The following is the third and final part 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. Thursday’s story focuses on the McFadyen family’s advocacy for other children diagnosed with the disease. Today’s story announces their lofty new fundraising project.
CAMPBELLFORD — Today is the launch of a million-dollar project for The Isaac Foundation.
That is what this story is supposed to be about.
First, though, it’s worth updating Thursday’s story which talked about the McFadyen family from Campbellford and their advocacy for provincial funding for life-sustaining treatment for children who suffer from the rare disease MPS VI.
Andrew and Ellen McFadyen’s eight-year-old son Isaac has the disease, which is caused by an enzyme deficiency. As previously reported, there is a treatment — Enzyme Replacement Therapy — available through the country’s Special Access Program, but it can cost up to a million dollars per year and the respective provincial governments must agree to pay for it.
When Isaac was first diagnosed years ago, the McFadyens were initially turned down, but the decision was reversed after a successful lobby effort. Last year, the Darren and Pam More from Palmerston had the same problem with their two-year-old son Jasper. With help from the McFadyens and their Isaac Foundation the Mores also eventually got funding.
This past week, the McFadyens have been advocating for Violet Revet, a three-year-old girl from Saskatchewan who was diagnosed with MPS VI seven months ago. Her parents — Jennifer Yanke and Larry Revet — had been waiting all those months to hear whether their daughter would be approved, or denied, for funding. They turned to The Isaac Foundation for help and McFayden advocated behind the scenes while public pressure through social media to the Saskatchewan government also aided the lobby effort.
Wednesday night it was announced that Violet has been approved for the funding and she will get the treatment she needs.
“I was unbelievably overcome with emotion when I got that notification that Violet was going to get covered,” Andrew McFadyen said yesterday. “It’s bittersweet to have to battle, but then to win that battle again on behalf of a family, on behalf of a child is really special to us, probably because we know how important it is for these kids to have this treatment. Being down this road for six years, I know what it means for Violet and her family.”
McFadyen expressed gratitude to those who made their voices heard by the Saskatchewan government.
“It’s one thing for one person or one family or one tiny organization to be calling governments into account. Governments can weather that storm, they can weather the storm of me calling them every day and advocating and telling them all of the different things that I want them to do on behalf of children,” McFadyen said. “They can get through that, but once there’s momentum behind it, once people are sharing the story, once people are talking about it and tweeting about it and calling into the minister’s office to express their concern…then that prompts action. We’ve been incredibly blessed that there are a lot of people who are interested in The Isaac Foundation and always looking for ways that they can give back and help. For the second straight year, this is a way they’ve been able to do that.”
Violet, like Isaac and like Jasper, will now get Enzyme Replacement Therapy.
While ERT treatment has proven to be essential, McFadyen has repeatedly stated that it’s not a cure.
Today, The Isaac Foundation is launching Project One Million. Andrew and Ellen McFadyen established the foundation in 2006 to not only raise awareness for MPS VI, but to also help fund innovative research projects.
Currently, they are ecstatic about a research project in New York City related to bone and joint disease in children affected by MPS VI.
“We’re starting to discover that the disease progression is the buildup of these complex sugars in the body and attacking the organs, but it’s also the inflammation that’s taking place in the bones and joints,” McFadyen said in an interview earlier this week. “It’s that inflammation that is leading to many of the devastating physical symptoms that our kids are seeing; the stiffening of joints, the spinal cord compression, in my son his hands are starting to become very ineffective — he has no strength and can’t make a fist. He can’t even do up the buttons of his shirt anymore.”
McFadyen said through The Isaac Foundation they focus on research projects that aim to find a cure or offer a short-term solution. The researcher in New York City has discovered oral anti-inflammatory medication that’s already on the market, already FDA approved, already approved by Health Canada.
This anti-inflammatory has already been tested on lab rats that are affected by MPS VI.
“Rats and cats develop MPS VI naturally and so they’re a really good indictor for what medications work and which medications don’t work for humans,” McFadyen said. “The progression of the disease is almost identical in these animals as it is in humans. What she’s found is this anti-inflammatory is reversing almost all of the bone and joint disease in these animals.”
A video was sent to the McFadyens showing two different rats. The untreated rat had difficulty walking, coarse facial features, matted fur, its eyes were clouded over and had still joints, “actually, very depressing to see,” McFadyen said.
As for the rat treated with the anti-inflammatory, “you would never know there’s a problem. The animal is trying to jump out of its box in the lab, it’s running around, the facial features aren’t as coarse.
“For us, we naturally looked at that and said let’s start a human clinical trial. We’re ready,” he added. “(The Isaac Foundation) funded a trial in lab animals using the anti-inflammatory in conjunction with ERT just so we know that it’s safe and those early results show that it’s very safe and no change in the blood chemistry.”
Of course, it’s not as simple as just starting the human trial.
“We need a million dollars,” McFadyen said. “This number continues to come up in my life. A million dollars a year to fund to fund my child’s treatment. Now we need a million dollars to do a human clinical trial.”
Pharmaceutical companies have said they not interested in funding the trials, McFadyen said. So once again, the McFadyens are taking it upon themselves to make something happen.
The result: Project One Million.
“Our hope is that we can start a million conversations about rare diseases across the world,” McFadyen said. “We want to have a million people view our video, we want a million likes and shares (on Facebook), a million tweets (on Twitter), and perhaps we can inspire a million people to donate $1 each so that we can fund this clinical trial.”
McFadyen said he felt the world of MPS shift a bit last month when the results of this study were presented.
“It will only enhance the quality of life for these kids,” he said, while expanding using Isaac as an example.
“We have every hope that his hands will begin to work again, and that he’ll be able to raise his arms up above his head, that his stamina will increase because the tightness in his hips will decrease, that there won’t be any more danger of spinal cord compression in him. The hope for this drug is off the charts.”
One million dollars is a lot of money to raise and that fact hasn’t been lost on the McFadyens.
“We know we have lofty goals, but if we just sit around and wait for something to happen, we’re never going to accomplish anything,” he said.
They will get some help, including from international music star John Mayer, who is a big supporter of Isaac’s. He’s met with the McFadyens several times prior to concerts in Toronto.
“We’re lucky that musician John Mayer is going to help get this video out,” McFadyen said. “He’s going to share it on his blog online and ask people to go and view it, and that’s a big boost. He’s got a lot of followers online.”
For instance, if you go to Mayer’s Facebook page, he has well over 5,300,000 ‘likes’.
They are also taking a ‘Share It Forward’ approach. They would like everyone to share the video and information about Project One Million, but they’ve also specifically identified 16 of the most influential social media celebrities, like Ellen DeGeneres, Johnny Depp and Anderson Cooper.
“The idea is the first celebrity that shares it, in this case John Mayer, will not only share it with the people that follow him online, but he’ll also share it forward to the next person in our chain (of celebrities) ask to send it forward,” McFadyen said. “We don’t know how far we can have this chain go, perhaps it stops at one, but maybe it goes all the way through that chain.”
McFadyen knows the million-dollar goal is a lofty one.
“We’re not naïve,” he said. “We may launch this and have 100 views and that’s it, but our business is hope and sometimes that’s all you have left.”
The website for Project One Million is www.projectonemillion.ca. The link for their video, which they hope people will watch and pass on, is http://www.youtube.com/watch?v=iPhisB8_-wM.