Godspeed, Heather. You Will Be Missed!

As the Alberta Election race enters the final hours, so too does the official time in office for one of the most honourable and compassionate souls I have ever met. Once the results are announced tonight, the retirement of Heather Forsyth officially begins, bringing an end to a highly successful political career.

I met Heather a few years ago when I was struggling to find help for a little girl, Aleena Sadownyk, who needed immediate access to life-saving treatment.  Because this treatment is expensive (the fourth most expensive drug in the world), we were getting nowhere with the PC government, at that time led by Premier Alison Redford.  Desperate for help, I contacted Heather to see if there was anything she could do in her capacity as the Official Opposition Health Critic.

The moment I spoke with Heather, I knew I had connected with someone that would do everything in her power to help this little girl.  She listened to everything I had to tell her, keyed in on the most important facts of our case, and sprang to action immediately after our initial phone call was over.  I was impressed, and had Hope again for helping little Aleena.

Now, a quick aside.  Over the years, I’ve had the pleasure (sometimes) of working with many politicians throughout the country.  Oftentimes, these politicians offer their help during advocacy cases to further their own stature within political circles – helping with these advocacy cases usually translates into scoring political gain against the government of the day, and members of the Official Opposition jump at the chance to score such points.  I’m not naive in the slightest – I understand that many politicians get involved in my advocacy pushes because of the prospect to score those sought after blows against the government.  And I’ve never minded that fact – I’ll do anything to help our kids suffering from rare diseases, and if a politician wants to help out – for whatever reason – who am I to turn down the help offered, especially when we are talking life and death situations for our kids?

4d5f8030f60e11e2ad2b22000ae80c6b_7While I was impressed with the speed that Heather and her team sprang to action to save Aleena, I was more impressed that I felt she was doing it out of a love for helping people, and a passion for doing what’s right.  Moreover, I truly felt she was working as hard as she did because of the heartache and heartbreak she felt for what Aleena and her family were dealing with.  However, my close friends were skeptical – they have heard me speak often about the help I’ve received in the past and they were sure Heather took this case on for those same reasons.  They were sure she was doing this for political gain and for personal ambitions within that political world.  I vehemently disagreed – I consider myself a good judge of character, and I was sure that Heather was one of those rare people in the world of politics who were helping because they could, and because she felt that it was incumbent on her to make a difference in the life of this little girl if she were able.

And you know what?  My first impression was right.

Shortly after Aleena had her treatment approved – solely due to the hard work of Heather and her team led by Matt Solberg – Heather shared with me that she would not be seeking re-election the next time the Province when to the polls.  Win or lose, Heather didn’t have a political future she needed to worry about.  Political gain didn’t play into the situation at all, though I felt that was the case all along.Aleena is doing incredibly well today – she had her life saved by one of the most caring and compassionate people I’ve ever met – ever – in the world of politics.  When she looks back on a career filled with a long list of accomplishments – from serving in Cabinet to being the Leader of the Official Opposition – I know that Heather will be able to remember Aleena and the impact she had on her life as being one of the most satisfying and rewarding moments.

I’m sad to see Heather leave the world of politics.  She’s one of a kind – one of the few that go into the office everyday to try and make the world a better place for those around them.  She’s always been honest, truthful, kind, caring, and compassionate.  And she saved the life of a little girl.

0b6291d2079211e3943422000a9f1416_7The Alberta Legislature is better for having Heather serve amongst its storied halls, and the people of Alberta are better for having her represent them for so very long.  Personally, I’m a better person for knowing her, and I’m proud to call her Friend.

Godspeed, Heather Forsyth.  Enjoy your retirement, it’s well deserved.  Thank you for your service and your love for our kids battling rare diseases.  We will be sure to send you updates on Aleena as she makes her way through school, as she gets her first job, as she walks down the aisle on her wedding day.  Thank you for your kindness, for your love.  We will forever be indebted to you for that, and more.

