B.C. health minister promises decision on potentially life-changing drug coming 'soon'
B.C. lags behind 4 other provinces now paying for expensive drug to treat degenerative disease
Like most 13 year olds, Miles Ambridge loves super hero movies.
But unlike others his age, he doesn't wish he had super powers. He says he just wants to be able to walk one day. Or at the very least, crawl.
Ambridge says he's fighting two foes keeping him dependent on his electric wheelchair; spinal muscular atrophy (SMA), a rare neuro-muscular disease that's slowly robbing him of his strength — and the B.C. Health Ministry, which has yet to give him access to an exorbitantly expensive drug, Spinraza, that could arrest or reverse his decline.
"I'm pretty much at the brink of losing anything I can do independently," says Ambridge. "In the future, what will it be like if I don't get this? Sometimes I imagine it could be pretty bad."
His mother, Anne Belanger, is blunt.
"This is life saving and life changing. We need this now," said Belanger, 51.
B.C.'s health minister, when pressed on Miles' case Friday, said medical experts are looking into how Spinraza should be covered in the province — adding he expected a decision "soon."
Health Minister Adrian Dix on B.C.'s look at expanding Spinraza coverage:
$118,000 a shot
The problem: Spinraza is one of the most expensive drugs in the world.
Its U.S. manufacturer, BioGen, normally charges $118,000 a shot; $708,000 for the first year of treatment, $354,000 for each subsequent year.
Studies show the drug is effective in improving mobility for 40 to 50 per cent of SMA sufferers, but its benefits may decrease with increasing age.
There are about 30 people in B.C. with varying degrees of the disease.
Last October, the province approved the use of Spinraza to treat Type 1 SMA, its most severe form. It's normally detected in babies within their first seven months and they rarely live beyond two years — so B.C.'s commitment was limited.
Other provinces provide drug
But Ambridge has Type II SMA, a slower developing form of the disease.
If he lived in four other provinces, he would be eligible for the drug.
Quebec pays for Spinraza for all of its citizens with SMA, with no age caps.
Saskatchewan and recently Ontario (June 12) and Alberta (June 18) announced they would extend drug treatment to a broad range of sufferers up to age 18.
Those provinces are believed to have negotiated lower costs for Spinraza.
B.C., however, has yet to announce expanded availability of the treatment.
CBC News requested an interview with Health Minister Adrian Dix, but was told he wasn't available.
When questioned at a Friday press conference, Dix reiterated the "enormous" cost of Spinraza and emphasized that experts are looking into covering it.
"That advice will come forward and a decision will be made," Dix said. "I expect it to be made soon."
Health Minister Adrian Dix explains why provinces grapple with expensive drug prices:
Last October, Dix called the drug "life changing."
At the time, he wouldn't comment on the cost to the province, but said it would be in the "millions."
'We live in the wrong postal code'
Belanger, when presented with Dix's comments Friday, said "soon" isn't fast enough.
"The evidence is there that this is life saving and life changing so I'm uncertain what other evidence there is to review and ponder," she said.
"This is a progressive disease and every day counts."
Miles Ambridge said he just wants to stop the disease from robbing him of what little movement he has.
"I remember I could raise my arms when I was four. Now I can't," he said. "My breathing has probably gotten ... worse as well. I feel like that also kind of degrades your appetite, slowly takes it away."
His mother said trying to maintain his 27 kilogram (60 lb) weight is increasingly a struggle — and denying Spinraza for her son, is "heart wrenching."
"Miles is not getting it because we live in the wrong postal code. Every single day since he was diagnosed …he's getting worse," she said, fighting back tears. "And we can't do anything. Our hands are tied."
'Extremely angry': Cure SMA Canada
The executive director of Cure SMA Canada, Susi Vander Wyk of Chilliwack, said she's "extremely angry" B.C. hasn't extended the availability of Spinraza.
She has a 22 year old daughter with the disease.
"Now knowing that there's treatment that we can't access is far more painful than anything we've ever done," said Vander Wyk.
Dr. Alex MacKenzie, a pioneer in the treatment of SMA at the Children's Hospital of Eastern Ontario, said all indications are the drug stops the progress of the disease, even in older patients.
"I completely get the fact that there's only so many health dollars to go around," said Dr. MacKenzie. "But from my unbiased patients' advocacy, I think it would be great if (Ambridge) could get it."
'Our only hope'
Ambridge said he no longer wakes up in the morning hoping B.C.'s health minister will provide greater access to Spinraza.
"I give up on the dude," said the teen.
But his mother isn't giving up.
"This is a human right," said Belanger.
"This is the only treatment available. We have no other option. This is our only hope."
With files from Paisley Woodward and Liam Britten