11 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Blog Postings, Fight For Aleena Permalink

ERT – Proof of Effectiveness From Around The World

Sticky Post By mcfadyena On 11 August, 2013

The question of whether Alberta Health will fund treatment for Aleena will be based solely on whether the province wants to pay the high cost associated with their decision. How do I know this? Well – if they were basing their decision on evidence based science alone, they would have rendered their decision by now – and that decision would have to be a YES. To support that claim, I thought I’d take the time to include some snippets from some of the finest MPS Researchers in the world. These quotes are taken directly from readily-available and reputable Journals, with sources included for further study. There is irrefutable proof that this treatment is effective. There is irrefutable proof that this treatment MUST begin at a very early stage. Delaying a decision on providing this treatment for Aleena is detrimental to her long-term health!

Here’s some things to ponder and share. It took me less than 1/2 day to do this review of the available scientific literature regarding the safety and effectiveness that ERT yields patients suffering from MPS VI. How it’s taken Alberta Health over 4 weeks to find this information is beyond comprehension. And if they didn’t find this information during their STEDT review, they haven’t done a thorough job evaluating this case at all! I should note that this is just a small sampling of the literature that is available on the subject, and every article I dug up promotes the same overall message – ERT is the best treatment for children suffering from MPS VI, it’s EFFECTIVE, and it must begin EARLY. One final note – all articles point to this treatment being SAFE. With all this clinical data available, Alberta Health must simply be weighing whether they want to pay to save Aleena or not, and shame on them if that’s the case.

Please read and share. #Treatment4Aleena

“Recently, a consensus panel of international experts in medicine, genetics and biochemistry drafted management guidelines for MPS VI. The expert panel recommended ERT, when available, as first-line therapy.” (Schlander, M. and Beck, M. Current Medical Research and Opinion, 2009)

“Within 24 weeks of treatment, most patients treated with ERT demonstrated significant and sustained improvements in performance in [6 and 12 minute walking tests]. Long-term safety data show that the therapy has an acceptable safety profile.” (Harmatz, P. Turkish Journal of Pediatrics, 2010)

“…initiating ERT at an early age is safe and improves overall morphology, clinical outcome, quality of life and the safety profile related to immune response. The main benefit was in scoliosis, joint range of movement, cardiac valves and facial appearance.” (McGill JJ, Inwood AC, Coman DJ, Lipke ML, de Lore D, Swiedler SJ, Hopwood JJ. Clinical Genetics, 2010)

“As ERT slows down the accumulation of GAG in cells and tissues, it is thought that early treatment might prevent or delay the development of irreversible disease manifestations and limit or prevent growth deceleration.” (Harmatz, P. Turkish Journal of Pediatrics, 2010)

“…this trend toward decline in pulmonary function can be halted and partially reversed during ERT…” (Harmatz, P., Yo, Z., Giugliana, R., Schwartz, V., Guffon, N., Teles, E., Miranda, C…Decker, C. Journal of Inherited Metabolic Disease, 2009)

“One recently published case control study assessed the impact of [ERT] in two siblings: one treated from the age of 8 weeks, one from 3.6 years. After 3.6 years of treatment with [ERT], the youngest child had a lack of scoliosis and preserved joint movement, cardiac valves and facial morphology, unlike the older sibling at the same age. The older sibling had improvements in joint mobility and cardiac valve disease after 3.6 years of treatment with [ERT].” (Harmatz, P. Turkish Journal of Pediatrics, 2010)

“…it seems desirable to start treating these patients at an early stage, before irreversible damage has occurred.” (Schlander, M. and Beck, M. Current Medical Research and Opinion, 2009)

“[ERT] was found to be safe and effective in this young patient population and similar to that seen in clinical trials with older patients.” (Horovitz, D., Magalhaes, T., Acosta, A., Ribeiro, E., Giuliani, L., Palhares, D., Chong, K…Llerena Jr., J. Molecular Genetics and Metabolism, 2013)

“This improvement in respiratory function relative to baseline may lead to a decrease in the severity of respiratory illnesses and number of hospitalizations, and an overal improvement in the quality of life of MPS VI patients.” (Harmatz, P., Yo, Z., Giugliana, R., Schwartz, V., Guffon, N., Teles, E., Miranda, C…Decker, C. Journal of Inherited Metabolic Disease, 2009)

