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 https://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.


Health minister under pressure to approve expensive drug for sick boy

CHRIS MORRIS Legislature Bureau

December 9, 2015

Andrew McFadyen, executive director of The Isaac Foundation, travelled from Ontario to Fredericton on Wednesday to lobby the Liberal government on behalf of Morgan Doucet, a 10-year-old boy from the Miramichi region, who has been denied treatment for a life threatening condition.

Photo: Chris Morris/Legislature Bureau

FREDERICTON • The New Brunswick Health Department is under pressure to reconsider the case of a little boy in northern New Brunswick who has been denied access to an expensive medical treatment that could improve and possibly lengthen his life.

Jake Stewart, opposition Tory MLA for Southwest Miramichi-Bay du Vin, raised the issue in the legislature on Wednesday, appealing to Health Minister Victor Boudreau to reconsider his department’s earlier decision to deny the child, Morgan Doucet of Baie-Sainte-Anne, a promising new treatment for the rare disease, Morquio Syndrome.

The treatment, which would cost about $300,000 a year for Morgan, has been approved by Health Canada but it was not given the go-ahead in New Brunswick following a review. The New Brunswick Health Department formally rejected the Doucet family’s application for help in October and has not provided an avenue for appeal.

Stewart said the family now has decided to allow their situation to become public. He said it is a heartbreaking case, made worse by what he described as a seemingly callous and bureaucratic response to the plight of a child in need.

“It is really simple what they (health officials) have to do: he is a 10-year-old boy; he is the only citizen of New Brunswick who has this disease; it is life-threatening – help him,” Stewart said in an interview.

“That is all we are asking. It’s not about politics – just help this little boy.”

Boudreau told the legislature that he cannot comment on the case specifically because of privacy issues. But he insisted he is not heartless.

“These are very difficult situations,” Boudreau said. “These are not easy files to deal with. I can assure you of that. Everybody in this legislature has a heart and understands that these decisions are difficult to make.

“Without talking about the case in particular, I can tell you that, across the country and in New Brunswick, some cases get approved and some cases do not. We follow a process.”

After question period on Wednesday, Boudreau met privately with Andrew McFadyen of the Ontario-based Isaac Foundation who travelled to Fredericton to press the minister on Morgan’s case.

“He made no commitments or promises,” McFadyen said of Boudreau. “He believes in the process currently in place but he will take a step back and look at this case and be in touch with me.

“I’ve been trying to talk with him about this decision since it was rendered in October and I have been stonewalled. It is encouraging but I had hoped for a bit more, at least a commitment to have a second review of this.”

The Health Department would neither confirm nor deny that the minister has agreed to take another look at the file, citing privacy concerns.

The process followed by the Health Department involved sending the file to an independent reviewer in Ontario who felt the best that could be expected from the costly treatment is “some slowing of the progression” of the disease.

McFadyen’s own son has a different form of Morquio Syndrome. It is believed there are only 33 cases in Canada, and Morgan Doucet is the only known case in New Brunswick.

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.

The treatment currently is funded in Saskatchewan, Ontario and Quebec.

Source: https://www.telegraphjournal.com/telegraph-journal/story/45005525/health-minister-under-pressure

Saying Goodbye Has Never Been More Difficult

Everything comes to an end in this world, but sometimes we wish we could have a bit more time before the special things in life do.  If we could, we’d make time to stand still for a short while, just long enough that we could pause and enjoy a special memory, a special person, a special moment, for just a little longer.  Today was one of those days.

Dr. Julian Raiman came into visit with Isaac this morning for one last time before he heads back home to the UK to continue his stellar career in his home country.  He dropped in for his last visit with Isaac, his last check-up, his last report taking, his last few moments caring for our son.  It was a difficult time for us as parents and our kids, and I didn’t want the visit to end because, when it did, our time together would officially be over.

Julian has been with Isaac and our family for over 10 years.  He has grieved with us as he’s given us bad news, cried with us as we’ve adjusted to new circumstances in life, and celebrated with us during health improvements and triumphs.  More importantly, he’s guided us along this journey – always at the forefront, always in the lead, and always assuring us that we still had Hope for our son.

There aren’t many people I trust with my son’s life, and Julian Raiman has stood out amongst those that I do.  His kind and caring nature shine through when he is with our kids and his loving demeanour has always made them feel safe and secure in the hospital, especially when things have been difficult.

