B.C. funding decision on pricey drug leaves teen with rare disease facing 'scary' future

B.C. has expanded its coverage of an exorbitantly expensive drug that could make a huge difference for Miles Ambridge's future, butat 14 years old, he's just a bit too old to qualify.

Ambridge has a rare neuromuscular disease called spinal muscular atrophy (SMA), a progressive condition that is slowly robbing him of his strength and his ability to move and breathe.

A drug called Spinraza has been shown to slow the disease's progress andreverse its effectsin up to half of SMA patients, but just before Christmas,the B.C. government announced its coverage for Ambridge's version of the disease will only include patients up to the age of 12, a much tighter age limitation than in other provinces.

"It's beyond me and it's baffling," the New Westminster teen told CBCNews. "It really makes you want to punch a hole into the wall and scream, mentally and internally, and I wouldn't be surprised if it makes people really depressed and sad inside."

Ambridge uses a wheelchair and can barely move his legs or feet. Over the years, he's lost most of the ability to lift his arms, and he can feel his lungs weakening.

"It is life-limiting and that's the scary part," he said. "We don't know how long it'll take for me to lose the strength of my arms or my voice."

Ambridge had been waiting for years to hear if he'll have access to Spinraza, one of the most expensive drugs in the world. Health Canada approved the drug for SMA treatment in 2017, when Ambridge was 12 still young enough to meet B.C.'s newly announced guidelines.

Spinraza's U.S. manufacturer, BioGen, normally charges $118,000 a shot; $708,000 for the first year of treatment and $354,000 for each subsequent year.

B.C. patients with the most severe form of SMA, Type I, have been eligible for coverage since November 2018. Type I is normally detected in babies within their first seven months, and they rarely live beyond two yearswithout treatment. The drug can help extend their lives.

Ambridge has Type II SMA, a form of the disease that develops relatively slowly. B.C.'s expanded Spinraza coverage only includes Type II patients if they're infants without symptoms who've been diagnosed genetically,and children under 12 who've never been able to walk.

Most Type II patients survive into adulthood with proper care.

There's no coverage at all for patients with Type III, the mildest form of the disease.

'It's devastating'

Susi Vander Wyk, president of the advocacy group Cure SMA Canada, pointed out that most other provinces coverSpinraza for all SMA patients under the age of 18, and make exceptions for some older patients.

"It's devastating," Vander Wyk said of B.C.'s funding decision. "I was able to at least meet and give a patient's perspective to [government in] all other provinces. However in B.C., my own province I was not allowed to meet with the government."

Because he's a teenager, Ambridge still has a chance of accessing Spinraza. B.C. says that patients between the ages of 13 and 18may be considered "on an exceptional case-by-case basis." It's not clear what criteria the province will use to decide who qualifies, but a Health Ministry spokesperson said it could include patients like Ambridge who are just over the age cut-off.

Holli Vander Wyk, Susi's daughter, won't have that opportunity.

At 23 years old, her only chance would be moving to another province. That would mean uprooting her life in the middle of her studies at the University of British Columbia'sOkanagancampus, and moving even farther away from her family in Chilliwack.

She said she has friends across the country with SMA, and the majority of them have access to Spinraza.

"They're gaining strength, they're gaining energy. They feel like they can do more in a day. I know one individual that whenever they get the injection, they call it their Spinraza glow," Vander Wyk said.

"I feel very happy for them but it also makes me sad because I know that I don't have the opportunity to have access to the drug myself."

Health Ministry responds

There are about 30 people in B.C. with the disease.

Studies show Spinraza improves strength and mobility for between 40 and 50 per cent of SMA patients, but the benefits may be reduced as the age of the patient increases.

Health Minister Adrian Dix was not available for an interview, but in an email last month, a ministry spokesperson said the coverage decision is in line with the recommendations of the Canadian Agency for Drugs and Technologies in Health, an independent body that provides governments with advice on health-care decisions.

Ambridge's mother, Anne Belanger, is president of the B.C. chapter of Cure SMA Canada, and she questions the ministry's decision to release the bad news just before the holidays.

"I think it's appalling shows an absolute lack of empathy and compassion," she said.

As for Ambridge, he dreams of working as an anesthesiologist or a lawyer one day, but worries that without Spinraza, his condition will make that impossible. He's furious with the health minister for that.

"He clearly does not understand the value in this, and it's truly f--king heinous," Ambridge said.