government is covering the cost after an evaluation by the executive officer of the provincial drug program, who looked at a variety of factors such as the therapy itself and assessments by other jurisdictions, and a rare diseases review committee.
Absolute conclusions, Matthews said, are difficult in cases of ultra-rare illnesses such as Jasper’s form of MPS, but she was happy with the funding decision.
“I’m very pleased,” she said. “It’s the right thing to do.”
The Mores have been helped in learning about MPS and appeals to the government by the Canadian MPS Society and the Isaac Foundation, a Campbellford, Ont. research funder founded by the parents of Isaac McFadyen.
Andrew McFadyen said his seven-year-old son, who has received government-supported therapy since 2006, is doing extremely well.
“(He’s) probably the healthiest he’s ever been. Essentially, we’ve stabilized the disease.”
McFadyen termed the treatment a godsend. “It saved his life, for sure.”
The foundation is funding research into treatments with an ultimate goal of a cure. It’s about to release findings on a research program at a New York hospital on MPS Type VI.
“That’s what Jasper and Isaac both have,” McFadyen said, stressing any advances made will have positive implications for all versions of MPS.
Jasper’s April diagnosis was confirmed through further testing in July, said his father, a purchasing manager for an Arthur-area firm. The couple has two other children, Daphnie, 9, and Clayton, 5, who are free of the disease.
More said when both parents are carriers, as they are, a child has a one-in-four chance of getting the genetic disease. “We had absolutely no idea we were carriers at all,” he recalled.
Jasper was born with two hernias and later developed a growth on his spine. Today, the boy has some enlarged organs and bone deformities, but the condition will improve with therapy. “Type VI is treatable. (It’s) not curable, but treatable.”
The enzyme replacement therapy offered by a California firm is tailor made, so it can’t be mass produced. “It has to be specific to the patient,” More said.
Jasper will have to travel each week to a London, Ont. hospital for an intravenous drip.
The treatments will last “as long as he lives, or until they find a cure,” More said.
Compassionate Funding Approved For Palmerston Child With Rare Condition
by Mike Robinson – August 26, 2011
PALMERSTON
Friends and supporters of the More family here can now breathe a collective sigh of relief.
After an initial denial of funds for life-sustaining treatment required by 2-1/2 year old Jasper More, a campaign by friends, family members and the Issac Foundation has successful changed that verdict by getting funds to be instated for his treatment.
In a recent interview, Darren More explained his son, Jasper, was first diagnosed in April.
However, Darren said, additional bloodwork, biopsies, and DNA testing were needed to determine exactly which type of Maroteaux-Lamy Syndrome (MPS VI) was present. That was not learned until July.
Jasper suffers from a rare enzyme deficiency called MPS VI (also known as Maroteaux-Lamy Syndrome).
Victims lack an enzyme in their blood that breaks down cellular waste in the body. That waste builds up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.
To date, there are eight confirmed cases in Canada, and roughly 1,100 worldwide.
Darren explained that while the majority of Maroteaux-Lamy Syndrome symptoms are similar, “the one benefit of MPS VI is that it does not affect the cognitive functions.”
Once Jasper’s condition was verified, the family began the process to get Ministry of Health funding treatment, Darren explained.
While there is no known cure for MPS VI, a treatment does exist. Naglazyme is an Enzyme-Replacement Therapy (ERT) designed to provide patients with a synthetic version of the enzyme they are lacking, by infusing small doses into the patient’s bloodstream on a weekly basis.
The treatment for the disease can range from $300,000 per year for a small individual to $1-million for a young adult.
Due to the lack of an orphan drug policy in Canada, Naglazyme is available only to Canadian patients through the federal government’s special access program. It is being used for patients in Ontario, British Columbia, and Quebec through the program and is funded by the provincial governments respectively.
While the treatment does not provide a cure, it has been proven to slow the progression of MPS VI in patients.
Darren added that initially the family was told it would take about four weeks to learn if funding was approved.
The More family contacted their local MPP John Wilkinson’s office. Darren said the staff were helpful and helped to contact the Minister of Health’s office directly to get the application fast-tracked.
“In the meantime, John Wilkinson met with us personally and went over the process step-by-step for the funding of rare diseases.”
