treatment | The Isaac Foundation

04 Jul 2016

Sticky Post By mcfadyena Posted in Advocacy, Morquio, MPS IVA Permalink

Vimizim Compassionate Use Update

Sticky Post By mcfadyena On 4 July, 2016

Some exciting news to share – The Isaac Foundation spent the entire month of June ensuring continued access to Vimizim for our Canadian Clinical Trial patients suffering from Morquio A Syndrome.

The compassionate use program, which saw all patients who participated in the Vimizim clinical trial receive access to therapy for a certain period of time, was set to end on June 30, 2016. This would have resulted in the remaining 11 patients losing access to their much needed treatment until it was fully reimbursed by the government.

Working collaboratively with the Ministry of Health in Ontario and with Biomarin pharmaceuticals, I’m thrilled to report that we’ve been able to ensure access to treatment for all 11 patients until Biomarin and Canadian Provinces (pCPA) can negotiate terms and conditions for coverage for all patients. Both Biomarin and the government did fantastic work on this, and we’re thrilled with the outcome.

We will continue to do everything we can to help patients and their families receive the best care possible as they battle this and other ultra-rare diseases. Thanks to everyone for supporting us and helping along the way.

#Hope, always…

18 Feb 2016

Sticky Post By mcfadyena Posted in Blog Postings Permalink

LOCAL CHARITY CELEBRATES 10 YEAR ANNIVERSARY

Sticky Post By mcfadyena On 18 February, 2016

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 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.

13 Dec 2015

Sticky Post By mcfadyena Posted in Advocacy, Blog Postings, Morquio, MPS IVA, Newspaper Permalink

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

Sticky Post By mcfadyena On 13 December, 2015

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

09 Dec 2015

Sticky Post By mcfadyena Posted in Advocacy, Media, Morquio, MPS IVA, Newspaper Permalink

Health minister under pressure to approve expensive drug for sick boy

Sticky Post By mcfadyena On 9 December, 2015

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

09 Dec 2015

Sticky Post By mcfadyena Posted in Advocacy, Morquio, MPS IVA, Uncategorized Permalink

This Is Not OK – When Answers Aren't Really Answers

Sticky Post By mcfadyena On 9 December, 2015

To me, this is the most difficult part to accept. No appeal? No opportunity to ensure the decision was rendered correctly before we condemn this little boy to a life of pain and suffering…to death? Prisoners on death row – prisoners there for the heinous crimes they have committed – have multiple opportunities to appeal their decided fate. Before that, their fates get decided by collections of people, people who have the opportunity to hear all viewpoints, all expert testimony, everything. They exhaust all avenues in making death sentence rulings, and then we allow those prisoners the opportunity to appeal and fight their fate. Not so for this little boy, it appears, and that will never be OK with me. ~Andrew McFadyen, Executive Director, The Isaac Foundation

Late last night, I boarded a plane to Fredericton with hopes of meeting with the Minister of Health, Victor Boudreau, about 10-year-old Morgan Doucet. Recently, the Minister denied access to the life-saving medication that Morgan needs immediate access to, thereby condemning Morgan to a life of pain and suffering, and immediately signalling that Morgan’s care should enter a palliative approach to his disease.

This is NOT OK, for a whole host of reasons, not least of which is that we CAN help Morgan, we can stop his disease progression, and we can do it quickly with access to the treatment he needs.

This is not OK because the treatment Morgan needs has been approved by Health Canada for almost 2 years now, and it’s being used to help save the lives of 33 patients throughout the country, while being fully reimbursed for use in patients in Saskatchewan, Ontario, and Quebec. It’s also funded in most developed countries throughout the world – including the UK – where they recently took steps to fund every patient that needs access immediately.

This is not OK because Morgan’s review was done by one person alone, a person who has never used this treatment before and a person who isn’t a practicing physician anymore. He’s a bureaucrat, paid by the government of Ontario to review files and render life and death decisions about kids – life and death decisions that come without rigorous debate and input from the true experts in the field.

This is not OK because the overwhelming evidence and recommendations by those true experts in the field all conclude that this drug works and should be given to patients immediately in order to stave off the ravages of the disease. It has been prescribed by Morgan’s genetics specialist, it has been recommended for use in patients by the Canadian Expert Opinion on Morquio Syndrome and it is recommended as the front line treatment for Morquio Syndrome in the International Treatment Guidelines for Morquio Syndrome. In fact, it’s considered the gold standard of care for patients suffering from this disease, and it’s inhumane and unethical to deny patients the help they need when they need it most.

