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Stop This Train – An Update On Isaac

Stop this train.

I want to get off and go home again.

I can’t take the speed it’s moving in.

I know I can’t

But honestly, won’t someone stop this train.

~ John Mayer

Hey Everyone,

I’m a tired warrior. Exhausted, really. Over the past 10 years, I’ve been alongside Isaac as he fights his disease as bravely as anyone I’ve ever known.

At the same time, we’ve been working relentlessly to find a cure for his condition, and all other types of MPS at the same time. We’ve funded research projects around the world – some that are working out, some that haven’t. We have 3 gene therapy projects running concurrently, one that is imminently headed to clinical trial in humans.

We’ve fought for and brought his million dollar treatment here to Canada, and fought the government to pay for it. Along the way, we’ve fought alongside other families as we’ve looked for equal access to the health care system for them.

We’ve taken on, and won, battles against 3 different governments here in Ontario, two governments in Saskatchewan, two in Alberta, one in New Brunswick. We’ve even taken on the federal government itself.

We’ve never been unsuccessful getting access to treatments for kids suffering from rare diseases in this country. Ever.

I’ve traveled to many different countries and have presented the story of our kids everywhere I’ve been. Last year alone saw me on the road for 220 days fighting for our kids; fighting for our families. I’ve laughed with you all, and cried with you even more.

Each and every time I’m with people, I’m proud to say the I do what I do to honour my son and to honour the promise that I made to him to protect him from the world and keep him safe. I made that promise to him one week before he was diagnosed with MPS, and I felt like an incredible failure immediately. But I’ve been trying to make that up to him since day one, and it’s what pushes me to find a cure for him and all the other kids and families that have to deal with what we deal with every day.

A short while ago, I had an MPS mom remark to me that I don’t share much about Isaac anymore.  And she’s right, I don’t.  Not because I don’t want to, but because I’m still having a tough time dealing with things.  I always will, but I feel like I have to be strong for every other family out there waging this same war.

Which makes what I’m about to share with you that much more difficult. Sadly, I feel like we’re heading right back to where we started because Isaac will need a repeat of his spinal cord decompression surgery ASAP.

For those of you who have been with us since the start, Isaac had this surgery done when he was 20 months old, only a few months after his initial diagnosis. With MPS, the cellular waste that isn’t broken down by the enzymes in his blood end up storing in his bones, tissues, organs, and muscles. Essentially, every part of the body is affected, which is why his expensive treatment is so incredibly important to him.

It gives him the lifeboat he needs until we can find a cure.

Sometimes, patients suffering from MPS will see that buildup occur in the upper neck/spine area and they will need this risky surgery performed at some point in their lives. Usually this takes place later in life, but Isaac’s compression was so severe when he was diagnosed that it needed to be addressed immediately. In fact, it was so severe that his brain stem was compressed and was forming the letter “S” and any slight bump could have had catastrophic consequences.  Things were so bad at that time that they needed to take a diamond drill bit and route out a portion of his C-1 vertebrae.

We did the surgery under the careful hands of Dr. Cengiz Karsli and Dr. Rutka at Sick Kids, and both will be looking after things for us again.  At the time, we were young parents who were terrified about what was happening to our son, our lives, our family.

And we’re right back to where we started and we’re just as scared. Just as terrified.

Only this time, we’re going into things that much more exhausted.

I’ve hesitated writing this post – we found out the news last Tuesday while at the Neurosurgeon’s appointment in Toronto. I’ve hesitated because I’ve been fighting battles with other families for so long and I feel my role is to be the strong one out there. Someone that parents can come to when they need help or advice. Someone to turn to when they need a bit of Hope.

And I’ve been afraid to share my own vulnerabilities with those very families.

I’ve been finding myself saying the very things that other families and other parents have been saying to me over the past while. “I don’t think I can do this” and “I’m not strong enough to go through this.”

And I’m telling myself exactly what I’ve told you all at some point. “Yes you are. And you can do this, because you have to.”

I’m a Dad too. And I’m coming to realize that we’ve been able to help those families who have been shattered and scared because we know what it’s like to live this life. For 10 years, I’ve laid awake listening to be sure my son is still breathing. For 10 years, I’ve lived with the fear of losing him. For 10 years, we’ve been living in the now because we have no idea what the tomorrow looks like. For 10 years, we’ve been scared.

And so, here we are.  Knocked down once again but know we need to get back up off the mat and continue to fight.  And we’ll do that, because we have to.

The news last week didn’t change anything for us. It just brought it all back to the forefront and forced us to focus on our own battle once again. And we’ll need everyone we’ve been fighting alongside over the past decade alongside us this time.

And I know you’ll be there.

I’ll update more as we have information. For now, thanks for being along with us through it all.

With Love,
A.

Everything always works out in the end.  And if it doesn’t, it’s not the end.