Darren explained that Ontario’s health care system is geared to the health of 13 million people. He then noted there were three potential steps in the funding application process.
The first was for exceptional access; next, compassionate access; and the last, rare diseases access for funds.
The family was denied funding in the initial step of the application.
As a result, the Isaac Foundation drafted a press release calling on the provincial Liberal government to take action and ensure that Jasper More begin the treatment he requires immediately.
In addition, Darren added that family and friends who wanted to support them began sending emails, phone calls, tweets, and messages to their local MPPs, the Ministry of Health, and the premier’s office.
He believes those messages made a difference, because shortly after, the family was accepted for compassionate care access for funding.
“We are very relieved and grateful,” he said.
However, he was sad that it came down to having to campaign in order to obtain the health care funding.
While he recognized Wilkinson’s comments that the health care system is “designed for the average Joe,” there was already a precedent to allow the funding.
He cited the example of the parents of 7-year-old Isaac McFadyen, residents of Campbellford, who successfully lobbied the Ontario government to fund his expensive enzyme replacement therapy when he was diagnosed in 2006. It, too, required a very public campaign to secure funding. Isaac has been receiving his weekly infusions at The Hospital For Sick Children in Toronto for five years.
Prior to starting treatment, Isaac suffered from severe compression of his spinal cord that required the removal of a piece of his skull and a portion of his vertebrae. In addition, he endured numerous other surgeries to treat complications of the advancing disease in his body. Since beginning his weekly infusions, Isaac’s liver and spleen have reduced back down to a normal size, his rate of growth has increased, his heart function has improved, and his heart valve disease has stabilized. In addition, Isaac has had no further progression of his bone and joint disease, airway disease, and compression of his spinal cord.
Andrew McFadyen, Isaac’s father, believes that progression of the disease has virtually halted in his son, allowing him to lead a normal, active, lifestyle.
The McFadyen’s started a charity in 2006 called The Isaac Foundation, and have been raising money to fund innovative research projects that aim to find a cure for MPS VI. To date, the charity has given over $240,000 to research projects worldwide. In addition, the charity works nationally and internationally to advocate for and support families dealing with MPS VI.
Elizabeth Witmer, Progressive Conservative MPP for Kitchener-Waterloo, agrees. She worked tirelessly to advocate for the McFadyen family while they were fighting for Isaac’s treatment in 2006.
Witmer said, “We fought this battle once already for little Isaac, and we’ve witnessed the staggering results this treatment has brought to his quality of life. Jasper [More] deserves the same opportunities and hope that this life- sustaining treatment will provide him.”
“Since receiving this diagnosis, we’ve been sick with guilt, grief, and worry. Our world will never be the same,” said Jasper’s mother, Pam.
“The only comforting factor since the diagnosis has been that, unlike other types of MPS, type VI has a treatment available. We want to be able to do everything we can for our little boy to give him everything he deserves. This disease is not his fault.”
Darren added, “In our minds, that precedent should have meant the funding would be available in the first place.”
He fully realizes that it is a rare disease and the enzyme treatment is expensive.
The intravenous treatment provides enzymes missing in Jaspers body and will require a four hour treatment once a week for the rest of his life, or until a cure is found, Darren said. For the family, it means weekly trips to London for treatment.
And yet, Darren considers his family lucky.
Of the seven types of Maroteaux-Lamy Syndrome, only three respond to the treatment. Without treatment, those with the disease rarely live past their teens.
Even so, while the drug went to clinical testing only 10 years ago, those getting it have shown a vast improvement in the quality of life and Darren has high hopes for his son as well.
He extended thanks to Wilkinson, MPP Ted Arnott and Witmer; friends and family members for their love and support; and the Issac Foundation, which was one of the family’s biggest advocates.
“I don’t know what we would have done without them,” Darren said.
He later noted, “We, as a family, are taking part in the Toronto Waterfront 5km marathon this year, and all money we raise will be going to the MPS Society of Canada for support and research for sufferers of this disease.”
