Blog Postings | The Isaac Foundation

24 Mar 2015

Sticky Post By mcfadyena Posted in Advocacy, Blog Postings Permalink

To Change The World – Treatment Approved in New Brunswick!

Sticky Post By mcfadyena On 24 March, 2015

Never 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.

I 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.

From 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,

A.

06 Jan 2015

Sticky Post By mcfadyena Posted in Blog Postings Permalink

Dr. Cengiz Karsli

Sticky Post By mcfadyena On 6 January, 2015

Hi Everyone,

There are people in this world who do extraordinary things, simply by doing what they love to do, by the way they interact with others. People who change lives without knowing it, make the world better just by being in it. Dr. Cengiz Karsli is one of those people.

Cengiz has been Isaac’s Anesthesiologist since the very start – since Isaac was 18 months old and about to undergo a massive spinal-cord decompression surgery. We’ve written about him often from time to time, and many of you have asked about him (and if you should ensure you get him for your own children!)

He’s always one to respond to emails when we need him, and goes out of his way to ensure he’s available to be here for Isaac and our family for surgeries. Ellen and I couldn’t imagine going through the stress of one of these surgeries without knowing that he is on hand to look after our son. He’s the calming presence we need before things begin, he’s the utmost professional as the surgery gets started, and he’s one of the finest Doctors at Sick Kids in Toronto, so we’re rest assured knowing that Isaac is safe in his care. Once all is said and done, he finds us quickly and lets us know how things went in the warm way that parents need. We arrived at the hospital this morning at the height of our stress. We left feeling relieved, relaxed, and thankful. We have Dr. Cengiz Karsli to thank for that. And we will never be able to thank him enough.

Cengiz was only supposed to be on call this morning. But upon hearing that Isaac was going to be in surgery, he scheduled himself in for the procedure to look after him, and then he stayed on until 1 pm. As we were heading out of the hospital tonight, Cengiz was coming back in – he’s on call for the next 12 hours as well. I’m sure he’s tired after a long day and staring at an even longer night. But he caught us as we were heading out the door and checked in on our boy, patiently answered all of Isaac’s questions, and bid us all a good night.

There aren’t many people like Cengiz in this world, someone who goes out of their way to make things easier for kids and families. Many MPS specialists are moving to a system where only 1 or 2 experts are used for surgeries on our kids. Their airways usually dictate that only the best should be involved with these procedures. We’re lucky that we have the best, and that he’s a wonderful person at the same time.

Isaac is now resting comfortably at our hotel for the night. We’re back tomorrow for a check in on his eye and his infusion. I’ll keep everyone updated as I can.

Thank you to everyone who checked in on us today – we truly appreciate your support.

Best,

A.

05 Jan 2015

Sticky Post By mcfadyena Posted in Blog Postings Permalink

Transplant Tomorrow

Sticky Post By mcfadyena On 5 January, 2015

Hi Everyone,

Sorry for the delay in posting – we’ve been busy with work, family support, and working hard to access treatments for kids across Canada that need help. At the same time, we’ve been taking a few weeks to spend time together has a family and get ready for Isaac’s corneal transplant, set to take place tomorrow morning.

I wasn’t going to post anything until all was said and done. But I received a few notes over the past week from wonderful supporters asking how we are doing. They noted that we’ve been posting a lot about the work we are doing on behalf of other families and were wondering how Isaac and the rest of our family was doing. Fair enough, and I appreciated the kind notes.

To say the least, we’re nervous – afraid really – of all that comes with this procedure. It’s daunting to think about the possibilities, so we’re concentrating on the positives and the potential benefits that this will give to Isaac. Imagine – he may be able to see the stars again, something that he stopped being able to see a few years ago. We are thrilled that his vision may be improved and we desperately hope everything will go smoothly.

I’ll do my best to update how things are going. All 4 of us are heading into Sick Kids Hospital this evening, with the transplant set for early tomorrow morning. We’ve got the best team possible looking after Isaac’s care, including his longtime Anesthesiologist Dr. Cengiz Karsli, pictured with Isaac below.

Thanks for checking in. I’ll update when I can.

A.

30 Oct 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

On Leadership, Compassion, and Danielle Smith

Sticky Post By mcfadyena On 30 October, 2014

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.

Respectfully,

A “Liberal” from Ontario.

07 Oct 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

Isaac Update – Corneal Transplant

Sticky Post By mcfadyena On 7 October, 2014

Hi Everyone,

I’ve just returned from one of the most rewarding weeks I’ve ever had in my role as Executive Director of The Isaac Foundation. I’ll save updating on all that was the MPS and Adulthood Conference for another blog posting. For now, I wanted to give you an update on Isaac’s health as the conference, for me, was overshadowed by the news we received yesterday (and while I was still away) that Isaac requires and will be undergoing double corneal transplants soon.