His compassion always allowed him to connect with our rare disease families, and his empathy allowed him to grieve alongside us.   He understands us and recognizes how difficult these journeys are.  At the same time, he always gives families the strength to keep fighting, encouragement and hope that will sorely be missed by us all.

Julian has also led the charge for our children to get access to expensive treatments – treatments that save lives.  He’s traveled around the world presenting to patient groups, organizations, and government officials.

image2-2Julian has led clinical trials and helped pioneer new treatments for our kids.  I was with him at a Denver Patient Symposium recently and he passionately told the crowd that “It’s nice when we have medicines that make us get better.  But it’s just as important to have medicines that prevent us from getting worse.”  To me, that is at the heart of every battle I’ve fought throughout the past decade for our kids, and Julian eloquently summed up the need for us to have access to treatments for our kids in one powerful sentence.  He’s like that – inspirational and uplifting, yet modest and humble at the same time.

Julian has championed our childrens’ causes, he’s tirelessly joined our battles.

He’s been consoling and impactful.  And has made the world a better place for us all.

Julian has been a life changer and a life saver.

And Julian will be greatly missed.

Farewell, Dr. Raiman.  Thank you for all you’ve done for my son, our family, and all the families that The Isaac Foundation has been lucky enough to be involved with.  You are a true hero to us all.

With Love,

The Isaac Foundation


To Change The World – Treatment Approved in New Brunswick!

doubt that a small group
 of thoughtful, committed citizens can change the world; indeed, it’s the only thing that ever has. – Margaret Mead

The quote above is one that we have displayed prominently on our website, and they are words we live by.  Existing in the world of rare diseases requires one to have such a mindset – to make a difference in the world of rare diseases, you have to begin with a small group of people.  Here in Canada, Isaac is one of just thirteen individuals that suffer from MPS VI.  In the broader family of MPS, we’re still talking less than 250 people battling the disease country-wide.  That’s 250 people out of 35 million (or just .0007% of our population, for our mathematician supporters!)  In essence, we’re a small group.  But we think big, and truly believe that we can change the world for our kids.

I have this quote on our website for many reasons – one of them being to remind myself that things are possible, even when we’re facing odds that most people would bet against.  Little did I know, that quote would help a government approve a treatment for a little girl who desperately needs it.  Little did I know, that quote would help save her life.

The photo above is of Kamie Babineau, a beautiful little girl from a town near Moncton New Brunswick.  Kamie’s mom, Parise, connected with me in December 2014, worried that her daughter was suffering from MPS VI – the same disease that Isaac has. The same disease that Jasper and Aleena and Violet have.  The same disease we’ve worked tirelessly to find a cure for, the one we’ve had to battle governments to provide life-saving treatment for.  Parise was obviously worried for her daughter – she sent me a photo of Kamie and one of Aleena that she found in a newspaper report and felt they shared the same features.  One glance at Kamie’s photo told me she was on the right track, but confirmatory tests needed to be done before we could begin the process of setting up treatment for her.  Confirmation of MPS VI was given at the end of January, and an application for reimbursement for treatment went into the government of New Brunswick in early March.

If you’ve been following our blog, you’ve seen how difficult it is to access treatment for MPS VI in Canada.  It’s expensive (the third most expensive drug in the world, according to this website and many others like it,) and governments are reluctant to cover the cost of the treatment for our kids.  Reluctant, even though it’s life-saving.  Reluctant, even though it gives our kids the chance at a healthy life.  Reluctant, even though it staves off the ravages of the disease, slows down or halts its progression.  Reluctant, even though the cost of not treating the disease is sometimes far more costly than treating it when you take into account the costs associated with managing the multi-systemic failures that ensue in children without access to therapy.  If you need some reminders on how difficult it is for our kids to get access to treatment in this country, have a look through this collection of news articles and stories.  To summarize – it’s difficult, and treatment is rarely approved without a fight.