~Stuart Mclean

Foundation-Poster

LOCAL CHARITY CELEBRATES 10 YEAR ANNIVERSARY

THE ISAAC FOUNDATION CONTINUES TO MAKE SIGNIFICANT GAINS IN SEARCH FOR CURE FOR MPS DISEASES WORLDWIDE

On February 21, 2016, The Isaac Foundation will be celebrating its 10th anniversary. The Campbellford, Ontario charity was started to fund research aimed at finding a cure for MPS VI, a rare, progressive, and devastating disease that a local child Isaac McFadyen was diagnosed with in 2005. Since that time, The Isaac Foundation has donated close to $1 million to research projects, with $175,000 being donated in the last year alone. The charity has grown considerably and supports children and families throughout Canada and around the world as they cope with the new reality a diagnosis of MPS brings.

“We’re incredibly proud of the things we’ve been able to accomplish over the past decade,” said Andrew McFadyen, Executive Director of The Isaac Foundation. “Obviously, finding a cure is paramount for us and that drive and focus to help these children will never diminish. But we’re also lucky to have helped children across the country receive the life-saving treatments they desperately need while we search for that cure.”

The Isaac Foundation has funded research projects located in Australia, the United States, and Italy, projects that have led to advances in the treatment and outlook of MPS. “We’ve had some hits and some misses over the years, but we’ve been fortunate to play a role in two exciting projects going to clinical trial. That in itself is incredible, but the fact that these projects will have a direct impact on improving the quality of life for patients suffering from MPS is still unbelievable to us. If you asked us 10 years ago if we thought this is where our organization would be, I don’t think we could have hoped for much more.”

One of the projects The Isaac Foundation helped start is a gene therapy approach to treating MPS VI. The project goes into human clinical trials in 2016 and could prove to be a cure for MPS VI, the form of the disease that young Isaac McFadyen suffers from. “We’ve been very careful not to throw around the word ‘cure’ with respect to this research, but we firmly believe that it will prove to be the answer we’ve been looking for all of these years. We’re lucky to have been involved since the very beginning, and we’ve been directing most of our research funds to this project for some time now. If the trials run as well as we expect it will, it won’t be long until we see the hard work behind this research come to market for our kids and patients. It’s incredibly exciting.”

The organization also has been administering a fund for MPS II research for the past number of years and has 3 gene therapy projects for MPS II underway. Last month, the fund provided another $62,000 grant to one of those gene therapy projects, research that looks very promising and is moving at a rapid pace.

“When we started The Isaac Foundation, we wanted to ensure that money that came into our organization was turned around quickly and sent back out to researchers as soon as possible. We also wanted to ensure we were responsible with the funds people donated and we’re proud to note that almost 99% of all money donated goes directly to our charitable program aimed at finding a cure for MPS. Last year, we hit the 100% mark and we expect to do the same this year. There aren’t many charities in this country that can meet those goals it’s means a lot to us that we can.”

With respect to advocacy and patient support, the organization has helped pave the way for patients across Canada to receive life-prolonging enzyme replacement treatments as the search for a cure continues. “These are incredibly expensive treatments, sometimes upwards of $1 million dollars per year, per patient. But the impact treatment has on the lives of patients is dramatic, and a price should never be placed on the life of a child. The help we’ve been able to provide these families suffering from MPS II, IVA, and VI is probably something that I’m most proud of in my life.”

The organization has garnered the attention and support of many big names throughout its 10-year history, from International music star John Mayer, to retired MLB star Roy Halladay, to Canadian icons The Tragically Hip, Ron Sexsmith, Sarah Harmer, and Danny Michel.

To celebrate their anniversary, Canadian graphic artist Andrew Kolb (www.kolbisneat.com) designed artwork that sought to capture the essence of The Isaac Foundation in one piece. The work features a smiling sun rising over a map tracing a long and winding route. At the end of the route is a large ‘X’ and the tag line “Hope Is Here”. On the horizon, kids and adults rush toward each other to join hands. One of the kids is in a wheelchair.

“It’s beautiful,” says McFadyen. “It sums up perfectly what we’ve been about and where we are going. Hope definitely is here, and I’m glad we’ve arrived there together with all the families we’ve been fortunate to help along the way.”

# # #

The 10th Anniversary artwork can be viewed online at http://www.theisaacfoundation.com/anniversary.jpg

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

10thAnniversaryPoster_Web

MINISTER ORDERS REVIEW OF DECISION TO DENY LIFE-SAVING DRUG

Treatment Required Immediately For Ailing New Brunswick Boy; Family Elated With News

“It doesn’t feel real.  We have hope again for our son.” – Carolle Mazzerole, Morgan’s Mom

The Minister of Health in New Brunswick has ordered a full review of his recent decision to deny access to a life-saving medication to 10-year-old Morgan Doucet.  Morgan suffers from MPS IVA (Morquio Syndrome) and requires the life-saving treatment immediately in order to halt further progression of his devastating disease.  The family was informed in October that Morgan’s application for the life-saving drug was denied and immediately asked how they could appeal the decision.  Health Minister Victor Boudreau informed the Canadian Press this evening of the new review.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, was happy with the news.  “We are very pleased with this development – it’s incredible news for the family.  For Morgan, I truly hope that this review takes a serious look at the vast amount of available evidence that exists both in this country and internationally, and I’m calling on the Minister to ensure the review is fair, transparent, and that the opinions and recommendations of multiple experts that deal with this disease and treatment first-hand are considered.”