“This therapy opens the door to a more proactive approach of managing the disease, i.e slowing down the accululation of GAG rather than alleviating the resulting clinical manifestations.” (Harmatz, P. Turkish Journal of Pediatrics, 2010)

“…conventional cost-effectiveness criterion currently in widespread use does not offer sufficient basis for rejecting reimbursement of expensive treatments for exceptionally rare disorders.” (Schlander, M. and Beck, M. Current Medical Research and Opinion, 2009)

“The prescribed dosage of 1 mg/kg IV weekly with galsulfase ERT is shown to be safe and effective in slowing and/or improving certain aspects of the disease.” (Horovitz, D., Magalhaes, T., Acosta, A., Ribeiro, E., Giuliani, L., Palhares, D., Chong, K…Llerena Jr., J. Molecular Genetics and Metabolism, 2013)

“[ERT] weekly has shown to be safe and effective in slowing progression and/or improving the burden of the disease for MPS VI in young children. As early treatment initiation results in improved patient outcomes in this young cohort, early recognition of the more subtle symptoms associated with slowly progressing disease should be a priority to ensure early diagnosis and treatment initiation.” (Horovitz, D., Magalhaes, T., Acosta, A., Ribeiro, E., Giuliani, L., Palhares, D., Chong, K…Llerena Jr., J. Molecular Genetics and Metabolism, 2013)

“ERT positively affected mobility of the shoulder joint, the size of the liver and spleen, cardiac parameters, pulmonary function, certain domains of [quality of life], and the level of GAGs in the urine.” (Brands, M. Oussoren, E., Ruijter, G., Vollebregt, A., van den Hout, H., Joosten, K., Hop, W., Plug, I., Ploeg, A. Molecular Genetics and Metabolism, 2013)

“…results of ERT treatment in MPS VI have been promising, demonstrating clinically and statistically significant improvements in endurance along with a reduction in urinary GAGs.” (Harmatz, P., Giugliani, R., Schwartz, I., Guffon, N., Teles, E., Miranda, M., Wraith, J…Decker, C. Molecular Genetics and Metabolism, 2008)

08 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Editorial: Life-saving treatment a worthwhile investment

Sticky Post By mcfadyena On 8 August, 2013

August 8, 2013 by editor

What’s good for the goose, they say, is good for the gander.

That’s all fine and dandy for birds who fly south in the winter, but is what’s good for the Ontarian good for the Albertan? What about what’s good for the Quebecer?

When it comes to treatment for the life-threatening medical condition known as MPS VI, it certainly is.

This extremely rare condition — there are currently only nine cases reported in Canada, and 1,100 worldwide — came into the limelight earlier this week when we found out that a three-year-old girl from

St. Albert, Aleena Sadownyk, was diagnosed with MPS VI earlier this year and that her family is wrestling with the Alberta government to have the treatment that could save her life funded through health care programs.

The drug she needs has not been fully approved by Health Canada, meaning that the Alberta government is dragging its feet by conducting its own clinical review. But similar reviews have already been conducted by governments in Ontario, Quebec, British Columbia and Saskatchewan, where funding for MPS VI treatment is already in place, and there is very little chance Alberta is going to find anything different. So why not get the funding in place already? There is ample evidence to prove that it works and is a worthwhile investment, even at $300,000 to $1 million per year.

And kudos to St. Albert MLA Stephen Khan for speaking out on Tuesday and calling for the treatment to be funded sooner rather than later. It’s refreshing to hear an MLA remember that his first job is to represent and advocate for the people of his riding, rather than to simply toe the Progressive Conservative party line.

Every day that passes puts Aleena Sadownyk’s young life further in jeopardy. The least the Alberta government — and especially Health Minister Fred Horne — could do is stop dragging its feet and take action.

— GLENN COOK, St. Albert Leader

07 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena Permalink

PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP

Sticky Post By mcfadyena On 7 August, 2013

FOR IMMEDIATE RELEASE

AUGUST 7, 2013

PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP

EDMONTON – Today, New Democrat health critic David Eggen sent a letter to Minister Fred Horne requesting that he immediately approve funding for Aleena Sadownyk, a St. Albert toddler, who requires life-saving medical treatment. Eggen is also calling on the PC government to list the necessary treatment for future patients.