DSC_0144I had been told that New Brunswick would be especially difficult to deal with.  Indeed, I had already reached out to the Province a few months earlier upon learning that a boy with MPS IVA needed access to treatment and the provincial bureaucrats wouldn’t accept the application for funding because they didn’t have a process in place to review the application.  I called and stated my concerns and frustration that an application wouldn’t even be accepted for this child, let alone reviewed and denied.  I was told to wait until the bureaurcratic process of a CDR Review was completed and that they would not consider any request for treatment until that time (that review gets completed in 2 days from now!)  So, I was prepared for a difficult start to accessing treatment for Kamie, and I wasn’t wrong when I began the process a few weeks ago.

I again called the bureaucratic branch at the Ministry of Health in New Brunswick that looks after pharmaceuticals, and talked to the same person I had talked with many months earlier.  I patiently explained the situation.  I had previously sent an email with background information (it wasn’t read until I was on the phone with them), and explained that there were already 9 other children in Canada receiving the same treatment that Kamie desperately needed.  I offered to provide all the resources they could ever need to produce a review of the application, including the International Treatment Guidelines (which assert that treatment should begin immediately after diagnosis for best outcomes and that the primary way to deal with this disease is to provide the treatment we were seeking), and the recent 10 Year Resurvey Data (which shows the incredible outcomes for patients with access to treatment over a 10 year period).  I also sent in a full folio of MPS VI and other articles showing the benefits that treatment brings to kids suffering from this disease.  I offered to connect them with the leading MPS VI experts throughout North America to help them with the data.  I expressed and provided ample evidence to show that symptoms that appear prior to treatment beginning cannot be reversed.  Many of these symptoms are devastating, and I expressed that we were racing against time to stave off such symptoms and provide Kamie with the quality of life that she deserved, not the quality of life that a disease untreated would leave her with.  At the end of our conversation, I asked how the application would be reviewed and when an expected decision would be rendered.  I was told, quite bluntly, that they had “no idea.”

This isn’t uncommon when dealing with this disease and applications for reimbursement of treatment.  When a disease affects .0007% of a population, we can’t expect governments to know everything about the disease and the process that would be used to review applications for treatment.  However, what should be expected is that these types of situations are taken seriously.  What should be expected would be for the bureaucrats to spring into action, figure out what review process was needed to deal with the situation, with an immediate and expeditious review undertaken.  This is not what happened, unfortunately.  Two weeks passed with the same update – they had no idea how they would review the file.  In fairness, they did express their hope that it would be approved, but couldn’t figure out exactly how to initiate the file set before them.

DSC_0201From here, my impatience got the better of me.  But it’s an impatience that stems from necessity – necessity to get treatment initiated immediately, the knowledge that Kamie’s disease is progressive and unrelenting, the hope that she can get the help she needs before her body is further deteriorated by MPS.  This impatience is where the story changes for the better.

I placed a call into the Minster of Health, Mr. Victor Boudreau.  I’ve been fortunate to work with may of the Ministers throughout the country, and I hoped he would take the time to get back to me, hear my concerns, and put a plan in place to look after Kamie’s application.  Mr. Boudreau had his staff look into the issue.  We connected numerous times over email, and I provided him with details about the frustratingly slow action being taken by the Ministry.  What ensued thereafter is something I’ve not seen in this country, from any official dealing with treatment for MPS VI – a determined effort to review the application for funding, and a promise for a prompt resolution/decision for the family.

We scheduled a direct meeting with Mr. Boudreau at his constituency office in Moncton.  I hopped on a plane, met with Parise and her husband, and planned out how we were going to handle the meeting.  In previous fights for treatment for other kids, we learned to be prepared for anything, and we learned to show governments that we would do anything to help save our children.  We were prepared to let Mr. Boudreau know that we weren’t going to go away, that we would do everything necessary to help save Kamie.  We were in this for the long haul because treatment for Kamie was our only Hope for her future.

At the meeting, Mr. Boudreau began by telling Parise that cabinet had approved treatment for Kamie.  Just like that, her life was changed.  Just like that, her life was saved.  In two days – two days! – the application was reviewed by Mr. Boudreau’s team, a presentation was put to the government’s cabinet, and an approval was passed.  Quick action by Mr. Boudreau changed the lives of this family forever, and I’m incredibly indebted to him for it.

How does this story relate to the quote above?  The Minister told us that he was on The Isaac Foundation website to research the disease, our organization, and read about the work we’ve been doing for kids across this country.  Displayed prominently at the top of our page is Margaret Mead’s quote, he told us.  Minister Boudreau informed us that Premier Gallant, the leader of the government in New Brunswick, uses that quote all the time around the cabinet table.  He uses it to inspire his government to do what is right for people.  He told us that he took that quote from the website when presenting Kamie’s case to the Premier and told him that his government could be the “small group of people” in this situation.  They could be the ones to change the world, even if just for one family.

