Patients see hope in new ALS drug not approved for sale in Canada

CTV National News: Difficult drug ALS patients hold out hope for a drug that may provide a new treatment. Avis Favaro reports.

Some Canadians diagnosed with amyotrophic lateral sclerosis, or ALS, are spending thousands of dollars to obtain a new drug from abroad they hope will slow the progress of the terminal disease.

The drug called edaravone, which is sold under the brand names Radicava and Radicut, slowed the progression of ALS, also known as Lou Gehrig's disease, by 33 per cent in a small Japanese study.

It's unclear how long the drug's benefits last because the clinical trial, funded by the Japanese drug company that makes Radicava, lasted only 24 weeks. The trial also only involved 137 patients with early-stage ALS. A small percentage of patients in the study, it should be noted, experienced adverse side effects like bruising, headache, skin inflammation, breathing problems and issues with gait.

ALS is a fatal, neurodegenerative disease. As it progresses, patients typically lose the ability to move, speak, swallow, and breathe. They usually die within three to five years of diagnosis.

Based on the results of the Japanese study, the U.S. Food and Drug Administration approved the use of edaravone to treat ALS patients in May. But the drug has not been approved for sale in Canada.

Terri Bishop, a Truro, N.S. woman who was diagnosed with ALS in March, 2016, travelled to Japan this year to receive edaravone infusions. She and her husband Bruce spent more than $4,000, plus travel costs, to get an eight-month supply of the drug.

Bishop is now being infused with the drug at her local hospital. The infusions take an hour and have to be done for two weeks each month, with two weeks of rest in between.

"When you have ALS and life expectancy is three to five years…to us it is not crazy, it is hope," Bishop told CTV News.

She believes the drug is helping her. After the first few days of treatment in Japan, she said, she was able to open up a pill bottle and do up her zipper -- simple tasks she previously couldn't do.

Bishop's husband said he also noticed a slowdown in the progression of his wife's symptoms.

"To have something like this happen totally shocked us," he said.

Edaravone, which was initially developed to treat stroke victims, is the first promising drug for ALS approved by the FDA in more than two decades. One Canadian expert said the medication seems promising, even though it's not a cure.

"Although the study is very small…the fact that we appear to have a new weapon against this disease is incredibly encouraging," Dr. Lorne Zinman, a neurologist at the Sunnybrook Health Sciences Centre, told CTV News.

He also believes edaravone is a "step in the right direction" that will promote the development of "newer and better medications" for ALS patients in the future.

Jeff Perrault, an ALS patient who lives in Sudbury, Ont., has asked friends travelling to Tokyo to buy edaravone for him.

He's confident the drug is safe and feels "good" about taking it, but he was diagnosed with ALS three years ago and is not sure how effective it will be for someone like him. Still, he said, all ALS patients have the right to try new treatments.

Some worry that, even if Health Canada does approve edaravone, the drug will be too expensive for most patients. A year's worth of the drug currently costs more than $145,000 in the U.S. In Japan, it's a more reasonable $4,000-$5,000 for a year of infusions.

Dr. Zinman said that, while some people have flown to Japan to get the drug themselves, that option is out of reach for most ALS patients, who don't have the money or aren't well enough to travel.

"I think it is important for the ALS community to come together in Canada and do everything we can to try and get this drug approved in Canada as quickly as possible," he said.

Since their trip to Japan, Terri and Bruce Bishop have been able to organize a system for getting edaravone from Japan to Canada via Japanese neurologist Dr. Hiide Yoshino, who was involved in the clinical trials.

Health Canada allows patients to import the medication for personal use.

With files from CTV's medical specialist Avis Favaro and Elizabeth St. Philip