McFadyen applauds the developments in New Brunswick and the willingness of Minister Boudreau to seek another opinion.  The Isaac Foundation has requested that a panel of 3 or 5 international and Canadian MPS experts conduct the review.  “This way, we have the opportunity for rigorous debate about the drug and its efficacy, and any decision made will always be a majority ruling.”

Jamie Myrah, Executive Director of the Canadian MPS Society, also hopes the new review will be thorough and transparent, free from Ministry bias and interference.  “The Canadian MPS Society also calls on the Minister to ensure a fair, objective, and transparent review of Morgan’s application for treatment.  It is our hope that the true experts – experts from around the world that deal with this disease, experts that treat this disease on a daily basis – have an opportunity to see the baseline data and weigh in on the merits of providing therapy for Morgan.  We also feel like it’s imperative that the reviewers are identified to our organizations, again so we can be sure the process is as transparent as possible.  We want to make certain they are using the true experts to look at this, unlike the initial review that took place.”

Morgan’s mother, Carolle Mazzerole, was elated by the developments.  “We’re thrilled.  It doesn’t feel real.  We have hope again for our son.  I pray that the new review is done quickly and is fair for our Morgan.  The last review took a year for us to receive a decision and Morgan can’t wait much longer.  I really want to say thank you to Minister Boudreau for revisiting this decision, and a sincere thanks to our MLA, Jake Stewart, for his incredible support for our family.”

  #

 For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com.  The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.

HEALTH MINISTER REFUSES TO HEAR EXPERT OPINION REGARDING DENIAL OF LIFE-SAVING MEDICATION FOR MORGAN DOUCET

Minister Refuses To Allow Renowned MPS Expert To Join Family Meeting; Doucet’s Family In Fredericton to Meet with Child And Youth Advocate Thursday

The family of ailing 10-year-old Morgan Doucet has cancelled a scheduled meeting with the Minister of Health in New Brunswick after the Minister refused to allow an internationally renowned expert to participate in the discussions. Doucet’s case has made headlines throughout the Province after the Minister of Health denied access to the life-saving drug that he requires. Morgan suffers from MPS IVA (Morquio Syndrome) and requires the life-saving treatment immediately in order to halt further progression of his devastating disease. Without access to therapy, Morgan faces a long and painful decline and death at a young age.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, has slammed the Minister for his refusal to meet with one of the world’s foremost experts in MPS and related diseases. “It’s unconscionable to me that Minister Boudreau would refuse to allow this expert to participate in the meeting he had scheduled with Morgan’s family. Unbelievable, really. He has denied Morgan access to the life-saving medication he needs, hasn’t allowed his family an avenue to appeal his ruling, and then refuses to hear from a true expert that deals with this disease on a daily basis – an expert who is internationally recognized as the best in his field. It leads me to believe that he’s afraid to be proven wrong on such an important issue, which is deplorable because a life hangs in the balance. Pride and politics should never play a role in the health and well being of a child, and it appears that’s exactly what is happening with Morgan Doucet with respect to the Minister’s steadfast refusal to hear more evidence on this matter.”

Doucet’s parents will be joined McFadyen at the provincial legislature in Fredericton on Thursday, December 17 to watch the morning legislative session and to meet with the province’s Child and Youth Advocate, Norman Bosse. PC Jake Stewart, the Doucets’ MLA, will join the family during that meeting.

Bosse has agreed to look into the process used to deny access to Morgan’s medication, a process that McFadyen says was destined to return a negative decision all along. He notes that after an initial application for treatment in October of 2014, the file was finally sent to Ontario for review by one individual alone, with no opportunity allowed for a rigorous discussion with the rest of the MPS Experts throughout Canada and Internationally –experts that deal with the disease on a daily basis and some of whom have seen first-hand how well other children receiving the treatment are doing.

“The Minister and the Ministry’s only comment on this case is that the drug didn’t get a positive review from the Common Drug Review (CDR). What he doesn’t say is that all drugs used to treat other MPS diseases have received negative CDR reviews and the Province pays for access to all. Right now, Morgan is the only MPS patient in the province who has been denied this coverage, and that’s disgraceful.”

Health Canada approved the treatment Morgan requires in July of 2014 and it has been recommended for use in patients by the Canadian Expert Opinion on Morquio Syndrome. In addition, the International Expert Opinion on Morquio Syndrome considers the treatment their gold standard of care for patients throughout the world. It is reimbursed for patients in Saskatchewan, Ontario, and Quebec.

In Canada, there are 33 patients receiving the treatment. According to McFadyen, all of them are doing exceptionally well. “These patients have seen dramatic improvements and have had their disease progression either halted or dramatically slowed down. Morgan deserves the same chance at life that those other patients have. This is Canada – where equal access to our health care system isn’t a privilege, it’s a right.It’s callous and cruel that the Minister of Health is forcing this child and this family to enter a palliative approach to his disease when we know he can be helped. We know he can be saved.”