“This is another sad and stark example of the PCs’ approach to health care—they prefer to diminish and delist services instead of strengthening and expanding them,” said Eggen. “As a result, this young girl and her family have been forced into a crisis constructed from PC neglect.”

Aleena suffers from the rare enzyme deficiency disease MPS VI, and the treatment, Naglazyme, is not currently funded by the province. Previous attempts by the family to secure funding for Aleena’s treatment have been unsuccessful.

“The PCs must immediately fund treatment for this young girl,” said Eggen. “Beyond that, the Minister needs to commit to funding treatment for others in the future.”

A copy of Eggen’s letter to the Minister is attached. (HERE)

For more information, please contact:
Brad Lafortune, Communications Officer: 780-446-2375 or Bradley.Lafortune@assembly.ab.ca<mailto:Bradley.Lafortune@assembly.ab.ca>

07 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena Permalink

PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP

Sticky Post By mcfadyena On 7 August, 2013

FOR IMMEDIATE RELEASE

AUGUST 7, 2013

PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP

EDMONTON – Today, New Democrat health critic David Eggen sent a letter to Minister Fred Horne requesting that he immediately approve funding for Aleena Sadownyk, a St. Albert toddler, who requires life-saving medical treatment. Eggen is also calling on the PC government to list the necessary treatment for future patients.

“This is another sad and stark example of the PCs’ approach to health care—they prefer to diminish and delist services instead of strengthening and expanding them,” said Eggen. “As a result, this young girl and her family have been forced into a crisis constructed from PC neglect.”

Aleena suffers from the rare enzyme deficiency disease MPS VI, and the treatment, Naglazyme, is not currently funded by the province. Previous attempts by the family to secure funding for Aleena’s treatment have been unsuccessful.

“The PCs must immediately fund treatment for this young girl,” said Eggen. “Beyond that, the Minister needs to commit to funding treatment for others in the future.”

A copy of Eggen’s letter to the Minister is attached. (HERE)

For more information, please contact:
Brad Lafortune, Communications Officer: 780-446-2375 or Bradley.Lafortune@assembly.ab.ca<mailto:Bradley.Lafortune@assembly.ab.ca>

07 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Local man fights for Alberta child’s health

Sticky Post By mcfadyena On 7 August, 2013

By Jeff Gard, Northumberland Today

Wednesday, August 7, 2013 1:43:27 EDT PM

Andrew McFadyen is ready for the launch of Project One Million.<br />JEFF GARD/Northumberland Today

Andrew McFadyen is ready for the launch of Project One Million. JEFF GARD/Northumberland TodayPrint

CAMPBELLFORD – For a Campbellford father, this is just another fight for funding, albeit with a different province.

Andrew McFadyen is lobbying Alberta Health to approve life-sustaining treatment for three-year-old Aleena Sadownyk, who was recently diagnosed with the rare enzyme deficiency MPS VI (Maroteaux-Lamy Syndrome). Those affected lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG), which builds up in the bones, tissues, organs and muscles. It can lead to devastating symptoms such as heart and airway disease, corneal clouding, stiffening of the joints, shortened stature and premature death.

Treatment — an Enzyme Replacement Therapy called Naglazyme — can range from $300,000 for a small individual to $1 million for a young adult per year. There are currently nine children suffering from MPS VI in Canada and about 1,100 cases worldwide.

McFadyen and his wife Ellen’s nine-year-old son Isaac suffers from MPS VI, but he has been receiving treatment on a weekly basis for seven years at The Hospital for Sick Children in Toronto after he was original denied funding. The McFadyens lobbied and they won.

Since that time, the family has also successfully lobbied for funding for another Ontario boy, Jasper More, in 2011 and a Saskatchewan girl, Violet Revet, in 2012.

Now the focus is on the St. Albert, Alberta toddler Aleena Sadownyk, who was denied funding by Alberta Health through the Alberta Rare Diseases Funding Program. A second application through the Short Term Exceptional Drug Therapy program is currently being reviewed, McFadyen said.

“It’s awful… the fact this has to continue to play out the same way every time a child gets diagnosed in a new province,” McFadyen said, while noting it spotlights Canada’s need for an Orphan Drug Policy. “The little girl’s family has to wait (for the decision) and it’s not right. It’s tough reliving this experience, but you empathize with the family.”