In all my dealings with governments, bureaucrats, and politicians over the past decade, I have rarely been as impressed as I was with Minister Boudreau on Monday morning.  He saw a problem, saw a need for prompt action, saw the need to help a family in need, and went about doing everything he could do to make things better.  While I’m sure it’s too early for him to understand what his actions, his passion, and commitment to make a difference really means, I know from experience that he will look back on this and see this as part of the legacy he’s left behind, part of the good he was able to do while in office.  Saving someone’s life will remain with you forever and, usually, change who you are for the rest of your life.  Just ask Heather Forsyth, leader of the Wildrose party in Alberta, the person who worked tirelessly to save the life of Aleena Sadownyk.  Just ask Elizabeth Witmer, the person who did everything in her power to save my son Isaac’s life.  I hope Minister Boudreau looks back on this with pride, and I hope his family can learn just how much of a difference he made to the life of a little girl this week.

I know this entry is long, but before I sign off I want to leave you with the other quote I live my life by, one that guides me wherever I go, whenever I get the chance to meet with families battling a rare disease like we are.  It comes from my favourite book, To Kill A Mockingbird, from a character whom I wish I could be more like in this world – Atticus Finch.  In this novel, Atticus works hard to teach his children about courage.  He leads by example, and hopes his children realize that they can make a difference in this world if they truly believe in what they are doing, no matter what the consequences may be for themselves.  Atticus tells his daughter that:

“Courage is not a man with a gun in his hand. It’s knowing you’re licked before you begin but you begin anyway and you see it through no matter what. You rarely win, but sometimes you do.”

In many ways, this is exactly what we face when we’re dealing with rare diseases and treatment for our kids.  The initial diagnosis is devastating for families.  Knowing a treatment exists provides hope, so much hope during those dark days.  Families are devastated again when they find out that governments deny access to those treatments – take that hope away – solely based on financial considerations.  Expensive treatments aren’t the fault of patients and families, but they bare the consequences.  Oftentimes, we fight with grace and courage to get the access to treatment we need, against all odds.  Rarely do we win, but sometimes we do.  We did yesterday, with the help of Minister Boudreau, and I can’t thank him enough.

So – where do we go from here?  Well, there is another child suffering from MPS in his Province who needs access to treatment.  He’s the same boy I called the Province about many months ago without success.  Perhaps the application we tried to submit many months ago can now be reviewed, and hopefully Mr. Boudreau can do what he can to assist.  With help, perhaps the little boy in need can now get the help he deserves.  I know I’ll keep trying to make it happen, and I hope to be met halfway by the Government of New Brunswick.

Thanks for hanging in to the end of this entry.  It’s a long one, but one of the most important ones we’ve ever logged.

Till next time,


Foundation Poster



On Leadership, Compassion, and Danielle Smith

Hi Everyone,

Just wanted to drop in to comment  on the recent Alberta election and the aftermath that the PC Sweep of all 4 seats has had on the political landscape there.  To say the least, I’m dumbfounded that the results of the by-election have led to questions about Wildrose leader Danielle Smith’s leadership.

To begin, for those that don’t know me, I’ve always been known as a staunch Liberal.  I’ve run as a Liberal nomination candidate, have been deeply involved in riding associations, and have even been the campaign manager for a strong candidate running for President of the Federal Liberal Party.  Most of my friends identify me alongside the Liberal Party, and whenever talk over a glass of wine or dinner turns to politics, I’m often turned to in order to offer the Liberal Perspective on things.

But to label me as a Liberal wouldn’t be entirely accurate.  I like to believe that I dedicate my life to helping protect the most vulnerable in our society – the ones that need a hand up; the people who need support so that they can reach their full potential in life.  Many parties identify with those beliefs, though some more than others. Obviously, I put my passion into practice when I decided to become a teacher, with my focus being on educating our future.  And more obviously, that focus changed, quite dramitically, when my son was diagnosed with a very rare and progressive disease.  On that fateful day in 2006, I promised my son that I would do everything in my power to protect him, to help him battle his disease.  And to help him win.  Along the way, I’ve been fortunate to play a small role in helping other children and other families fight back against this terrible disease, and along the way I’ve met some incredible people – people just as passionate about fighting for those in need as I am.  Danielle Smith is one of those people.