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, says Doucet’s case has angered and galvanized the people of New Brunswick. Doucet was front-page news on Monday, and has been featured by numerous provincial and national media outlets since his treatment was denied. The Isaac Foundation’s online posts about Morgan have received over 100,000 visitors during that time, and hundreds of emails have been sent to the Premier and the Minister of Health supporting access for Morgan. McFadyen has posted an online form that the people can fill out and all messages get delivered instantly to the Minister’s and the Premier’s email inbox.

“People in New Brunswick and across Canada are understandably upset about this decision and they want their voices heard,” says Myrah.

McFadyen finishes, “We won’t accept the Minister making medical decisions and overruling and contradicting medical experts, clinicians, and researchers regarding best practice and treatment for Morgan Doucet. If he won’t look at the clinical expert opinion, the patient reported outcomes or the peer-reviewed published journals, then who will he listen to?”

Both The Isaac Foundation and The MPS Society are renewing their call for Health Minister Victor Boudreau and Premier Brian Gallant to take action, review and reverse their initial decision, and to ensure that treatment begins immediately for Morgan Doucet.

# # #

The Doucet Family and The Isaac Foundation will be available for interested media at the Legislature on Thursday, December 17. To schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.

 

BACKROUNDER  

While not a cure for Morquio Syndrome, the necessary Enzyme-Replacement Therapy (ERT) is 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 slows down or halts progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. Vimizim was approved by Health Canada in July 2014, and is currently being reimbursed for use by patients in Saskatchewan, Ontario, and Quebec. Recently, the National Institute for

 

Photo Source:  CBC.ca

Tentative Meeting Scheduled

Hi Everyone,

As promised, I’m back to provide another update.  After speaking with the Ministry this afternoon, the Minister has set aside some time to meet with The Isaac Foundation and Morgan’s parents.  Things are tentatively set for Thursday.  However, that meeting will only be taking place if the Minister agrees to have Dr. Paul Harmatz on the line from Oakland Children’s Hospital and if Morgan’s MLA, Jake Stewart, can join us.

The rationale behind having both Dr. Harmatz and Mr. Stewart join us is simple – Dr. Harmatz is a world renowned expert in the field of MPS and this treatment and it’s important for him to be on hand to answer any questions that might be raised in the meeting and to try to shed some clarity on what this treatment can and can’t do for patients.  If the Minister doesn’t want Dr. Harmatz present, it indicates to us that the Minister doesn’t want to seek information and advice from the people that are the best in their field.  If that’s the case, we see no reason to meet.  We want to hear from the best, and we are hopeful Minster Boudreau does as well.

And Jake Stewart?  Well, he’s been the support for this family since the very start of this process, someone who’s been kind and caring when they need it the most.  The compassion he’s shown for Morgan and his family has been wonderful, and his support during such a meeting will be important.

I’ve made the request to the Ministry and await their reply.  So, for now we have a tentative meeting.  Hopefully we can get things sorted out soon.

In the meantime, if you haven’t had the opportunity to send Premier Gallant or Minister Boudreau your thoughts on this situation, you can use the form below.  Your message will land directly in their in-boxes, and your voice can be added to those that are speaking out in support of Morgan, of the life-saving treatment he needs, and for doing what’s right in the Province of New Brunswick.

Thanks, as always, for your support.

A.

#Hope4Morgan – Send Premier Gallant and Minister Boudreau An Email!

Are you concerned with the recent decision by the Gallant Government to deny Morgan Doucet the life-saving treatment he needs?   We want the tens of thousands of people who have read Morgan’s story to have their voice heard.  Please take a moment to send your thoughts and concerns to Premier Gallant and to Minister Boudreau.  The form below will ensure your message will land directly in their in-box.  Please be respectful in your messages and thank you for your continued support as we work to ensure our kids get the help they need.

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Short Update – Talking With The Minister's Office Later Today

Hi Everyone,

Just wanted to let you all know a few things.  First off, thank you for the incredible support you are showing Morgan and his family.  Thus far, we’ve had over 80,000 people (more than 10% of the population of New Brunswick!) viewed Morgan’s story online, shared it with your family and friends, and commented – both online and via email – to the Minister and Premier.  Emails continue to pour in and your participation and efforts to have your voices heard is heartwarming and appreciated.

I spoke with the Minister’s Office today and we are set to talk a bit later this afternoon.  I will continue to push for a fair, open, and transparent review of Morgan’s file, a review conducted by the real experts that deal front line with this disease.  Anything less is unacceptable, cruel, and unfair for Morgan.  I’ll log online to update after that conversation takes place.

In the meantime, keep those comments rolling in, keep sharing Morgan’s story, and keep holding your government to account.

Till our next update…

 

Short Update – Talking With The Minister’s Office Later Today

Hi Everyone,

Just wanted to let you all know a few things.  First off, thank you for the incredible support you are showing Morgan and his family.  Thus far, we’ve had over 80,000 people (more than 10% of the population of New Brunswick!) viewed Morgan’s story online, shared it with your family and friends, and commented – both online and via email – to the Minister and Premier.  Emails continue to pour in and your participation and efforts to have your voices heard is heartwarming and appreciated.

