Costly drug crucial for teen
Trevor Paré and his family are afraid that come May, they won’t be able to afford the drug that’s keeping him alive.
The Innisfail teen is one of only a handful of Canadians with Pompe Disease, meaning he was born without the enzyme that metabolizes glycogen (starch) in cells.
Without that enzyme, starch builds up on a person’s muscles, causing serious damage and eventually death.
For the last four years, the 17-year-old has been part of clinic trials for the enzyme replacement drug Myozyme.
Since taking the drug, Trevor, who uses an electric wheelchair, said he has seen dramatic improvements.
“It gives me a lot more energy,” he said. “Before, I had a hard time holding my neck. Now, I’m able to move my neck better and my arms, too.”
But the clinical trial that Trevor is a part of ends in May and the drug that is saving his life is not covered by Alberta Health and Wellness.
This means that continued treatment will cost the Parés about $500,000 a year.
Every two weeks, Trevor travels to Alberta Children’s Hospital in Calgary to receive the drug through an IV.
“It would basically take all 20 members of our extended family working full time and all living under one roof, for us to afford the drug,” Trevor’s mom Linda said. “It’s not mathematically possible and we don’t know what to do.”
The Canadian Organization of Rare Disorders (CORD) has dubbed today, the rare Feb. 29, Rare Disease Day.
The organization is asking the government to cover the medication to help people like Trevor with rare diseases.
Trevor said sharing his story is important because “when there’s not a lot of people with a disease, they don’t get as much attention.”
Trevor’s mom said if the medication had been available when he son was born, she believes he would be still walking today.
“When he was first diagnosed, we were told to take him home and love him because he wouldn’t live long,” she said. “But he just kept fighting and now there is this drug that can help him and we’re basically being told we can’t have it.”
Dr. Robin Casey, medical director of the inherited metabolic disorders program at Alberta Children’s Hospital, said prior to the drug there was very little doctors could do for patients like Trevor.
He said Myozyme doesn’t appear to reverse muscle damage already sustained from excess starch, but it does seem to slow further deterioration.
The cost of treatment, he said, is “beyond the means of virtually all the patients” he treats.
In June 2007, about two years after Health Canada approved Myozyme for sale, the national Common Drug Review committee recommended provinces pay for the medication for patients with Pompe who begin showing a weakening of the heart muscle before they turn one.
Mike Tierney, vice-president of the Common Drug Review with the Canadian Agency for Drugs and Technologies in Health, said while someone living to be 17 with Pompe is rare, the committee’s recommendations suggest the province would pay for Paré’s treatment since his heart began weakening before he was one.
Alberta Health and Wellness has not decided whether it will cover the drug.
“We are currently reviewing how Myozyme most appropriately fits into our drug treatment programs. No decision has been made,” spokesperson Howard May said in an e-mail.
May said there is “no timeline” as to when the government will make its decision.
For the Parés, the decision can’t come soon enough.
“Doctors have told us that in three months (after Trevor stops taking the drug), he will be on a respirator and in six months, he will be dead,” Linda said.
Contact Ashley Joannou at ajoannou@reddeeradvocate.com