This news was shocking and upsetting to us, but not surprising. We’ve always known that this was on the horizon – or that the possibility of this was on the horizon. But he’s been so stable in his health during the past 6 months that it left our minds as a possibility. But stable isn’t quite the right word – he’s improved since January, improved considerably. I guess this is why the news we got yesterday was a tough to take.

First off – Isaac’s being incredibly brave. Please know that. Me? Not so much, but I’ll stay strong because it’s what I have to do. He expressed very eloquently that how his eyes are important to him because he need to be able to read (he’s a veracious reader and it’s unimaginable to him that that could be put in jeopardy.) He wants the surgery to protect that hobby, which is both incredibly cute and heartbreaking.

Now – corneal transplant…what is this and why are we here now?

Kids with MPS accumulate a buildup of cellular waste in their bodies known as glycosaminoglycans, or GAGS. Enzyme Replacement Therapy (ERT) helps to break those GAGS up and clear them out of the body. But sometimes ERT doesn’t prevent the clouding (caused by the GAGS) that takes place in the eyes. And Isaac has always had corneal clouding. Quite severe, in fact, but it’s been severe since he was very young. In fact, if you were to look at Isaac’s eyes today, they don’t have much colour – they are grey due to the clouding.

Isaac’s glasses have corrected his vision over the course of the past numbers of years. The clouding has continued to get worse – to the point where the doctors haven’t been able to see into his eyes for about 2 years. But still – his vision has been stable. A big change happened, however, over the past 6 months. Vision in his left eye has deteriorated considerably – 20/30 down to 20/80, and this is quite concerning. The result? A decision to move on with the transplants that we thought were still a number of years off, in the very least.

Doctors are very good at doing this surgery, though I have to admit that I’m still very scared and worried. But our MPS community is amazing, and many parents are rallying around us with love and support – something I’m truly grateful for. And Isaac’s friend since childhood, who also suffers from MPS, has had both of her cornea transplanted. Her family will be a great resource for us as we embark on this latest battle, and she will be able to talk to Isaac about what to expect.

And quite serendipitous – when I arrived home, there was an invitation in my email for me to tour one of the best (if not THE Best) transplant clinic for kids with MPS in the US. I’m excited to attend and, perhaps, garner a bit of information on the process along the way. The clinic specializes in bone-marrow transplants and stem-cell transplants, but they be a wealth of information for me for this comparatively smaller and less serious procedure.

Anyhow – I feel a tad better now that I’ve written this and the decision has been made. It was hard to get the news while I was away. All I wanted to do was hug my boy, struggle together with my family, and be here for each other when we needed it most. It was a tough night last night, and a long flight home. But as soon as I arrived at the school to pick up my boys, they both jumped into my arms and hugged me for what seemed like an eternity. And Isaac looks good, and strong, and – as he always does – brave. He can do this, and so can I.

Isaac shared the news with his best friend at school, Amy. I’m so thankful that he has someone he can trust to talk to, and I’m sure it made him feel better to share the news with his friends.

I’m listening to Danny Michel as I type this blog update – poignant because he’s playing at our upcoming Gala For A Cure. The song below is called “Just The Way I Am”, and it’s providing the perfect soundtrack for my frame of mind right now as I think about Isaac, his bravery, and the joy I know he takes in having a supportive friend to talk to when things get tough. It’s below for you to listen to as well – it really is a beautiful (and perfect) song for this posting.

Thanks for letting me ramble on. I’ll update with more information when I can. Thanks for always being here for us and our kids.

With Love,

A.

16 Sep 2014

Sticky Post By mcfadyena Posted in Blog Postings, Instagram Photos Permalink

Always a trooper. #Brave

Sticky Post By mcfadyena On 16 September, 2014

Quick…well not that quick, visit to Sick Kids Hospital in Toronto today for Orthopaedic Surgeon followup. It was a 4 hour wait (really, this needs to be fixed) to see the surgeon, but he had great news!

Isaac’s kyphosis (curvature of the spine) has improved by 4%, and his slipped disc has corrected itself. While we can’t be certain, we’re convinced it’s the effect that Elmiron has had on him.

Truly grateful.

04 Sep 2014

Sticky Post By mcfadyena Posted in Advocacy, Blog Postings Permalink

FOUNDATION RENEWS CALL FOR COMMITMENT TO RARE DISEASES

Sticky Post By mcfadyena On 4 September, 2014

– FOR IMMEDIATE RELEASE –

PC Leadership Candidates Continue Silence On Funding For Rare Disease Treatments in Alberta

In a leadership race widely criticized as being void of ideas and real policy debate, The Isaac Foundation is again offering candidates an opportunity to get on record about an issue affecting the lives of a number of Alberta children.

Last week, the Foundation requested candidates state their position on treatments for rare diseases that affect Albertans; treatments that traditionally come with a high cost but also prove lifesaving for affected individuals. Thus far, all three candidates have refused to share their views on the importance of such treatments and how they can be accessed by those in need.