I spoke with the Minister’s Office today and we are set to talk a bit later this afternoon.  I will continue to push for a fair, open, and transparent review of Morgan’s file, a review conducted by the real experts that deal front line with this disease.  Anything less is unacceptable, cruel, and unfair for Morgan.  I’ll log online to update after that conversation takes place.

In the meantime, keep those comments rolling in, keep sharing Morgan’s story, and keep holding your government to account.

Till our next update…

 

An Open Letter to Brian Gallant and Victor Boudreau

When I last visited with Victor Boudreau, it was concerning a little girl (Kamie Babineau) who lives near Moncton that needed a life-saving treatment that is very similar to the treatment that Morgan needs. In fact, the only difference is the enzyme that each of these kids need. It’s simple really – unbelievably simple. Kids suffering from MPS lack an enzyme in their blood that breaks down cellular waste. The cellular waste that doesn’t get broken down needs to find a place to go so it stores itself in the bones, tissues, organs, and muscles of our kids fighting the disease.

The treatment our kids receive to help fight off the ravages of MPS is a synthetic version of the enzyme they are missing in the blood. Basically, our kids are missing this enzyme and we are giving it back to them. The enzyme treatment breaks down all of the cellular waste that is building up in the body and helps patients lead a more normal and healthier life.

It really is that simple, and it’s why Minister Boudreau approved access to the drug Kamie needed within days of receiving her application.

When we met at that time, Mr. Boudreau told me that one of the main reasons that he was so passionate about the work we are doing was because of a quote that tops my website, and a quote I work to live my life by. It’s a quote by Margaret Mead that read “Never doubt that a small group of thoughtful, committed citizens can change the world; indeed, it’s the only thing that ever has.”

Mr. Boudreau told me he saw this on my website and it struck a cord with him. He told me that Premier Gallant often uses this quote during cabinet meetings, telling his team that they have the opportunity to be that small group of committed citizens, and to always work to change the world in everything they do.

When he saw this on my website, Minister Boudreau told us that he went directly to the Premier with Kamie’s application for treatment and told him they needed to do something, and fast.

And it was fast – days. In a matter of days, Kamie’s application for treatment was approved. A short while later, she began her life-saving treatments and is thriving today. Thriving because the treatment works. Her life had been saved.

During that same meeting, I told Mr. Boudreau about Morgan Doucet. I told him there was another boy needing a very similar treatment and that he needed it fast. The treatment had been approved by Health Canada 8 months earlier and the Ministry of Health wouldn’t accept the physician’s application for reimbursement for Morgan. At that time, the Ministry said they “didn’t know what to do with the file.” Mr. Boudreau told me he would take the application and he would get back to me very quickly.
In reality, The Ministry of Health DID know what to do with Morgan’s file because they have been part of a larger collective in the country – a collective of all of the pharmaceutical managers throughout the country who made an agreement that they wouldn’t approve payment for the drug Morgan needed. Why? Cost. It’s an expensive drug and the Provinces wanted to work together to bring the cost of this treatment down. They discussed the drug together, made a commitment that “nobody would be the first to approve” and began work to bring the cost of the drug down.

In theory, brining the cost of pharmaceuticals down in this country is a good thing. I’m all for getting the best price on things, especially expensive things. But not at the expense of the people who have to suffer while the bureaucrats play hardball with the pharmaceutical industry.

I’ve had conversations with different provinces about this issue over the course of the past 2 years. They stand by their approach to this drug, even when told that it’s putting our kids in the middle of a battle that they want no part of, even when told that our kids are dying while they try to get the best deal possible for their respective Provinces.

Unfortunately for Minister Boudreau, he didn’t discover this until he took Morgan’s application and promised to get back to us quickly. Days turned into weeks, which turned into months. A battery of tests were ordered on Morgan and the Province worked to delay a decision on Morgan’s treatment by adding new tests after the original batches were complete.

When a decision on Morgans treatment was deemed “imminent”, the Ministry of Health ordered Morgan to undergo a sleep study, knowing full well that the New Brunswick didn’t have the capabilities to conduct such tests in the Province and that Morgan would have to fly to the Hospital for Sick Children in Toronto to have the tests done.

This is when I started to move from frustrated to upset. Why? Because the Ministry was well aware that the test Morgan required would take months to complete due to a wait list at Sick Kids in Toronto. Add to that the stress and burden placed on Morgan and his family by leaving their home, boarding an airplane, and finding their way around a huge city (something they have never done before).

But it wasn’t the delay which was incredulous to me, it was the test that was ordered with the clear intention of delaying things further for Morgan. They wanted a sleep study, even though Morgan has never had difficulty with his sleeping. Ever.

With some perseverance, we were able to remove the sleep study from the tests the Ministry ordered for Morgan. After that removal, the Province denied Morgan’s application. I immediately asked how we could appeal the ruling. I asked the Ministry, the Minister, and the Premier in multiple emails and phone calls. I was ignored each and every time.

Which brings us to where we are today. I relayed all of this to Minister Boudreau when we met yesterday and he told me he would take another look at Morgan’s file. He told me he would look at things after I told him the pharmaceutical managers have agreed to work together to deny access for our kids as they wage war on Big Pharma. He told me he would look into things after I told him the review process was flawed and set up to fail from the very beginning. He told me this after I told him that Saskatchewan saw through this flawed system and took it upon themselves to initiate their own proper, thorough, and transparent review. A review free of bias from Ministry officials. A review meant to provide REAL feedback.

