British Columbia

B.C. teen with rare disease wins fight to receive costly drug from province

Miles Ambridge, 14, who has spinal muscular atrophy, has finally won his fight with the B.C. Health Ministry to get access to one of the most expensive drugs in the world.

Health ministry won't explain change of heart in granting Miles Ambridge treatment for spinal muscular atrophy

Miles Ambridge, 14, and his mother Anne Belanger, 52, hope the gene therapy drug will stop or reverse the devastating effects of spinal muscular atrophy. (Mael Thebault/CBC)

Miles Ambridge greeted news he would finally receive a life-changing drug with a couple of fist pumps — the most he could manage from his motorized wheelchair.

Ambridge, 14, has spinal muscular atrophy (SMA), a rare neuromuscular disease that's slowly robbing him of strength.

He can't walk and is losing the power to use his arms. Breathing and swallowing are getting more difficult.

But some of that might be about to change.

Ambridge has finally won his fight with B.C.'s Ministry of Health to get access to Spinraza, one of the most expensive drugs in the world, which is shown to slow or reverse SMA in up to 50 per cent of young patients.

"When I heard, I was like, oh my God, finally! It's about time," says Ambridge. "It was super exciting and I was amazed. I was happy and I was relieved."

His father says the drug approval is still sinking in.

"We're thrilled but it's kind of like, almost, you know, slaying the dragon after so many years, where you just go numb as well," says Don Ambridge.

Miles's mother says she cried "tears of joy, relief and happiness."

"We never gave up hope," says Anne Belanger.

In the five months it's taken to win his appeal to receive Spinraza, Miles Ambridge has lost more of his motor skills. (Harman/CBC)

$700K for 1st year of treatment

It's been a long battle stretching back to 2017, when Health Canada approved the use of the U.S.-manufactured gene therapy drug.

B.C. lagged behind other provinces that agreed to pay Spinraza's exorbitant cost: $118,000 a shot, more than $700,000 for the first intense year of treatment, and $350,000 for each subsequent year.

It's not known if B.C., like some jurisdictions, has been able to negotiate a lower price with Biogen, the Massachusetts-based drug maker.

In October, the province approved Spinraza to treat SMA Type 1, the most common and severe form that's normally fatal in infants by age two.

In December, B.C. extended the drug's availability to those with Type 2, a slower developing form of the disorder, but limited treatment to children 12 and under.

Miles, at 14 years of age, was denied access — and appealed.

B.C.'s health ministry won't say why it's now had a change of heart, citing patient confidentiality. But it issued a general explanation.

"Requests for this expensive treatment are reviewed on a case-by-case basis through B.C.'s Expensive Drugs for Rare Diseases process," a ministry spokesperson wrote in an email.

There are about 30 people in B.C. with varying degrees of SMA.

COVID-19 complications

Ambridge says he almost gave up the fight when COVID-19 hit, knowing the health ministry would be consumed with that crisis.

"That's just the point where I kind of lost pretty much all hope and that's where I didn't really want to be patient and stay optimistic," says Ambridge.

"I knew that would get in the way and it did for a while." 

Miles Ambridge is lifted from his bed by his mother. The teenager is unable to move his legs and is slowly losing the use of his arms. (Mael Thebault/CBC)

The five months since the drug was approved but denied to Miles has taken a toll on the teenager, his parents say.

His father says he has watched his son deteriorate.

"He was no longer able to play one of his favourite [video] games because his arms weakened," Ambridge says. "He couldn't react quick enough … That was painful to bear."

He believes at the very least, SMA will be kept at bay by Spinraza. Any slight improvement, even the wiggling of a toe, will be a major victory.

"These sound so small, I think, to some some folks, but [to us] they're massive wins," he says.

'I hope most of the impact ... is on my arms'

Miles says the drug approval has given him hope for the future.

"I would be happy knowing that I'm not losing strength and I'm only gaining it," he says.

"I hope most of the impact of Spinraza is on my arms so I can use them more and then therefore I can be more independent and do more things I want to do." 

His mother has similar dreams.

"[That] he'll be able to stay healthy, strong, capable of living what is normal life," says Belanger. "He'll be able to tackle a job and graduate high school and, you know, do what every 14-year-old should be doing."

'Thank you for the approval'

In the past, Miles blamed B.C. Health Minister Adrian Dix for the approval delay, at one point saying, "I give up on the dude."

Now, he has a new message for Dix.

"Thank you for the approval," says the teenager.

B.C. Health Minister Adrian Dix has said he doesn't decide who gets Spinraza; that decision is made by a review committee. (Mike McArthur/CBC)

But the teen and his family vow they won't give up the fight to push the health ministry to make Spinraza available to everyone with SMA.

"Try to approve Spinraza for as many people as you can, while you handle COVID," Miles urges.

He also has advice for others fighting the crippling disorder: "Stay optimistic and stay patient."

CBC Vancouver's Impact Team investigates and reports on stories that impact people in their local community and strives to hold individuals, institutions and organizations to account. If you have a story for us, email impact@cbc.ca.

 

About the Author

Eric Rankin

Investigative journalist

Eric Rankin is an award-winning CBC reporter. His honours include the 2018 Canadian Screen Award for Best Local Reportage, the 2017 and 2015 RTDNA awards for Best In-depth/Investigative Reporting, and the 2009 Jack Webster award for Best News Reporting.

Comments

To encourage thoughtful and respectful conversations, first and last names will appear with each submission to CBC/Radio-Canada's online communities (except in children and youth-oriented communities). Pseudonyms will no longer be permitted.

By submitting a comment, you accept that CBC has the right to reproduce and publish that comment in whole or in part, in any manner CBC chooses. Please note that CBC does not endorse the opinions expressed in comments. Comments on this story are moderated according to our Submission Guidelines. Comments are welcome while open. We reserve the right to close comments at any time.

now