Under Danielle’s leadership last summer, the life of a little girl suffering from a rare disease was saved.  The government did not want to take action to provide little Aleena with the treatment she needed.  Indeed, her request for approval of her treatment was denied by Alberta Health.  Heather Forsyth, the opposition health critic for the Wildrose Party, worked tirelessly to help get that decision reversed.  And Danielle Smith was with us the entire way, supporting our efforts and ensuring time was allotted in the Legislature to hold the government to account for their inaction.  Heather and Danielle’s efforts made news from coast to coast, with National organizations covering the plight of Aleena. Because of her commitment to this family in need, Danielle helped save this young girl’s life.  And today, Aleena is thriving, growing, and living a rich, full, and happy life.

Danielle didn’t have to allocate the resources of her party or her time during Question Period to helping this one child.  But she did, because it was the difference between suffering or not for Aleena;  the difference between life or death.  Danielle ensured help was available because it was the right thing to do, and I was incredibly impressed with the way she committed herself and her party to wholehearted and unconditional support of Aleena and her family.

Since that time, I’ve had the opportunity to revisit the legislature and connect with Danielle in person.  And she’s renewed her commitment to fight for Albertans suffering from rare diseases, she’s committed to holding the government to account for their lack of action to help our kids – she’s committed to protecting the most vulnerable of Albertans – our sick children fighting a battle they need help to win.

After watching Danielle’s commitment to Aleena, I’ve watched the policies she’s put in place for her party – health care reform, ending extra school fees that have become an added burden on families, protecting taxpayers, and the list goes on and on.

So to those of you out there questioning Danielle’s leadership in the wake of the four by-election loses this past week (losses in what have always been considered SAFE PC SEATS, I may add), I ask you this – what Leadership qualities ARE you looking for if you feel Danielle isn’t the person you need?

To me, having someone at the helm of your party who has shown a strong commitment to those in need, who has demonstrated her ability to help the most vulnerable in our society, who believes that change can happen if people work together, and who continues to show a passion to protect our social safety net, would be a true blessing.  Danielle is a good person, and represents everything the Wildrose Party has come to stand for over the past few years.

General Douglas MacArthur once said, “A true leader has the confidence to stand alone, the courage to make tough decisions, and the compassion to listen to the needs of others. He does not set out to be a leader, but becomes one by the equality of his actions and the integrity of his intent.”  Centuries earlier, Napoleon said “A leader is a dealer in hope.”  From what I’ve seen and experienced over the course of the past two years, Danielle Smith embodies these definitions perfectly.

Far be it for me to tell the Wildrose what to do about their party and about their leader.  But please remember this before you pass judgement at the upcoming leadership review – Winston Churchill lost 5 elections, Lincoln lost 8.  True leaders aren’t judged by wins or losses, they are judged by quality of character, ideals, compassion, and vision.  More often than not, true leaders end up on the winning side of things eventually – sometimes patience is required, but leaders tend to win out.  And Danielle Smith is a true leader, one you should be proud and are lucky to have.

So, before you vote, take a step back and see the forest for the trees.  I bet you’ll find you are in good hands with Danielle.


A “Liberal” from Ontario.


10358314_229339850610555_1309889474_nHi Everyone,

Isaac’s just gone in for some routine surgery – removal of a dental cyst and a full eye exam under general anesthetic (his eyes have clouded over so badly that they can’t see in anymore, hence the more advanced look at things today.)  For most families, that is routine.  But for families dealing with MPS, anything under a general anesthetic is anything but routine, which is why Ellen, Gabriel, and I are sitting here, stressed and worried for our little boy.

Children suffering from MPS have compromised airways, and general anesthetics should be avoided at all costs.  I’ve heard of too many complications that our beautiful kids have had while under a general to sit here and be relaxed about the process.  MPS Specialists always recommend ensuring the best anesthesiologists handle our kids, and we are lucky to have the best today.

We are in the Surgical Waiting room at the Hospital for Sick Children, a place that I’ve grown accustomed to hating.  The tension in this room is unbearable, and the waiting is worse.  I can’t count the number of blog entries I’ve written from here – it gives me something to do to keep my mind off things.