During that meeting, however, I couldn’t help but wonder where the Minister’s gusto for being part of the small group of citizens working to change the world together went. If this wasn’t the time to step forward and take a stand, make a statement on behalf of a young child, then when? We know this treatment works. We know because we have 33 Canadian children receiving the treatment throughout the country. We know because the International and Canadian experts have all said it should be used in our kids as soon as possible.

And I know because I see it working each and every day, with every patient I visit, with every drop of drug that gets dripped into their body. Their lives are changed – saved – and we can see it in everything they do.

Saskatchewan took the lead and moved away from this review process. They did this because it is the right thing to do by patients.

So I again implore Victor Boudreau and Premier Brian Gallant to be part of that small group of citizens working to change the world. Take it upon yourselves to be the true leaders your Province needs you to be. I’m not asking for immediate access to treatment, just the commitment to a new, thorough, and transparent review of Morgan’s file. A review completed by the real experts out there, free of bias and bureaucratic interference.

Morgan deserves that opportunity, and it’s incumbent on your offices to be sure that he gets that chance. Please be part of the change you purport to stand for and don’t hide behind the excuses being handed to you by the bureaucrats.  The government has nothing to lose by ordering a new review, but Morgan has EVERYTHING to lose if they don’t.

This shouldn’t be about cost, about the Provinces of Canada vs. Big Pharma. It should be about Morgan, and I ask you humbly to refocus things on that little boy. In the end, it WILL change his world.

I stand by my offer to bring in Dr. Paul Harmatz to talk with you about this disease, this treatment, and this case.  He’s the best in the world when it comes to this disease.  If your government is open, transparent, and about the change you want to see in this world, a good first step toward showing this would be to talk with Dr. Harmatz and get the real information you need.

I’ll be in Fredericton this week and hope we can discuss while I’m there.

 

 

 

 

Mom pleads with Liberal government to allow potentially life-saving treatment for child

CHRIS MORRIS Legislature Bureau

December 13, 2015

Carolle Mazerolle with her 10-year-old son Morgan Doucet. Doucet sufferers from a rare disease called Morquio Syndrome and is believed to be one of only 33 people in the country and the only known case in New Brunswick.

Photo: Jeremy Trevors/ Miramichi Leader

Carolle Mazerolle has been battling medical bureaucracies to get help for her son, Morgan, almost since he was born 10 years ago.

Now she has another fight on her hands, this time to get a new treatment that could finally help her beloved and brave little boy enjoy not only a pain-free life but also, possibly, a longer life.

“I want to give him this drug as quickly as possible,” Mazerolle said in an interview, sending a message directly to Health Minister Victor Boudreau.

“I want them (New Brunswick health officials) to make another decision on this. Morgan needs this right away. He gets worse by the minute and every moment he waits, his body is deteriorating. I don’t want to wait another year, not even another day. Please, just make the right decision.”

Mazerolle fights tears as she speaks. It has been a long, difficult time for Carolle, her partner Glen Doucet, Morgan, and other members of the family in Baie-Sainte-Anne as they cope with Morgan’s rare genetic condition called Morquio Syndrome.

The family finally felt hope last year after it was announced that Health Canada had approved a treatment, an enzyme replacement therapy, that has been shown to slow down or halt progression of the disease in patients.

Morgan is the only known person in New Brunswick with Morquio Syndrome and Carolle and Glen quickly applied to have the treatment covered for their son. After about a year, their request was denied in October, and no avenue for appeal was offered.

It has been a roller-coaster ride for the family.

“We all were all so hopeful when the treatment was approved,” Carolle said. “We thought everything is going to fall into place and Morgan will have a better life and live a lot longer. Then they refused us. I still have trouble believing it”

The treatment is expensive.

Andrew McFadyen, executive director of The Isaac Foundation, an Ontario-based advocacy organization that specializes in diseases like the one afflicting Morgan, says the list price for the enzyme replacement drug is about $300,000 per year.

However, McFadyen said a lower cost likely could be negotiated with the drug developer once Morgen gets the go-ahead for treatment from the province.

McFadyen was in Fredericton last week to meet with Boudreau and apply pressure to give Morgan a second chance at the treatment. He did not get the reassurances he was seeking, although he said Boudreau did agree to look at the case.

“New Brunswick health officials are telling this family to enter a long-term palliative approach to care for their child instead of ensuring that disease progression is halted now,” he said.

“They are allowing him to die instead of taking leadership and doing the responsible and ethical thing by providing him with the help he needs. It’s shameful, disgraceful.”

The Health Department says it is bound by privacy rules and cannot speak about Morgan’s case. Jake Stewart, opposition Tory MLA for Southwest Miramichi-Bay-du-Vin, has raised the issue in the legislature, appealing to the Liberal government to put politics aside and “just help this child.”

Carolle, Glen and McFadyen are planning to travel to Fredericton on Wednesday to press their case to have the treatment approved. Morgan may be there as well, although it is not certain.