Andrew McFadyen, Executive Director of The Isaac Foundation, highlighted the recent case of Aleena Sadownyk, a 4-year old St. Albert toddler that suffers from MPS VI, a very rare, progressive, and debilitating disease that attacks the bones, tissues, organs, and muscles. Without access to a weekly enzyme replacement therapy (ERT), Sadownyk would have developed bone and joint disease, heart and airway disease, progressive stiffening of the joints, and had a severely shortened life-span. Initially, Sadownyk was denied access to the life-saving treatment through the Alberta Rare Diseases Funding program due to its high cost, estimated at close to $300,000 per year. After a very public advocacy campaign that garnered national support, and with tireless help from Alberta MLA and Wildrose Health Critic Heather Forsyth, Sadownyk had her treatment approved. Today, she is thriving, happy, and the disease progression has been halted.

Currently, there are 5 other children in Alberta suffering from a similar form of the disease. Morquio A, or MPS IVA, manifests itself with the same devastating symptoms that Aleena was facing. Health Canada approved the first and only treatment for the disease 10 weeks ago and the only hurdle for patients in Alberta to starting treatment immediately is reimbursement by the Alberta government. Without the political will to ensure that rare disease treatments are a priority in Alberta, reimbursement for treatment could take upwards of 3 years, a timeline that McFadyen says is detrimental to the long-term health and well being of patients.

McFadyen admits that he is frustrated by the lack of response from the candidates vying to become the next Premier. “The leadership vote is set to begin Friday morning, and Albertans deserve to know where the candidates stand on rare disease treatment funding prior to casting a ballot. To date, the race has been preoccupied with expense and travel scandals but these children need action and they need a commitment from their future Premier today. I’ve had healthy and productive discussions with high-level bureaucrats at Alberta Health Services, and I’ve had a series of meetings with Wildrose Health Critic Heather Forsyth and leader Danielle Smith, who have both been committed to ensuring those in need get the equal access to the health care system that they deserve. But there continues to be a lack of will from the candidates to do the same.”

He adds, “Leadership is about recognizing issues of importance to those that you hope to represent, and silence on this issue leads one to question where health care priorities lie with these candidates. Any one of these politicians can make an immediate and significant impact on the lives of children suffering from MPS, and I implore them to share where they stand on this issue before Election Day.”

# # #

For more information about this topic, or to schedule an interview with The Isaac Foundation, please call our our Executive Director at 613-328-9136 or email Andrew at mcfadyena@me.com.

18 Aug 2014

Sticky Post By mcfadyena Posted in Blog Postings, Instagram Photos Permalink

MPS Dads – West Coast Edition ;)

Sticky Post By mcfadyena On 18 August, 2014

Had a great visit with Dane Sadownyk today on the anniversary of Aleena’s treatment approval! What a great family, and an inspiration to all families fighting MPS.

I look forward to many years working together, Dane. Thanks for a great visit.

14 Jun 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

2014 Research Grant Award Recipients

Sticky Post By mcfadyena On 14 June, 2014

RESEARCH GRANTS ANNOUNCED!

We are thrilled to announce that The Isaac Foundation has granted $200,000 in research grants to 3 innovative projects aimed at finding a cure for MPS!

We are proud to award $100,000 to Dr. Alberto Auricchio in Italy for his incredibly exciting work on Gene Therapy. We hope to see this work head to clinical trials in early 2015!

We are also very excited to award 2 $50,000 research grants from the MPS II FUND, under the leadership of Deb Purcell! The recipients of these awards are granted to Dr. Scott McIvor at the University of Minnesota for his work entitled “AAV Mediated IDS Gene Transfer for MPS II” and to Dr. Brian Bigger at the University of Manchester for his work “Evaluating Stem Cell Gene Therapy for Treating the Brain in MPS II”

This brings the total awarded over the past 6 months to $250,000 – all in hopes of finding a cure for our children.We look forward to seeing results from this research and will continue our work raising money to support these projects and more during the course of the next few years.

Thank you for your incredible support. None of this can happen without you.

12 May 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

Surgery

Sticky Post By mcfadyena On 12 May, 2014

Hi 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,

A.

21 Mar 2014

Sticky Post By mcfadyena Posted in Blog Postings Permalink

Research Grants

Sticky Post By mcfadyena On 21 March, 2014

The Isaac Foundation is proud to announce that our application for our 2014 Research Grants is now available. $100,000 is available from our general MPS Research Fund, and another $50,000 grant is available from the MPS II Research Fund.

Application deadline is May 1. A decision will be reached by June 15, with funds dispersed on or before June 31, 2014.

Our Grant Application Form Can Be Downloaded Here – RFA 2014 – MPS VI and MPS II

If you have any questions regarding the process, please email Ellen Buck-McFadyen – ellen@theisaacfoundation.com