Brothers with rare condition trying experimental treatment in Toronto
Published Tuesday, December 24, 2013 2:19PM EST
Two brothers from the Wingham area, diagnosed with a rare disease, are hoping to add some years to their lives.
The teenagers, Zane and Luke Braun, travel to Toronto every two weeks – and have been doing so for the past two years – for an experimental treatment for Morquio syndrome, a disease that causes dwarfism, heart and lung issues and pain and discomfort.
The treatments are working, but are not likely to reverse the effects of their incurable condition.
Zane, 14, and Lucas, 16, have had 11 surgeries between them.
“They see nine specialists at Sick Kids in Toronto. We made 72 trips to Toronto last year,” says their mother, Debbie Braun.
While most of those trips over the past decade have been to deal with the ongoing problems, the past two years have been spent trying to find a remedy for their deteriorating condition.
The experimental treatment involves a new drug called vimizim. It is meant to replace the enzymes they’re missing, which break down complex sugars in their body. It's a six-hour treatment once a week they’ve undergone for the past 121 weeks, but it's finally working.
“I’ve noticed big changes in them; how they feel. They're not falling asleep like they used to. They’re breathing tests are better," Braun says.
Five other patients in Toronto are on the drug and many more across Canada and the U.S. are trying it.
“What we've seen in the research trials is that it has improved mobility, endurance and energy levels in participants. It has also decreased the excess sugars in the body and hopefully will slow down the progression of the disease," says Julian Raiman, a metabolic geneticist at The Hospital for Sick Children.
Vimizim is not a cure, but it's hopefully buying time for Zane and Luke. The life expectancy for Morquio patients is between 20 and 40 years.
“It's slowing the progression of our disability down. It’s not really helping us, but keeping our problems to a minimum,” says Lucas
Approval for the drug is now in the hands of Health Canada and the FDA. It could take more than three years for that approval. The boys will stay on the drug until a government decision is made.
“I don't want anyone to go through what I’ve gone through for the past 12 years,” says Zane. I couldn't imagine what it would be like right now to be sitting in a wheelchair doing nothing. I can't imagine that."
Neither can their mom.
“I'd drive to the moon and back if it meant one more day with you. I think I have put that many miles on the van. But if it means one more day. That's what we're doing it for. We'd like other families with Morquio to have the same opportunities we've had, too."