Carolle said she knew something was wrong with Morgan shortly after he was born, although most children with the syndrome do not have it confirmed until four or five years of age.

Her baby seemed in pain right from the start and she remembers spending the first four months of his life sleeping on her back, holding him.

Carolle kept telling doctors something was wrong and they kept telling her he was just a bit slow and would be fine. Finally, at 15 months of age, a doctor in Saint John took x-rays and discovered bones in Morgan’s neck were broken.

Now in Grade 5, Morgan is fully developed intellectually and socially, and has lots of friends. But he has never walked, can stand only for a few seconds holding onto something, has almost constant headaches and is often sick to his stomach.

The syndrome has deformed his body, but not his spirit, Carolle says.

“This is a small community and everyone knows him,” she said. “The other kids are all great with him.”

McFadyen said sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. The waste builds up in the bones, tissues, organs, and muscles and leads to such symptoms as heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.

He said the treatment being denied to Morgan dramatically slows or halts the disease, improves pulmonary function, walking ability, and gives patients the chance at a normal life.

There are 33 cases of Morquio in Canada. The treatment currently is funded in Saskatchewan, Ontario and Quebec.

Source: https://www.telegraphjournal.com/telegraph-journal/story/45053995/mom-pleads-with-liberal

LIFE-SAVING TREATMENT DENIED FOR AILING NEW BRUNSWICK CHILD

FOR IMMEDIATE RELEASE

PDF Version – www.theisaacfoundation.com/nbtreatment.pdf

Ministry of Health Denies Funding; Premier and Minister of Health Refuse to Allow Appeal; Treatment Already Being Funded in Saskatchewan, Ontario and Quebec

The New Brunswick Ministry of Health has denied funding for a life-saving treatment required by an ailing 10-year old Baie-Sainte-Anne child. Morgan Doucet suffers from MPS IVA (Morquio Syndrome) and requires the life-saving treatment immediately in order to halt further progression of his devastating disease. The Isaac Foundation and The Canadian Society for Mucopolysaccharide & Related Diseases (Canadian MPS Society) are calling on Health Minister Victor Boudreau and Premier Brian Gallant to take action, review and reverse the decision, and ensure that treatment begins immediately.

Without access to therapy, Morgan faces a long and painful decline and death at a young age. The Isaac Foundation met with Minister Boudreau on December 9, 2015 but the Minister declined to make any commitments regarding an appeal for Morgan. The family has publicly given permission for the Minister to comment on Morgan’s application and condition so that the Minister can be forthcoming with the public about why he isn’t allowing an appeal.

Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. This waste builds up in the bones, tissues, organs, and muscles of affected individuals and leads to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death. The treatment being denied to Morgan dramatically slows or halts the disease, improves pulmonary function, walking ability, and gives patients the chance at a normal life. It is currently being funded in Saskatchewan and for patients in Ontario and Quebec.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, is disappointed by the decision and is urging an immediate reversal to save the Morgan’s life. “The decision to deny this life-saving treatment for Morgan is outrageous, heartless, and cruel. This therapy has been recommended by a Canadian Expert Panel comprising of MPS experts from BC, Alberta, Saskatchewan, Ontario, and Quebec, and International Treatment Guidelines for Morquio Syndrome highlight this treatment as the gold standard of care for affected patients. To prevent this child from receiving the care he requires is needless and callous, and shows a reckless disregard for what our Canadian Health Care System purports to stand for.” He adds, “The Ministry is telling this family to enter a long-term palliative approach to care for their child instead of ensuring that disease progression is halted now. They are allowing him to die, instead of taking leadership and doing the responsible and ethical thing by providing him with the help he needs. It’s shamefully disgraceful.”

McFadyen is puzzled as to why access for treatment was denied, especially considering the weight of available evidence and Canadian and International expert guidelines urging it’s approval. In addition, the Province already provides access to patients suffering from every other form of MPS requiring the same kind of treatment. He expects it comes down to financial considerations due to the high cost of the drug, coupled with a recent negative recommendation from the Common Drug Review (CDR), that has clouded the judgment of decision makers at the Ministry.

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, notes that there were serious issues with the initial review that denied Morgan the treatment he needs.   “The initial review was done by a reviewer who has never used this treatment, using the flawed CDR report as the basis for his decision. This recently happened with three children in Saskatchewan. However, when the Minister realized the review for those files was flawed, he ordered a new review using real experts dealing with this disease and this treatment. He chose International experts to take a second look at the files and those experts came back with a decision to treat. We feel the Premier should also take a second look at Morgan’s file. We’re not asking for much – just a second look at this decision. Morgan’s life depends on it and I would hope the Premier and the Minister of Health would want to ensure they get things right.”

McFadyen also questions the process used by the New Brunswick Ministry of Health in making their decision, noting the family first requested access in October of 2014. He notes that in March of 2015, the file was finally sent to Ontario for review by one individual alone, with no opportunity allowed for a rigorous discussion with the rest of the MPS Experts throughout Canada –experts that deal with the disease on a daily basis and some of whom have seen first-hand how well other children receiving the treatment are doing. “The only thing stopping this child from beginning his treatment is the lack of funding by the Province. It’s incredulous to me that one individual gets to pick and choose who does or doesn’t get access to the life-saving treatment they need, especially considering the fact that a precedent has been set with other children in this province and in other provinces across the country receiving access to similar medications. One individual is making life-altering decisions on behalf of this Ministry and the people of New Brunswick, and it’s led to Morgan’s life being left in peril.”