Alberta will help fund critical treatment for young St. Albert girl

BY SARAH O’DONNELL, EDMONTON JOURNAL AUGUST 13, 2013 7:34 AM

Alberta will help fund critical treatment for young St. Albert girl

Aleena Sadownyk has a rare enzyme deficiency called MPS VI that causes buildup of cellular waste in their body. They need a synthetic form of the enzyme to be injected each week.

Photograph by: Supplied , Edmonton Journal

EDMONTON –  St. Albert father Dane Sadownyk picked up his three-year-old daughter Aleena and “just hugged her” Monday morning when the family learned the Alberta government will fund a crucial treatment for her rare medical condition.

“It was an extremely emotional moment,” Sadownyk said. “I was so elated. It felt like I could come up for a breath of air. That’s what it felt like, that I can breathe again.”

Aleena’s family and their supporters have been lobbying Alberta Health for a month to approve treatment for Maroteaux-Lamy Syndrome, a rare disease that means she lacks glycosaminoglycan, an enzyme that helps break down cellular waste.

Instead, the waste builds up, restricting movements, damaging organs and clouding eyesight, among other serious health complications. Without treatment, sufferers see their life expectancy cut short. Naglazyme, a synthetic enzyme approved in the U.S. but not Canada, can help break down that cellular buildup.

Though not a cure, weekly infusions could help prevent Aleena’s symptoms from getting worse, the family’s supporters say. Four other provinces have agreed to fund the treatment for seven children with the syndrome — also known as mucopolysaccharidosis type VI or MPS VI — and Aleena’s family was pushing Alberta to quickly approve the expensive but critical treatment, which is expected to initially cost about $300,000 a year.

Sadownyk, who was in Connecticut Monday with his family attending a conference on MPS VI when they heard the news, said they are feeling immense relief. Aleena was diagnosed with MPS VI in April.

“Today is definitely a day we are joyful for her and look forward to the future,” he said.

Andrew McFayden, director of the Isaac Foundation, went through a similar struggle in Ontario when his son Isaac was diagnosed with MPS VI as a toddler and was the first to receive treatment in Canada. He stepped in to assist the Sadownyks with their case and said he shared their feeling of elation Monday.

But McFayden also said the happiness is mixed with frustration that it took so long and that the family’s supporters felt they had to mount a public campaign with the support of MLAs such as Wildrose health critic Heather Forsyth, after the family’s original funding application through the Alberta Rare Disease Program was denied.

“We’ve gone through this numerous times,” McFayden said. “To me, that’s a big problem there still hasn’t been a process put in place by Health Canada for provinces to deal with funding these rare diseases.”

Health Minister Fred Horne said Monday he signed off on the funding for Aleena’s treatment through Alberta’s Short Term Evaluative Drug Therapy program, instead of the Rare Disease program, because Aleena’s case involves a drug not licensed for sale in Canada.

Horne said he weighed several factors, including the clinical evidence, the rarity of the disease, affordability of the drug and the best interest of the patient. Public pressure was not one of those factors, he said.

“I think Albertans would expect their minister and government to make these decisions based on evidence and looking at each case individually,” Horne said. “I’m pleased it’s able to be a positive outcome in this particular case. But these kinds of situations are becoming more common in Canada and it’s because we have more drugs coming out every day and more and more of these drugs are geared to rare diseases.”

Horne said he plans to talk about the need for an orphan drug program — a term used to describe medications for rare diseases — with other provincial health ministers and federal Health Minister Rona Ambrose.

“It’s an issue where we really need to collaborate,” Horne said. “There are only going to be more of these situations in the future.”

Forsyth, MLA for Calgary-Fish Creek, said Alberta Health must work to make the provincial system easier to navigate for families who suddenly find themselves seeking help with a rare condition. “The whole thing is just convoluted,” said Forsyth, who said she was overwhelmed to hear Aleena will receive treatment. “I think they have to simplify things and make it easier for the public to understand.”

NDP health critic Dave Eggen said Alberta Health also needs to speed up the process. “I’m glad something moved,” the Edmonton-Calder MLA said. “But in the future I don’t want to see people’s health compromised by being run through the wringer again.”

With Aleena’s funding approved, the Sadownyks’ next learning curve will be tied to her treatment.

