Rare Disease
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
Family Lobbies For Sick Tot’s Treatment
Former Blue Jay Roy Halladay with Isaac McFadyen who was diagnosed with the very rare disease called MPS VI. (QMI Agency)Jeff Gard, QMI Agency
CAMPBELLFORD, Ont. – An Ontario family is lobbying for provincial funding so their two-year-old boy can get treatment for his rare and debilitating enzyme deficiency disease.
And they’re doing it with the help of another Ontario family who went through the same fight five years ago.
Seven-year-old Isaac McFadyen from Campbellford, Ont., is one of eight people Canada diagnosed with Maroteaux Lamy Syndrome (MPS VI). Only 1,100 worldwide 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, which they had to lobby to get.
About five years after Isaac’s diagnosis, a Palmerston, Ont., 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,” said Andrew McFadyen, Issac’s father. “To me, it’s a travesty and it’s not representing equal access to health care for everybody.”
However, Health Minister Deb Matthews noted 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.
While there’s no cure for MPS VI, there is the life-sustaining treatment available called Naglazyme, which provides patients with a synthetic version of the enzyme they are lacking by infusing small doses of it into the bloodstream.
The treatment can cost anywhere from $300,000 for a small individual to $1 million for a young adult each year.
“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.
“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.”
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 has been receiving the treatment once a week for five years at the Hospital for Sick Children in Toronto.
Told of the More family’s circumstances, Ontario Premier Dalton McGuinty said his heart goes out to the family in their difficulties, but that he had no first-hand knowledge of the case and could not comment. He suggested getting in touch with Health Minister Deb Matthews.
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.
The Mores issued an appeal to the rejection of funding on Tuesday.
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.
– With files from Sheena Goodyear
Family Lobbies For Sick Tot's Treatment
Former Blue Jay Roy Halladay with Isaac McFadyen who was diagnosed with the very rare disease called MPS VI. (QMI Agency)Jeff Gard, QMI Agency
CAMPBELLFORD, Ont. – An Ontario family is lobbying for provincial funding so their two-year-old boy can get treatment for his rare and debilitating enzyme deficiency disease.
And they’re doing it with the help of another Ontario family who went through the same fight five years ago.
Seven-year-old Isaac McFadyen from Campbellford, Ont., is one of eight people Canada diagnosed with Maroteaux Lamy Syndrome (MPS VI). Only 1,100 worldwide 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, which they had to lobby to get.
About five years after Isaac’s diagnosis, a Palmerston, Ont., 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,” said Andrew McFadyen, Issac’s father. “To me, it’s a travesty and it’s not representing equal access to health care for everybody.”
However, Health Minister Deb Matthews noted 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.
While there’s no cure for MPS VI, there is the life-sustaining treatment available called Naglazyme, which provides patients with a synthetic version of the enzyme they are lacking by infusing small doses of it into the bloodstream.
The treatment can cost anywhere from $300,000 for a small individual to $1 million for a young adult each year.
“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.
“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.”
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 has been receiving the treatment once a week for five years at the Hospital for Sick Children in Toronto.
Told of the More family’s circumstances, Ontario Premier Dalton McGuinty said his heart goes out to the family in their difficulties, but that he had no first-hand knowledge of the case and could not comment. He suggested getting in touch with Health Minister Deb Matthews.
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.
The Mores issued an appeal to the rejection of funding on Tuesday.
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.
– With files from Sheena Goodyear
Ontario Ponies Up For Sick Tot’s Medical Bills
By Jeff Gard, QMI Agency – August 08, 2011
CAMPBELLFORD, Ont. – Jasper More, the two-year-old Palmerston, Ont., boy recently diagnosed with a rare and debilitating enzyme deficiency disease, will receive treatment through provincial government funding after all.
As reported Wednesday by QMI Agency, Jasper was diagnosed six weeks ago with Maroteaux Lamy Syndrome (MPS VI).
He is one of eight Canadians who has 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, Jasper’s dad, said he received a call Thursday from the family’s genetics doctor to inform them Jasper 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,” More said. “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 that provides patients with a synthetic version of the enzyme the disease’s sufferers lack by infusing small doses of it into the bloodstream. It 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.
It was surprising news to a Campbellford, Ont., 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 Monday, the McFadyens helped the Mores 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. “I do know how important this is, and we want to do the right thing.”
Andrew McFadyen heard the news through 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.”
The Ministry of Health could not be reached for comment Thursday.
Ontario Ponies Up For Sick Tot's Medical Bills
By Jeff Gard, QMI Agency – August 08, 2011
CAMPBELLFORD, Ont. – Jasper More, the two-year-old Palmerston, Ont., boy recently diagnosed with a rare and debilitating enzyme deficiency disease, will receive treatment through provincial government funding after all.
As reported Wednesday by QMI Agency, Jasper was diagnosed six weeks ago with Maroteaux Lamy Syndrome (MPS VI).
He is one of eight Canadians who has 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, Jasper’s dad, said he received a call Thursday from the family’s genetics doctor to inform them Jasper 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,” More said. “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 that provides patients with a synthetic version of the enzyme the disease’s sufferers lack by infusing small doses of it into the bloodstream. It 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.
It was surprising news to a Campbellford, Ont., 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 Monday, the McFadyens helped the Mores 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. “I do know how important this is, and we want to do the right thing.”
Andrew McFadyen heard the news through 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.”
The Ministry of Health could not be reached for comment Thursday.
Isaac Foundation Gala Raises $20,000
The McFadyen family from Campbellford is determined to help find a cure for MPS VI.
Andrew and Ellen McFadyen created The Isaac Foundation in 2006 after their first-born son Isaac, now six years old, was diagnosed with the ultra-rare disease, caused by enzyme deficiency, the previous year.