Funding Approved For Boy's Treatment
By Jeff Gard, Northumberland Today
ONTARIO – Jasper More, the two-year-old Palmerston boy recently diagnosed with a rare and debilitating enzyme deficiency disease, will receive treatment through provincial government funding after all.
As reported Wednesday, Aug. 17 through QMI Agency, Jasper was diagnosed six weeks ago with Maroteaux Lamy Syndrome (MPS VI). He is one of eight Canadians who the progressive disease, and its symptoms include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span. Only 1,100 people worldwide have MPS VI, for which there is no cure.
Darren More said he received a call Thursday, Aug. 18 from the family’s genetics doctor to inform them his son would receive the treatment Naglazyme through Canada’s Special Access Program and it will be funded by the Ontario government.
“It was absolutely breathtaking… literally breathtaking. I couldn’t breathe,” Darren More said of learning the news.
He and his wife Pam also have a nine-year-old daughter and five-year-old son.
“It’s such a huge weight off our shoulders. We’re so happy our child is going to get that treatment and have a chance at a normal lifestyle.”
Naglazyme is a treatment which provides patients with a synthetic version of the enzyme they are lacking by infusing small doses of it into the bloodstream. The treatment though can cost anywhere from $300,000 for a small individual to $1 million for a young adult each year. 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.
Jasper was originally denied funding for the treatment. The More family received that news on Monday, Aug. 15.
It was surprising news to a Campbellford family. Andrew and Ellen McFadyen’s son Isaac, who is now seven, has been receiving the treatment for five years because of provincial funding, which they had to lobby to get. Isaac makes a weekly trip to the Hospital for Sick Children in Toronto for the treatment.
The McFadyens established The Isaac Foundation in 2006 to raise money for innovative research projects that aim to find a cure for MPS VI. They also help families touched by the disease.
Since Aug. 15, the McFadyens helped lobby the government, and created awareness of the More case through social media, bringing it national attention.
“I 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,” Ontario Health Minister Deb Matthews told QMI Agency on Wednesday, Aug. 17. “I do know how important this is, and we want to do the right thing.”
Andrew McFadyen heard the news through Darren More.
“For Ellen and I, we’ve been through that and to experience with them in just a small way was very special,” he said.
McFadyen said the treatment for his son has made a world of difference.
“Essentially given him a new life,” he said. “We were told initially he wouldn’t make it to this age, but he has and right now he’s out playing with all of his friends like seven-year-olds should be doing. He has every chance in the world, like every kid should, and that’s all because of treatment. Jasper will have that chance now, too.”
jgard@northumberlandtoday.com
Funding Approved For Boy’s Treatment
By Jeff Gard, Northumberland Today
ONTARIO – Jasper More, the two-year-old Palmerston boy recently diagnosed with a rare and debilitating enzyme deficiency disease, will receive treatment through provincial government funding after all.
As reported Wednesday, Aug. 17 through QMI Agency, Jasper was diagnosed six weeks ago with Maroteaux Lamy Syndrome (MPS VI). He is one of eight Canadians who the progressive disease, and its symptoms include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span. Only 1,100 people worldwide have MPS VI, for which there is no cure.
Darren More said he received a call Thursday, Aug. 18 from the family’s genetics doctor to inform them his son would receive the treatment Naglazyme through Canada’s Special Access Program and it will be funded by the Ontario government.
“It was absolutely breathtaking… literally breathtaking. I couldn’t breathe,” Darren More said of learning the news.
He and his wife Pam also have a nine-year-old daughter and five-year-old son.
“It’s such a huge weight off our shoulders. We’re so happy our child is going to get that treatment and have a chance at a normal lifestyle.”
Naglazyme is a treatment which provides patients with a synthetic version of the enzyme they are lacking by infusing small doses of it into the bloodstream. The treatment though can cost anywhere from $300,000 for a small individual to $1 million for a young adult each year. 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.
Jasper was originally denied funding for the treatment. The More family received that news on Monday, Aug. 15.
It was surprising news to a Campbellford family. Andrew and Ellen McFadyen’s son Isaac, who is now seven, has been receiving the treatment for five years because of provincial funding, which they had to lobby to get. Isaac makes a weekly trip to the Hospital for Sick Children in Toronto for the treatment.