His advocacy work includes checking in with Alberta Health several times per day, ensuring the ministry has received all of the information it needs to make a proper decision and know that precedent has been set. In addition, he works with opposition health critics and prepares news releases.

McFadyen said when a child was diagnosed with MPS VI in Quebec, that province followed precedent already set by Isaac and subsequent cases. He hopes Alberta will do the same.

These funding fights engender “mixed emotions” in McFadyen, who used the words “angry” and “stressful” to describe the process.

“This takes over everything in life but it’s a small sacrifice for a little girl and her family,” McFadyen said. “It’s rewarding when success comes, knowing what it means for the family and the kids.”

jeff.gard@sunmedia.ca

twitter.com/NT_jgard

06 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media Permalink

FOR IMMEDIATE RELEASE – PC MLA Calls on Government to Fund Treatment for Rare Disease

Sticky Post By mcfadyena On 6 August, 2013

– FOR IMMEDIATE RELEASE –

PC MLA Calls on Government To Fund Treatment For Rare Disease

Life-Sustaining Treatment Required Immediately; Family Continues To Wait For Decision From Alberta Health

Stephen Khan, MLA for St. Albert, released his first public statement today about the case of 3 year-old Aleena Sadownyk. Sadownyk, a St. Albert resident, was recently diagnosed with MPS VI, a rare, progressive, and debilitating disease caused by an enzyme deficiency in the blood. She requires a life-sustaining treatment immediately to prevent irreversable symptoms from progressing. However, treatment cannot begin until Alberta Health renders its decision as to whether they will fund the expensive treatment or not. The treatment is already being funded in numerous provinces throughout the country, including BC, Saskatchewan, Quebec, and Ontario.

Khan released a statement on his Facebook page urging Alberta Health to quickly render “a positive decision for the Sadownyk family.” Khan notes that “Every day is an eternity for this young family, knowing that a treatment for this debilitating disease is at hand.” He acknowledges that other provinces are already funding treatment for the same disease.

Khan’s call for a quick decision from Alberta Health backs up Wildrose Health Critic Heather Forsyth in her own campaign for immediate action from Alberta Health. Forsyth has been publicly advocating on behalf of the Sadownyk family. On Friday, she wrote a letter to Health Minister Fred Horne asking him to “immediately reverse the denial of coverage under the Alberta Rare Diseases Drug Program or at the very least, provide temporary access to Naglazyme by immediately approving the STEDT application.” Forsyth notes in her letter to Horne that “major barriers thrown up in accessing this drug for Aleena” have caused delays that have left Aleena suseptable to “irreversible symptoms” of the very progressive disease.

While the drug in question isn’t yet approved by Health Canada, it’s official recognition as a legal medication for use in this country is imminent, perhaps only weeks away. It is already approved for use in many countries throughout the world, including the United States, the European Union and Austrailia. There are currently 7 children receiving the medication throughout Canada, all funded by provincial health care plans.

Andrew McFadyen, director of The Isaac Foundation, a charity that advocates on behalf of families affected by the disease, has grown frustrated with the delays and indecision. “There is ample evidence in this country and throughout the world as to the merits of this life-sustaining treatment. I’ve seen it personally in my son Isaac, where it has virtually halted the progression of the disease and has given him the opportunity of a normal life. It’s heartbreaking to know that while bureaurocrats are slowly make their way through the STEDT process – with no real indicaton or urgency, the disease is wreaking havoc on Aleena’s body. We know this works. We know other provinces already fund this treatment because they also know it works. Why Alberta Health is making this family wait to learn the fate of their daughter is beyond comprehension and it’s downright cruel.”

McFadyen adds that he is hopeful the public statement from Khan will prompt Minister Horne to take immediate action. “The Provincial government has the opportunity to do the right thing and ensure that Aleena receives the treatment she so desperately needs. It’s the role of government to protect and ensure fair and equitable access to Health Care for all Canadians, regardless of which Province they happen to live in. The Isaac Foundation joins Mr. Khan in calling on this government to take action and save the life of this child. She can’t afford to wait.”

# # #

For more information about this topic, or to schedule an interview to discuss, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com.

Stephen Khan’s full statement can be found here.

Heather Forsyth’s full letter to Minister Horne can be found here.

