NORTHUMBERLAND – Government funding for live-sustaining treatment for MPS VI (Maroteaux Lamy Syndrome) is already extremely important. The only thing better would be finding a cure for the rare enzyme deficiency disease.
That was only reinforced to the McFadyen family of Campbellford last month when they helped another Ontario family lobby the provincial government for the same funding their own son receives to help battle MPS VI.
Seven-year-old Isaac McFadyen — the son of Andrew and Ellen and brother of Gabriel — is one of eight confirmed cases in Canada (and 1,100 worldwide) who have the progressive disease. Symptoms include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span. The McFadyens established The Isaac Foundation in 2006.
There is no known cure yet for MPS VI, but there is the life-sustaining treatment available called Naglazyme, which is an Enzyme Replacement Therapy designed to provide patients with a synthetic version of the enzyme they are lacking. Small doses are infused into the patient’s bloodstream.
Isaac has been receiving the treatment once a week for five years at the Hospital for Sick Children in Toronto. The cost of the treatment, produced by Biomarin, can range from $300,000 for a small individual to $1 million for a young adult each year. The McFadyens lobbied the provincial government to get funding for the treatment and were ultimately successful.
In August, they helped a Palmerston family do the same. Darren and Pam More’s two-year-old son, Jasper, was diagnosed with MPS VI six weeks ago. Within days of being notified they were denied funding for the treatment, the provincial government reversed its decision.
Both families are grateful for the funding as they aim to find a cure.
The Mores will be attending the second annual Gala For A Cure, presented by The Isaac Foundation, Saturday, Oct. 1 at the Best Western in Cobourg.
Tickets are $100 (receive a $50 tax receipt) and will feature a special intimate musical performance by Juno Award winner Sarah Harmer, as well as wine tasting, dinner and a silent auction.
“Knowing that we desperately need to find a cure for these children makes these events that much more important and that much more special for everyone,” Andrew McFadyen said. “Knowing that every dollar goes directly into research is very important as well.
It’s also important to raise awareness of the disease.
“The last five years of us raising awareness was very crucial in helping Jasper get treatment,” McFadyen said.
Last year’s inaugural gala raised $20,000 and went towards research on behalf of The Isaac Foundation. The success was from those who bought tickets to the event as well as donated items to the silent auction.
“For me it was the community feeling that was there,” McFadyen said. “This sense of caring and wanting to help out. With it being such an intimate evening, I felt it was really special for everyone.”
Singer/songwriter Ron Sexsmith helped make last year’s gala a special evening, McFadyen noted, and he expects Harmer will help create a similar atmosphere. The McFadyens are thrilled to have her be a part of the event. They are friends with The Tragically Hip, who are friends with Harmer, and they also received help from Sexsmith’s wife in bring Harmer to this year’s gala.
“There’s a lot of connections that Sarah and we already had, we just needed to connect the dots in order to make it work,” McFadyen said. “When she looked at the website and learned about The Isaac Foundation, she was more than happy to come and pitch in and be a part of this. We were really touched when we got the call saying yes she’s going to be there and perform, and she’s going to spend the whole night with us.”
Anyone wishing to purchase tickets, or donate items for the silent auction, can contact Andrew by phone at 613-328- 9136 and e-mail at email@example.com or Ellen at 705-632-0983 and firstname.lastname@example.org.
Tickets are limited.