“This is something that is new territory for us,” her father said. “We’ll learn.”

sodonnell@edmontonjournal.com

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© Copyright (c) The Edmonton Journal

Aleena Sadownyk will have treatment covered by Alberta government

Little girl with rare disease had been rejected for funding previously

 | August 12, 2013

Aleena Sadowynk

The Alberta government has agreed to pay for the treatment of Aleena Sadownyk, who suffers from a rare disease.

After a month of impassioned pleas and lobbying the provincial government, Alberta Health will fund the necessary medical treatment for Aleena Sadownyk – a St. Albert girl with a rare medical condition.

Three-year-old Sadownyk has Maroteaux-Lamy Syndrome, a rare disease that causes cellular waste to build up in her joints and around her heart, restricting movements and damaging organs. A drug called Naglazyme will help, but while it is used in the United Sates, it has not been approved in Canada.

Early estimates indicated it could cost anywhere from $300,000 to $1 million a year to administer the drug, which Aleena Sadownyk would need to help fight the disease. Sadownyk’s parents had been pushing the government to pay for the treatment, along with dozens of others including opposition parties who joined the fight.

On Monday morning, Sadownyk’s father Dane said that the province had agreed to fund the treatment.

“The past few weeks have been very difficult for us, but we can now focus on improving Aleena’s well-being and look forward to her having a bright future ahead,” he said in a statement released by the Isaac Foundation – a patient advocacy group.

Aleena Sadownyk had been denied funding originally through the Alberta Rare Diseases Funding program back in July. During the last two weeks, all of Alberta’s opposition parties called on Health Minister Fred Horne to authorize the treatment.

“It is with tremendous joy that I thank all those who pushed so hard for the government to approve this life-saving treatment for Aleena,” said Wildrose health critic Heather Forsyth in a statement  Monday morning.

“I hope that this painful ordeal for the Sadownyk family will lead to better approval processes for rare disease treatment in our province. We owe it to all Albertans to make sure that their health care system is there for them when rare diseases strike and extremely expensive treatments are their only hope.”

A spokesman with Alberta Health confirmed late Monday that the province will be paying for the drug to treat Sadownyk. The province was waiting for a clinical review to be completed on the benefits of Naglazyme before agreeing to fund it, said John Muir.

“We’ve come back now and carefully considered the circumstances and looked at that clinical review and we will now be funding Naglazyme for this individual,” he said. “We don’t want to be in a situation where we’re rushing any type of medical review on it. We want to make sure patient safety is put first and foremost and ensure it’s the best option for any individual.”

It has been reported that nine children in Canada are currently afflicted by Maroteaux-Lamy Syndrome. The cost of covering the Nagalyzme for Aleena Sadownyk will be around $300,000 a year.

 

Aleena Sadownyk Treatment: Alberta To Pay For Drugs For 3-Year-Old With Rare Enzyme Deficiency

CP  |  By Dean Bennett, The Canadian PressPosted: 08/12/2013 12:28 pm EDT  |  Updated: 08/12/2013 5:31 pm EDT

Aleena Sadownyk Treatment
 

EDMONTON – The family of a three-year-old Alberta girl learned Monday she will receive a potentially life-saving drug for a disease that is causing cellular waste to build up in her joints and around her heart.

The province announced it will fund enzyme replacement therapy for Aleena Sadownyk of St. Albert, just outside Edmonton.

“It was very emotional,” Aleena’s father, Dane Sadownyk, said in an interview. “The first thing I did was pick up my daughter and give her a huge hug.

“It’s been a challenging and a tough road.”

A panel of medical experts with Alberta Health Services made the decision to fund the drug Naglazyme for Aleena.

Health Minister Fred Horne did not intervene in the decision, saying it needed to be made for medical reasons alone, but agreed it was a good day for the Sadownyk family.

“Obviously for the family it’s a positive outcome,” said Horne.

“These decisions are difficult, and we face more and more of them all the time in Canada as we have more drugs becoming available to treat rare … diseases.”