Parents and families dealing with MPS gain a unique perspective on life throughout the entire journey, and perhaps more so while sitting in a room like this.  Sitting here, we’ve given up our child to the hands and arms of some of the best physicians in the world, and we have to trust that things will go smoothly.  I’m comforted in the fact that Dr. Cengiz Karsli (pictured above with Isaac), an incredible anesthesiologist that has handled Isaac’s care since our first surgery here, is once again handling things for Isaac today.  We were initially told that he wasn’t scheduled for Isaac today, and our stress level went through the roof.  But he arrived and immediately made us feel better that he was there.

When Isaac was 2 1/2 years-old, he had a very major spinal-cord decompression surgery.  The compression was so bad that they had to route out a piece of his vertebrae with a diamond drill bit.  Needless to say, it was a very difficult surgery and we were terrified for our son.  A few hours into the surgery, Dr. Karsli came into the waiting room looking calm and relaxed – he was actually chewing away on an apple and had a smile on his face.  He dropped in quickly to tell us that things were going OK and not to worry.  That moment made us admire him immediately.  It was something he didn’t have to do, but he did so to put our minds at ease, and I’ve always been grateful to him for it.  He probably doesn’t even remember that moment, but we sure do!

His relaxed nature is so helpful, but the kindness and care he shows our son really sets him apart from the rest.  Even if Isaac’s airway doesn’t give him any trouble this morning, we’ll always do whatever we can to ensure that Dr. Karsli keeps Isaac under his care for the next surgery (and there will be more).  If nothing else, this process is easier on us all with him being here, and we wouldn’t have it any other way.

Thinking back to our first surgery, this room felt so lonely for us.  Ellen and I sat here worried sick for our son, and it felt like it was just us dealing with things on our own.  We had just started our charity, and were trying to figure out how we were going to find a cure for our boy before it was too late.  Today, 8 short years later, it feels like we have an army of support behind us, and this room doesn’t feel as lonely as it did before (I still hate it, however!)  I posted a quick photo of Isaac earlier, and we’ve received so many words of hope and encouragement, and I’m incredibly thankful for that.  And with that same help and support, we’re well on our way to finding a cure for our kids, and we can’ thank you all enough for being here for us always..

I’ll update once Isaac comes out of recovery and once I find a spare moment.

With Love and thanks always,


2014 Research Grants

research-grantsThe Isaac Foundation is pleased to announce two research grant opportunities with a call for a $100,000 grant through our general MPS Fund and a call for $50,000 from our MPS II Research Fund.

Last month also saw us renew a research project for $50,000, bringing our total research grants from The Isaac Foundation early 2014 to $200,000!

We continue to be proud of the work that we do in the MPS Community. To date, we are well over 1/2 million dollars granted to research projects throughout the world and we will continue to grow and support innovative research until a cure for MPS is found.

Calls for Applications to these grants will be issued soon.

We are also very pleased to announce that Dr. Barbara Burton and Dr. Paul Harmatz, both world-renowned MPS specialist from the United States, have joined our Medical Advisory panel to review and advise on research applications as they come in. Dr. Burton and Dr. Harmatz join Dr. Julian Raiman and Dr. Joe Clarke to round out our 2014 Medical Advisory Panel.

More details to follow soon. Thanks for you continued support as we seek to find a cure for our kids!

Welcome Back, Mr. Mayer ;-)

isaac mayerHey Everyone,

Just a quick post to Welcome Back to someone we’ve been proud to stand behind for a long time now.  Those of you that follow our blog  and our story regularly can attest to the many defences we’ve put up on John’s behalf. Through the many years of making incredible music, through the tough times in the media, and through the storm of public backlash and innuendo, we’ve always told everyone we know that John Mayer is a truly kind soul, and someone worth waiting around for.

The caring, kindness, and compassion that he’s shown our son Isaac has given him lifelong memories that he’s proud of.  And when things are tough, or he’s holed up in a hospital far more than any kid his age should be, Isaac’s always had the opportunity to look forward to a special moment or two with the person he looks up to most.  With an upcoming tour on the horizon, Isaac and Gabriel can’t contain their excitement!

