A Calgary boy battling a rare disease is one of only four Canadians granted access to a special drug — but it will cost him up to $400,000 a year so his parents are turning to crowd-funding to try to cover it.
Eli Wilson, 7, has Duchenne muscular dystrophy (DMD), a degenerative and fatal disease affecting mostly boys. Because it stops the body from producing a key protein needed by muscle cells, he will eventually lose the ability to walk, to use his arms, even to breathe.
"It's not a death. But it's a death of hopes and dreams for our son," said Eli's mom, Jennifer Klazek.
Knowing the disease would slowly rob their son of his independence, forcing him into a wheelchair and eventually onto a ventilator, Klazek and her husband have devoted their energy to searching for promising new treatments.
"Our goal is to ensure that Eli has as regular a life as possible," said Ken Wilson, Eli's dad.
"That he can learn to ride a bike, that he can go to the mall with his buddies. Whatever that looks like, we want to ensure that his life is as normal as possible."
New hope for boys with DMD
Studies show a new drug, called Translarna, may help slow the progression of the disease in boys who are just starting to experience symptoms. It is conditionally approved in the European Union and is sold in 18 countries.
"As a parent of a child with Duchenne muscular dystrophy today, hope is really all you have," said Wilson. "This drug showed hope. It showed promise."
But Translarna is still under review by Health Canada and is not approved for use in this country.
With the help of Dr. Jean Mah, associate professor of pediatric neurology at the University of Calgary, Eli is now one of four Canadians receiving the drug through Health Canada's special access program..
"From month-to-month, year-to-year, boys affected with this condition, they deteriorate," said Mah.
"That's why I could understand ... how important it is for families with genetic diseases such as muscular dystrophy, to be on the look out and to hope for any new development, new potential treatments to make a difference in their child's life"
Treatment is cost prohibitive
The first shipment of Eli's drug arrived recently, but it comes with a hefty price tag. According to PTC Therapeutics, the company that manufactures Translarna, a year's supply of the drug can cost more than $400,000.
"It's a huge financial hardship," said Wilson. "What family can afford that? Without insurance programs, without government programs, these rare disease drugs are inaccessible. So that's when you take matters into your own hands."
Determined to do what they can to offer their son some hope and to prolong his life, Wilson and Klazek have launched a crowdfunding campaign to raise money for their son's treatment which has raised thousands already.
"The support has been overwhelming in helping us make this possible for our son, and we'll just take this one day at a time."