The drug costs $300,000 or more per year for children, and because the dosage is tied to weight, can rise to $1 million a year for adults. Those on it are on it for life as the drug does not cure the illness, but simply stops it from worsening.

The Sadownyks had been working with the province since the spring, after Aleena was diagnosed with Maroteaux-Lamy syndrome, also known as MPS VI.

MPS VI patients lack the enzyme in blood that breaks down cellular waste. The waste then accumulates in the bones, tissues, and organs, leading to stiffened joints, heart and airway blockages, and potential death.

Dane said he and his wife, Laura, noticed something was wrong with Aleena — their middle child of three children — when she had trouble raising her arms, touching her shoulder or making a fist.

“We initially just thought she had arthritis because it does run in our family,” he said.

As they learned more about the disease they got in touch with Andrew McFadyen, who advocates for families dealing with MPS VI, to get funding for Naglazyme, which acts as an artificial enzyme to break down the cellular waste.

The drug is not approved yet for use in Canada, although it is in other countries such as the United States. It is permitted in special cases in Canada and is paid for in B.C., Saskatchewan, Ontario, and Quebec.

Of the nine children in Canada with the illness, Aleena was the only one not getting the drug prior to Monday, said McFadyen.

Aleena was initially denied funding for the treatment under the Alberta Rare Diseases Funding Program, but was approved Monday under the Short Term Exceptional Drug Therapy program, which provides the treatment for six months.

Horne said it’s up to the doctors to decide how and when the drug is administered after that.

“The medical experts will make decisions around how the drug is made available, how the monitoring takes places, and with respect to the ongoing coverage,” he said.

The parties had been quietly working on Aleena’s case for weeks when McFadyen, with the help of Heather Forsyth, health critic for official opposition Wildrose party, went public 10 days ago with a plea to Horne to intervene.

“I was hitting roadblock after roadblock (along with) misinformation and a lack of knowledge about the process,” said McFadyen.

“We couldn’t just leave it in the hands of the minister’s office to work through quietly. So that’s why we went public.”

Aleena’s plight took off on social media and the opposition parties, even St. Albert government member Stephen Khan, publicly pushed for an expedited decision.

Forsyth said she was overwhelmed Monday.

“The prognosis wasn’t good if she didn’t get it,” said Forsyth. “We’ve just given this little girl a whole new lease on life, and jeepers why didn’t the government do something about this (sooner).”

Horne said the criticism that the government moved slowly is unfair. He said those who had to make the decision were dealing with an unapproved drug and had to make sure it was right for this patient.

“I think the decision was made on a timely basis,” he said.

 

ERT – Proof of Effectiveness From Around The World

4d5f8030f60e11e2ad2b22000ae80c6b_7The 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)

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

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>

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

a picture– 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.

Toddler's family seeks relief from rare disease

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.

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.”

Toddler’s family seeks relief from rare disease

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.

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.”

St. Albert MLA urges quick approval for child’s medical treatment

August 6, 2013. 3:59 pm • Section: Capital NotebookUncategorized

Posted by:
Sarah O’Donnell

St. Albert’s Progressive Conservative MLA said Tuesday Alberta health officials should move quickly fund treatment for a three-year-old girl suffering from a rare medical condition.

Aleena Sadownyk’s family shared her story, and their push to get the provincial government to fund the expensive treatment that could keep the symptoms of Maroteaux-Lamy Syndrome at bay, last week. My Journal colleague Jodie Sinnema explained how Aleena is missing a crucial enzyme called glycosaminoglycan, which breaks down cellular waste in her body. Instead, the waste builds up, “restricting her movements, clouding her eyesight and damaging her organs. Without treatment, her life expectancy will dwindle.”

The St. Albert family has asked the province to approve funding for a drug called Naglazyme, which should help treat the condition.

On his Facebook page Tuesday, St. Albert MLA Stephen Khan said Alberta Health Services should approve funding through Alberta’s short term exceptional drug therapy program.

“As the MLA for St. Albert, I fully endorse the Sadownyk’s request for funding support from AHS,” Khan said. “My office and I have been advocating and working with the Minister of Health’s office on this issue since early July, when we first learned of the Sadownyk family’s situation.