Welcome Back, John.  Glad to see you becoming the person you’ve always known, and we’ve said, you were 😉

PS – Watch for Isaac and Project One Million in Canada’s GLOBE AND MAIL tomorrow morning, or sometime later this week.

McFadyens Fight For Children With MPS VI

The following is part two of an interview with Andrew McFadyen, the father of eight-year-old Isaac who has the rare disease MPS VI. Wednesday’s story featured Isaac’s continued battle with the disease. Today’s story focuses on the McFadyen family’s advocacy for other children diagnosed with the disease. Friday’s story will announce their lofty new fundraising project.

CAMPBELLFORD — First, back in 2006, it was for his own son Isaac. Then, last year, it was for another Ontario boy, two-year-old Jasper More. Now, it’s for a three-year-old Saskatchewan girl, Violet Revet.

All three young children (although Isaac is now eight) were diagnosed with the rare enzyme deficiency disease MPS VI (Maroteaux Lamy Syndrome). Two of them — Isaac and Jasper — were initially denied funding from their provincial government for the life-sustaining treatment that is available to help to help stave off the devastating symptoms of the disease. Violet, the most recent case, and her parents — Jennifer Yanke and Larry Revet — are still waiting to find out if she is approved or denied. She is only the ninth confirmed case of MPS VI in Canada. There are about 1,100 worldwide.

Andrew McFadyen has lobbied governments to ensure the children get the treatment they so desperately need.

Make no mistake, McFadyen pours his heart into every lobby effort, whether that’s for his own son or someone else’s child. He just wishes he didn’t have to. One child denied, one lobby effort is all one too many, as far as he’s concerned.

“This is now the third big advocacy push that we’ve had to look after and that’s a big problem to me,” he said in an interview earlier this week. “You would think after the first one that precedent has been set and people can go forward and change public policy for the better and ensure there’s an active review process for this type of treatment.”

When Isaac McFadyen was diagnosed with MPS VI, the news was devastating to his parents Andrew and Ellen. Symptoms of MPS VI include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span.

Somehow, they had to find the strength to fight for funding from the government. Treatment, called Naglazyme, provided hope. It’s an Enzyme Replacement Therapy designed to provide patients with a synthetic version of the enzyme they are lacking.

Naglazyme is only available to Canadian patients through the federal government’s Special Access Program. The treatment comes with a hefty price tag of a million dollars a year, to be paid by provincial governments. From the McFadyens perspective as parents, though, you can’t put a price tag on your children.

“We know that this (treatment) is not a cure. I never tell anybody Isaac needs this, or Jasper needs this, or Violet needs this because it’s going to cure the disease,” McFadyen said. “We’ve always said it’s a lifeboat. It’s something that we can provide our children with to slow the progression of the disease and in some cases halt the progression of the disease until we can find that cure. It allows children to have a more normal and healthy life.

“From a parent’s perspective, that’s what you want for your children, to give them the best opportunity at a normal, healthy life,” he continued. “From a government’s perspective, I know they look at this for dollars and cents. They don’t look at the cost of the disease for them if it goes untreated. They don’t look at the cost of corneal transplants, double hip replacements, heart and airway disease…they don’t take that into account. They just look at that cost of one million a year and balk at it. It makes me angry, it makes me upset, and as a parent it really makes me question the importance of children with rare diseases across this country.”

Back when the McFadyens were lobbying for funding, they were strongly considering a move to England, where the treatment is approved. The country was willing to take the family and pay for Isaac’s treatments.

“It’s a priority there, because they have an Orphan Drug Plan, they have policies in place to ensure this is dealt with at an early stage and the best interest of the child is looked at,” McFadyen said. “Kids diagnosed with the disease start treatment within weeks.”

Funding for Isaac’s treatment was eventually approved by the Ontario government. The McFadyens received help from former Conservative MPP Elizabeth Witmer, who was the opposition’s health critic at the time. She invited the Campbellford family twice to the Ontario Legislature. In July 2006, the family received word from then-minister of health George Smitherman that the provincial government would fund Isaac’s ERT treatments.

There couldn’t have been better news at that time. Surely it inspired the McFadyens to help others. That same year they established The Isaac Foundation to help raise awareness of the MPS VI and also money to help fund innovative research projects.

Having The Isaac Foundation has also provided a connection to other families who have had their children diagnosed with MPS VI.

