mps iv

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.”

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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.

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

LOCAL CHARITY CELEBRATES 10 YEAR ANNIVERSARY

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