“While I respect there is a process to follow for the Short Term Exceptional Drug Therapy Program review, I urge AHS to move quickly in rendering a positive decision for the Sadownyk family. Every day is an eternity for this young family, all the while knowing that a treatment for this debilitating disease is at hand. Currently four other provinces — Quebec, Ontario, Saskatchewan and British Columbia — have all made exceptions for families in need of this specific treatment.”

With his comments, Khan joins Wildrose health critic Heather Forsyth, MLA for Calgary-Fish Creek, who called on Health minister Fred Horne to immediately approve the funding for Aleena last Friday.

Alberta Health officials said Tuesday a clinical review by medical experts is underway. One of the issues is that Health Canada has yet to approve sale of the drug in Canada. “We’re not going to get involved to pressure them to rush that clinical review. We anticipate a response soon,” Alberta Health spokesman John Muir said Tuesday.

Aleena Sadownyk, Alberta Girl With Rare Disease, Awaits Word On Treatment Funding

Aleena Sadownyk, Alberta Girl With Rare Disease, Awaits Word On Treatment Funding

CP  |  By Dean Bennett, The Canadian PressPosted: 08/06/2013 5:19 pm EDT  |  Updated: 08/06/2013 9:31 pm EDT

Aleena Sadownyk

EDMONTON – A government member of the Alberta legislature added his voice Tuesday to the demand for immediate action to save a young girl wasting away due to a rare enzyme deficiency.

Stephen Khan, in a message posted on Facebook, wants Alberta Health Services to fast-track a decision on whether it will fund treatment for the disorder, which causes cellular waste to build up in the body. Khan is the Progressive Conservative member for St. Albert, where three-year-old Aleena Sadownyk lives.

“I urge AHS to move quickly in rendering a positive decision for the Sadownyk family,” wrote Khan.

“Every day is an eternity for this young family, all the while knowing that a treatment for this debilitating disease is at hand.”

Sadownyk has cellular waste building up in her joints and around her heart. A drug called Naglazyme can arrest the build up, but can’t do much for the damage already done.

The drug costs at least $300,000 a year to administer and has yet to be approved for use in Canada. It has been approved in numerous other countries including the United States.

Seven other Canadian children are receiving the synthetic enzyme through special exemptions funded by provincial governments in B.C., Ontario, Quebec, and Saskatchewan.

Khan said he has been working with health officials for a month trying to get the drug approved under the Short Term Exceptional Drug Therapy program.

Under the short-term program, the enzyme replacement would be provided for six months.

Health Department spokesman John Muir said Tuesday that an arm’s-length panel of medical experts is looking at the application.

He said the panel wants to move carefully given the drug has not been approved for use in Canada.

“We don’t want to be in a position where anyone is rushing a clinical review. We want to ensure that this drug is most appropriate for an individual and it’s done on a case-by-case basis,” said Muir.

He said the government received the request in mid-July and that the reviews generally take a few weeks to complete.

He said about 1,000 applications are made a year under the program and that 94 per cent are approved.

Sadownyk has already been denied funding for the treatment under the Alberta Rare Diseases Funding Program.

The Wildrose party wrote to Health Minister Fred Horne last week, criticizing the delays, urging he intervene personally to OK the treatment for Sadownyk.

“There have been major barriers thrown up in accessing this drug for Aleena, and each day of delay provides another opportunity for the irreversible symptoms of the disease to progress,” Wildrose critic Heather Forsyth said in a correspondence to Horne.

Muir said it would be improper for Horne to intervene.

“You don’t want to make it about politics at all. You want to leave those medical decisions to clinicians who have that expertise,” he said.

He said the Sadownyk family was denied funding under the rare diseases program because the drug didn’t qualify as it had not received general market approval through Health Canada.

The disease is called MPS VI, or Maroteaux-Lamy syndrome. Patients lack the enzyme in blood that breaks down cellular waste. The waste then accumulates in the bones, tissues, and organs, leading to stiffened joints, heart and airway blockages, and potential death.

Nine children have the disease in Canada.