06 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Toddler’s family seeks relief from rare disease

Sticky Post By mcfadyena On 6 August, 2013

St. Albert child awaits approval of costly treatment

By: Stu Salkeld

| Posted: Tuesday, Aug 06, 2013 12:15 pm

RARE CONDITION – Three-year-old Aleena Sadownyk of St. Albert is battling MPS VI, an extremely rare disease that governments seem to be slow to address.

Supplied photo

The MLA for St. Albert said a very young member of his constituency is front and centre in the government’s, and public’s, eye.

Stephen Khan said he spent the weekend talking to people involved in the situation facing three-year-old Aleena Sadownyk. The three-year-old St. Albert resident has been diagnosed with the extremely rare Maroteaux-Lamy Syndrome, also called MPS VI.

Sufferers of MPS VI lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG). These GAGs build 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 nine children suffering from the disease in Canada and roughly 1,100 worldwide.

“This adorable little girl, there is a drug that helps treat the deficiency that she has,” said Khan from his home Monday.

“The catch is, the drug has not been approved for use in Canada.”

MPS VI is rare, and the treatment required to keep Aleena healthy is also rare and quite expensive, ranging from $300,000 to $1 million per year, but urgently needed to keep her situation from worsening. Naglazyme is the only treatment known for the condition, and Alberta Health Services has already denied funding under the Alberta Rare Diseases Funding Program, but Khan said other avenues are opening up.

“The family is under unimaginable strain and stress,” said Khan. He said his St. Albert office was notified July 4 about Aleena and her story was quickly forwarded to the minister of Health. From there, the minister’s office is to forward it to AHS.

He said, although Naglazyme is not approved for use in Canada, some provinces have made exceptions. Khan said he’s been talking with the ministry and also with representatives of the Sadownyk family to get the ball rolling more quickly. Khan said he wanted to meet directly with the Sadownyk family, but was unable to. However, the minister’s office stepped up and has been handling the situation.

“We’re hoping to get access as soon as possible because every day is an eternity for the family,” said Khan.

Frustration

Andrew McFadyen, whose son Isaac lives with MPS VI, lobbied the Ontario government to cover Naglazyme and has been instrumental in helping the Sadownyk family.

“I’ve just become more and more frustrated with what’s been transpiring,” said McFayden Tuesday.

“We’re essentially waiting on the province to make a decision and that’s why it’s important we do get an expedited decision on this matter. Every day that goes by is another day that she’s not receiving the therapy that she needs.

“We’re reaffirming our call for an immediate decision on this so that the family can know what the fate of their daughter is. The future of your child sits on the desk of a few bureaucrats that haven’t had the opportunity to work through a process they haven’t put in place in an expedited fashion and we’re just calling on them to do the right thing.”

McFayden knows this disease well as it afflicted his son. He knows what Aleena is facing.

“Already at three-and-a-half years old she’s suffering from a lot of the irreversible symptoms of this disease, her joints are starting to stiffen up, her hands are starting to claw up, she can’t raise her hands above her head,” said McFayden.

“Her energy level is severely, severely deteriorating, her internal organs are enlarged, there’s already evidence of buildup on the bones. And those are only the things that are prevalent. We just don’t know what sort of symptoms are just waiting on the doorstep to appear. None of these symptoms can be reversed, but as soon as treatment starts we hope that the majority of these symptoms will stop appearing.

“Her lifespan will be severely shortened without treatment.”

Khan said the issue is bringing people together, even in the legislature, as all parties seem to want to see an approval made for the treatment to help Aleena. “I think everybody in Alberta can be supportive of the Sadownyk family and their cause.”

Khan said everyone is waiting for the provincial committee that oversees the Short Term Exceptional Drug Therapy program to meet and make a decision. He said the federal government is also working on this issue through the Special Access Program, which already approved Naglazyme for MPS VI sufferers in Ontario, B.C., Saskatchewan, and Quebec. Naglazyme is already approved in other countries around the world, including the United States and members of the European Union.

Khan said no one knows what the committee is going to do yet, but he is optimistic.

“From the time our office first contacted the minister’s office, we’ve been getting regular updates,” said Khan.

“The last update we got was very positive, very hopeful. The quicker we can get a decision, the happier everyone is going to be.”

06 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Toddler's family seeks relief from rare disease