The McFadyens established The Isaac Foundation in 2006 to raise money for innovative research projects that aim to find a cure for MPS VI. They also help families touched by the disease.
Since Aug. 15, the McFadyens helped lobby the government, and created awareness of the More case through social media, bringing it national attention.
“I 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,” Ontario Health Minister Deb Matthews told QMI Agency on Wednesday, Aug. 17. “I do know how important this is, and we want to do the right thing.”
Andrew McFadyen heard the news through Darren More.
“For Ellen and I, we’ve been through that and to experience with them in just a small way was very special,” he said.
McFadyen said the treatment for his son has made a world of difference.
“Essentially given him a new life,” he said. “We were told initially he wouldn’t make it to this age, but he has and right now he’s out playing with all of his friends like seven-year-olds should be doing. He has every chance in the world, like every kid should, and that’s all because of treatment. Jasper will have that chance now, too.”
jgard@northumberlandtoday.com
A Great Big Day For Little Jasper
FIRST POSTED:
When I started to write it Thursday, I was steamed about the cold-hearted treatment little Jasper More was getting from Ontario’s health ministry.
Just two-and-a-half years old, Jasper has mucopolysaccharidosis VI (MPS VI), an inherited, life-threatening disease, (commonly known as Maroteaux-Lamy Syndrome).
It’s a rare but deadly condition that, untreated, will cause him a short life full of pain.
Most children who suffer from it grow no taller than three feet.
Without treatment, he’d also suffer from clouded corneas and likely have bone and heart-related issues. Few children, without treatment, survive their teen years.
With treatment, Jasper can live a fairly normal life.
But it’s costly and this week, Jasper’s parents, Darren and Pam, who live in Palmerston, were — initially — turned down for funding for the drug.
I was set to froth and fulminate about how the government that blew a billion dollars on an eHealth boondoggle, wouldn’t pay to save a baby’s life.
I was set to snap the government that gave the former eHealth CEO more than $300,000 in severance pay, can’t now find the cash to help little Jasper.
I was going to comment that if you’re a fatcat bureaucrat at the health ministry, you can open the public treasury and help yourself.
But for the average family with a kid with a rare disease? Too bad. So sad. Those taxes you pay are for gluttonous government officials only. Don’t even bother applying.
Then a miracle happened.
Between 9 a.m. when I called Health Minister Deb Matthews’ office for comment, and 4:45 p.m. when she called me back, a light went on deep in the bowels of the ministry.
The government changed its mind. The ministry changed its mind.
Little Jasper will get his treatment paid for, Matthews told me.
“The executive officer just today made the decision that he (Jasper) would receive the drug,” Matthews said.
“I think it’s important to underline that we are absolutely committed to making evidence-based decisions and we will continue to do that, but in these ultra-rare diseases it is very difficult to get the evidence that a drug will be effective,” she said.
Since it’s a disease that only afflicts one in six million people, it’s tough to get the kind of evidence they need to evaluate the drug, Matthews added.
The medication Jasper needs, Naglazyme, is manufactured by the pharmaceutical giant, BioMarin.
His dad, Darren, doesn’t know exactly how much the treatment will cost. He’s been given estimates between $300,000 and $1 million.
“It is very patient-specific. They can’t make it in large doses, they have to make it specific to the patient,” he explained.
The family applied for the funding under the province’s Exceptional Access program.
Last week, Environment Minister John Wilkinson came to their home to talk to them about funding.
“He explained the different processes for applying for rare funding, but we got the feeling he was only there to smooth things over before we got the actual denial,” said Darren.
The bombshell dropped Monday, when the ministry told them they’d been refused funding. Now, that death sentence has been lifted.
Hallelujah! Someone has a heart.
I think it’s Matthews. Good for her for doing the right thing.
Reached Thursday night, Darren said he was, “ecstatic.” His doctor phoned him with the good news.
“I could barely breathe,” he said. “It is such a weight off our shoulders.”
Thursday was a bad day for cold-hearted bureaucrats.
And a great one for little Jasper.
McFadyens Join Fight To Get Government Funding For Boy's Treatment
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
McFadyens Join Fight To Get Government Funding For Boy’s Treatment
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