Only four children in Canada are currently known to have MPS VI while others around the world live with it as well.
Symptoms include stiffening joints, stunted growth, heart and airway disease, spinal cord compression, and a shortened life span.
“When Isaac was first diagnosed, my wife Ellen and I were struggling to come to grips with our new reality. Initially, we thought that the hopes and dreams that we had for our son were automatically taken away,” Andrew McFadyen told the crowd attending the foundation’s first Gala For A Cure on Friday night at the Best Western Inn and Convention Centre in Cobourg. “Obviously, we were worried about his quality of life, his life span and how we were going to cope with giving him the support he would need as his disease progressed.”
They wanted to do everything possible to help their son, but they knew they couldn’t do it alone.
Although is no cure for MPS VI, a type of Enzyme Replacement Therapy (ERT) called Naglazyme was approved in the United States in June 2005. However, the cost for treatment, depending on body weight, runs between $300,000 to $1,000,000 per year.
Andrew McFadyen recounted seeking the help of Conservative MPP Elizabeth Witmer, who was the opposition’s health critic at the time. He called her “in a panic one evening, desperate for someone to help us advocate and solicit the government to fund the treatment that Isaac needed in order to slow the progression of his disease.”
Witmer scheduled a meeting with McFadyen the following day at her office at the Ontario Legislature. He arrived with his literature and brochures as well as a copy of an Isaac Foundation DVD.
The meeting turned out to be a conversation about their families.
“After a few hours, Elizabeth told me that she was going to help me and not, as she said, because she was a politician, but because she was a mother,” McFadyen said. “From that day forward, she worked tirelessly for our son and, I argue, he is here today because of her steadfast efforts.”
Witmer successfully helped the McFadyens lobby the provincial government to fund the ERT treatment’s for Isaac.
McFadyen introduced Witmer as having “an illustrious 20-year career as an MPP,” including a stint as the Deputy Premier of Ontario. She has also served in numerous cabinet positions, including being the Minister of Labour, Minister of Health, Minister of Education and Minister of the Environment.
“But to us, more importantly, Elizabeth is the person who championed our cause in the Ontario Legislature and led the fight to save our son from the ravages of his disease,” McFadyen said.
McFadyen pointed out that it was evident to most in the room that he is a Liberal. He even ran to be the local federal candidate in 2009. He said the question he was asked the most during his campaign was about the involvement of a Conservative MPP and why he would seem support from a sitting member of another party.
“My answer then and my answer today is plain and simple — I wanted to save the life of my son,” McFadyen said. “As I look out at him now and I admire his incredible smile, and his amazing zest for life, I’m not sorry whatsoever that I sought the support outside my party lines. Advocacy for the life of a child should never be constrained to boundaries of any one party, and I feel sorry for those that feel it should.
“Elizabeth Witmer was there for us when no one else was and our son is here today because of her tenacious work, her kind and caring nature, and her steadfast refusal to accept no for an answer from our government.”
McFadyen noted that Witmer visited Isaac at the The Hospital for Sick Children about a month after his treatments began.
On Friday, The Isaac Foundation presented Witmer with its first recognition award, which will be presented annually to a different recipient on behalf of the foundation. The plaque reads, ‘Lifetime Impact Award – Presented to Elizabeth Witmer For Creating Positive Change in the Life of Isaac and Advocating for Those Affected By Rare Diseases.’
Following the presentation, Witmer said the award was “heartwarming.”
“It just makes you so happy that you could be a small part in giving him the quality of life he enjoys today,” Witmer told Northumberland Today. “I think Isaac is so fortunate to have parents like Andrew and Ellen that have advocated so much on his behalf. And he’s such an engaging little boy.”
Witmer recalled that when she first approached the government, “we weren’t given any reason for hope or optimism at that time.”
She invited the McFadyens to Queen’s Park and introduced them to other ministers.
She couldn’t have been happier when then-health minister George Smitherman announced the treatments would be funded.
“This was a child who needed help,” Witmer said. “This was something we should and needed to do.”
Of being a politician, “you can make a difference,” she said.
“We’re human beings like everyone else. I’m a mother,” added Witmer, who attended the event with her daughter Sarah. “I’m hoping we will find a cure. I’m confident we will.”
Witmer said of all the awards she has won, the award from The Isaac Foundation will hold special meaning to her.
“There’s nothing better than this,” she said.
* * *
Also on Friday night, The Isaac Foundation debuted its updated DVD, recorded and edited by Andrew McFadyen’s brother Cameron at Whistlestop Productions and their friend Jared Coleman from Shoreline Productions.
Wine tasting was provided by Black Prince Winery.
Canadian singer/songwriter Ron Sexsmith performed an intimate acoustic set for the crowd and a silent auction, including an autographed guitar from international music sensation John Mayer, was held. The auction was also well supported by numerous businesses throughout Northumberland County.
Funds raised by The Isaac Foundation are directed to support and fund innovative research projects that aim to find a cure for MPS VI.
Costs amount to about $100,000 each year to run one project with one full-time researcher, McFadyen has stated.
McFadyen was ecstatic to announce earlier this week that the gala — a new fundraiser for the Foundation — had raised $20,000.
“Raising $20,000 at the gala is wonderful because it gives us enough to top off the research grant that we were waiting to hand out. With these funds, we can now go ahead and send a cheque out the door, ensuring continuous funding for a project that we believe in very much,” McFadyen said. “And it’s nice to be able to inform our donors, and the guests at the gala, that their money is making a difference and that it will go a long way to providing a better quality of life by allowing these research projects to continue.”
jgard@northumberlandtoday.com
twitter.com/NT_jgard