In August of 2011, Darren and Pam More of Palmerston, reached out to the McFadyens when their son Jasper was diagnosed with the disease. Six weeks after the diagnosis, the Mores learned that Jasper had been denied funding to receive the treatment.

That was a shock to McFadyen, that a second child had been denied in this province.

“We were really surprised and obviously really angry,” he said.

With the story spreading through news coverage and on Twitter, led by McFadyen’s efforts, within days the More family received word that the decision had been overturned and Jasper would get funding for the treatment after all.

“When we finally got approval and knew that (Jasper) was going to be covered, that should have been in it and the rest of the provinces should have been on board,” McFadyen said.

British Columbia and Quebec were already on board following Isaac’s case when children in those provinces were diagnosed.

Saskatchewan, which has the newest case, was a different story when Violet was diagnosed.

“To have another province go through this exact same thing, for us it’s astounding that we can still be in this position — that we can’t look after our children that are suffering from these rare diseases,” McFadyen said. “It really goes to show that a lack of an Orphan Drug Plan here in Canada does impact our kids in a negative way.”

McFadyen is also concerned that it took 18 months for Violet to receive the proper diagnosis that she was suffering from MPS VI. Worse, he says, is that Violet has since been waiting an additional seven months waiting for the approval of treatment.

“The timeline between symptoms appearing, diagnosis and then beginning treatment is really detrimental to Violet and her health and it’s actually embarrassing for our health care system to have those statistics out there because we aren’t looking after the needs of these kids suffering and I really feel for the family and I really feel for Violet,” McFadyen said.

McFadyen noted the Saskatchewan government says they are looking into the safety of the treatment. He said that shouldn’t be a concern since it’s approved in the United States and England and is already being used in three other Canadian provinces through the Special Access Program.

“It’s just not approved here in Canada because of the limited number of children that are affected by this disease,” he said. “What they’re really doing is looking at the cost on a year-by-year basis. I understand that it’s not easy to commit a million dollars in funding for one child per year for a government, but it seems to be that we have a great health care system until you get really sick or in need of something that’s really expensive. Then there’s questions on whether they’re going to treat you or help you.”

Due to how rare these cases of MPS VI are, providing funding, McFadyen suggests, isn’t going to bankrupt a government.

In addition to the advocacy work McFadyen has been doing behind the scenes, he said a social media campaign evolved for this case. The Twitter and Facebook pages for Saskatchewan Premier Brad Wall were flooded with public comments calling to approve the funding for Violet.

At this point, McFadyen has the experience of knowing what governments need to ensure these files move quickly. He can also provide any safety data they require and the benchmark information they need.

“My issue was the timeline involved here,” he said. “It doesn’t seem to be the highest priority. When I take to Twitter to express that fact or when I put my press release out, sometimes it’s surprising how many people pick that up and are interested and engaged and start that snowball effect of tweeting the premier and hitting his Facebook page.

“We’re getting responses from the premier and responses from the people involved and they are signaling that they’re listening to us and they hear us and work is moving forward in a timely fashion. Now it’s my role to ensure that timely fashion is within the timelines that Violet needs and that we as Canadians expect the people making our decisions on our behalf.”

McFadyen said a decision could be announced any time now.

While Isaac has been on the treatment for several years now and benefitted from it, even little Jasper More has made great strides a year later.

“Jasper is doing so well,” McFadyen said. “He’s had no ill effects from the treatment that he’s receiving, the disease seems to have slowed or even halted in him, and that’s really positive because we know if children get on this treatment really, really early, the outlook for them is spectacular.

“There’s all the hope in the world that he won’t have to suffer any of the symptoms that this disease can lead to. The family is doing okay. I think they’ve come around to their new circumstances in life and are enjoying every moment that they have together. We see them often. They come to all of our Isaac Foundation events, and they’ve joined our board and the charity and they’re looking after a lot of the fundraising with us and they’re really engaged to find a cure. To see Jasper running around with the big smile he has on his face is really rewarding.”

Now, he wishes the same for Violet.

“Ideally, we win this one last battle and public policy can be set for the rest of the country and maybe it will spark Canada to come up with an Orphan Drug Plan so this never has to happen again for any child affected with any type of rare disease,” McFadyen said.

UPDATE: Violet’s family has received word that the funding has been approved and she will get the treatment she needs.