This is not OK because we are allowing those bureaucrats and politicians to overrule and contradict medical experts, clinicians, and researchers, regarding best practice and treatment for this little boy suffering from this devastating disease. In essence, the Minister and his bureaucrats are making clinical decisions where they are not qualified to do so.

And this is not OK because denying life-saving treatment to dying kids is not what our Health Care System is supposed to stand for, it’s not the principles that our country and our Medicare Act were founded on, and it’s not who we want to be as a Canadian People.

With all this on my mind, I took the late flight here for an arrival in New Brunswick in the middle of the night. I came to Question Period this morning to hear MLA Jake Stewart ask the hard questions of the Minister that he’s been avoiding answering. Jake was poignant, thoughtful, and strong in his belief that Morgan should receive access to this drug. After all, the Liberals themselves said last year that they “can – and MUST – do better” for our kids suffering from rare diseases.

With three questions asked, Minister Boudreau decided not to provide proper answers. He started by saying there is a process, and that process includes a CDR report and they won’t fund drugs until that process is complete (it is, and was completed a long time ago). He neglected to mention that no MPS drugs ever get the blessing of the CDR panel, yet New Brunswick is paying for every other drug for MPS diseases that are currently on the market. He neglected to point out that the only patient with MPS in this province that isn’t receiving the life saving drug they need is Morgan. He neglected to say that this has less to do with what the experts say and more to do with the cost of the drug, something that should never be a hinderance to proper care in this country.

Minister Boudreau commented on the process used – Morgan’s file was sent to a reviewer in Ontario to make a decision whether to treat or not. One reviewer. With no avenue for appeal after any decision is rendered. He didn’t comment on that in Question Period, but after the decision was delivered I asked the Ministry of Health how we can appeal the ruling. I was ignored. I asked the Minister of Health 7 times for a meeting or information about how we can appeal the ruling. I was ignored. And I asked the Premier multiple times for a meeting to discuss an appeal. As you all know, I received a note telling me that he was unable to meet, with apologies “for the inconveniences this may cause.”

To me, this is the most difficult part to accept. No appeal? No opportunity to ensure the decision was rendered correctly before we condemn this little boy to a life of pain and suffering…to death? Prisoners on death row – prisoners there for the heinous crimes they have committed – have multiple opportunities to appeal their decided fate. Before that, their fates get decided by collections of people, people who have the opportunity to hear all viewpoints, all expert testimony, everything. They exhaust all avenues in making death sentence rulings, and then we allow those prisoners the opportunity to appeal and fight their fate. Not so for this little boy, it appears, and that will never be OK with me.

As I was typing this entry, Minister Boudreau dropped over to meet with me to discuss Morgan and his plea for help. I am thankful that he did, as I was able to present everything I’ve written above. He made a commitment to take a look at this case but truly believes in the process put in place. In his words, he will make not promises or commitments, but he will look at things and get back to me.

Knowing this, I implore Minister Boudreau to take a real look at this case and seek a new review of Morgan’s application. I asked Minister Boudreau to seek outside opinions from a panel of 3 or 5 International and Canadian experts that deal front-line with this disease and this treatment each and every day. I asked Minister Boudreau to allow a rigorous discussion on all available evidence that exists on this drug and the impact it has on patients, and I asked for expediency because it’s unethical to make a child and his family wait.

I asked this to allow for a transparent and fair process for Morgan. His life depends on it and it’s the right thing to do. And in the end, if the Minister doesn’t want to listen to and seek feed back from clinical expert opinion, patient reported outcomes, peer-reviewed published journals, etc., then who will he listen to? The opinion of 1 person who has never used this treatment and didn’t engage with those experts to help make his decision on Morgan’s fate?

If that’s the case, it’ NOT OK.

I’ll be here for a short while and will be putting out a PR tomorrow morning so that media can cover this story if they choose. Thank you for all of your support over the past little while – our last entry was seen over 20,000 times in a few short days. Sharing Morgan’s story is helpful, and staying ready to fight means the world to us.

I’ll update with any news as it develops.

09 Dec 2015

Sticky Post By mcfadyena Posted in Advocacy, Morquio, MPS IVA, Uncategorized Permalink

This Is Not OK – When Answers Aren’t Really Answers