Myrah adds, “We know this treatment works and the Province knows that ERT’s for MPS diseases are currently the only chance patients have of living longer, healthier lives. We recognize that the CDR gave this treatment a negative recommendation, but it did so for all the other MPS ERT’s as well; yet every province in the country – including New Brunswick – still ensures access for those patients. While ERT treatment for MPS is expensive, it is often just as expensive – if not more expensive – to not treat these kids. It simply doesn’t make sense to make these children suffer a slow and painful decline when we could stop or dramatically slow the progression of the disease with the treatment being requested.”

Both Myrah and McFadyen are hopeful that a review and a reversal of this decision can take place immediately, and they are urging the Health Minister and the Premier to take action now. Says McFadyen, “Brian Gallant’s government has the opportunity to do the right thing and ensure that Morgan receives the treatment he so desperately needs. It’s the role of government to protect and ensure fair and equitable access to Health Care for all people in New Brunswick, regardless of whether they are impacted by a rare disease or not. This family needs to concentrate on treating their children’s disease, not wading through a bureaucratic maze of paperwork and pinning their hopes on the appeals process or the recommendation of one person alone –a person who has never used this therapy or seen it’s effectiveness – at the Ministry of Health. We strongly call on this government to take action and save the life of this little boy now. He can’t afford to wait.”

# # #

For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.

 BACKROUNDER  

While not a cure for Morquio Syndrome, the necessary Enzyme-Replacement Therapy (ERT) is 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 slows down or halts progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. Vimizim was approved by Health Canada in July 2014, and is currently being reimbursed for use by patients in Saskatchewan, Ontario, and Quebec. Recently, the National Institute for Health and Care Excellence (NICE) recommended reimbursement for all 88 patients suffering from Morquio Syndrome throughout the UK.

Baie Sainte Anne Boy Needs Drug To Manage Rare Disease

December 9, 2015

A young Baie Sainte Anne boy’s plight made it to the floor of the provincial legislature this morning.

Morgan Doucet, 10, is currently the only person in New Brunswick suffering from morquio syndrome.

On Wednesday morning, Southwest Miramichi-Bay du Vin MLA Jake Stewart called on the province reverse its decision not to fund a Health Canada approved treatment for Doucet’s illness.

According to the U.S. National Library of Medicine, morquio syndrome is an inherited disease of metabolism in which the body is missing or doesn’t have enough of a substance needed to break down long chains of sugar molecules called glycosaminoglycans (formerly called mucopolysaccharides).

Symptoms include:

– Abnormal development of bones, including the spine
– Bell-shaped chest with ribs flared out at the bottom
– Coarse facial features
– Hypermobile joints
– Knock-knees
– Large head (macrocephaly)
-Short stature with a particularly short trunk
– Widely spaced teeth

While Stewart didn’t name the treatment in question, the only Health Canada approved drug for the disease is Vimizim, which was approved last year.

Annual treatment can cost upwards of $100,000.

Stewart said Doucet needs treatment.

“Recently access to this lifesaving medication for Morgan Doucet was denied by the minister of health and the department, even though it is the gold standard of care as recommended by the international treatment guidelines, has been recommended by the Canadian Expert Panel on Morquio Syndrome and has been prescribed by Morgan’s genetic specialist at the IWK ” Stewart said in the legislature.

He accused the government of overruling the experts and said there is no process for appeal.

Health Minister Victor Boudreau said he could not discuss the specifics of the case on the floor of the legislature, citing privacy rules, but said their is a process in place for situations like Doucet’s.

“Because we do not have the level of expertise on these rare diseases and rare drugs here in the province of New Brunswick we rely on the province of Ontario and this is a process that was put in place by the member for Rothesay, the former minister,” Boudreau said.

He explained that the file is sent to experts in Ontario and they get back to the New Brunswick government with a recommendation.
But Stewart wasn’t happy with that reply.

“The drug that Morgan requires is currently being funded by Saskatchewan, Ontario and Quebec and in most developed countries.  Morgan’s application was denied by a single reviewer from Ontario, a reviewer who no longer practices medicine and has never used this treatment,” Stewart said.

He also said Saskatchewan found flaws with the review process and with that reviewer. He said that province now deals directly with the experts on specific diseases.

“I ask the minister of health and the premier, will they show the same leadership and do the right thing by ordering a new review of Morgan’s file and provide immediate access to this life saving treatment while that review is taking place?” Stewart asked.

Boudreau said these are very tough situations.

“Everybody in this legislature has a heart and understands that these decisions are difficult to make,” Boudreau said.

“I can tell you that across the country, and in New Brunswick, some cases get approved, some cases don’t. We follow a process, Mr. Speaker. I am not an expert, and we don’t even have those experts within our department or within our province,” he added, saying the process was followed.

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