An Abbotsford, B.C., family is sharing the success of a clinical trial that helped save their child in the hopes that a new program from BC Children’s Hospital will give others better access to similar studies.
In August 2015, Emmett was only a few months old when he was diagnosed with spinal muscular atrophy, a disease that impacts signals between the brain and muscles.
Without treatment, his life expectancy would have only been a few years, but with the help of BC Children’s Hospital, his family was able to start him on the drug Spinraza.
“It was some obviously earth-shattering for us,” Josh Willms, Emmett’s dad told Global News of the diagnosis.
“After the diagnosis, that same day, we met another neurologist who talked about this potential clinical trial that he might be eligible for.”
The family decided to give Emmett a chance on the clinical trial. He is now eight years old and the drug is still working.
“It’s been nothing short of a miracle drug for us and for him. It’s been it’s been really amazing,” Willms said.
He said Emmett plays power soccer and is able to be in a manual or power wheelchair at home.
He added he knows that without this clinical trial, his son’s life would have been very different.
“It’s really one of those things that that we’ve been so fortunate to connect with BC Children’s and this clinical trial and since then the clinical trial has ended and the drug has been approved for other kids with SMA to access across the province and across Canada,” Willms added.
“So it’s very exciting.”
The B.C. government announced in October 2018 that it would fund Spinraza for babies seven months old or younger.
The medication comes at an enormous cost as each dose of Spinraza costs $100,000. The first year of treatment can cost more than $700,000, and $350,000 every year after that.
“The staff at B.C. Children’s have been so amazing, so supportive and have continued to just be with us every step of the way, giving us the information we need,” Willms said.
The hospital is now hoping to help other families in similar situations by creating a clinical trial super hub.
“We’ve always known that clinical trials are important to bring therapeutic options to kids who are facing devastating conditions and no treatment options,” Quynh Doan, senior executive director for the BC Children’s Hospital research institute told Global News.
“But each trial had to go through a series of processes to be able to be conducted here. And that’s both expensive and challenging.”
Doan said the clinical trial’s super hub will build infrastructure so that as soon as an invitation to participate in a trial is extended, the child will be ready to enroll, take the medication and benefit from it.
“That will also encourage our local investigators to bring their research ideas to the clinical trial forum and find those treatments that can become the standard of care,” she added.
“Hopefully we will become the hub for not just B.C. but also the west of Canada and bring kids who need access to clinical trials to our partners.”
Doan said the budget to get the clinical trial hub set up to be sustainable after five years is $12.4 million.
They already raised $4.2 million at the Crystal Ball Gala last weekend and that will launch the super hub.
“We’ve already got some staff lined up,” Doan added. “We’re going to launch a super hub and start operating in the new year.”
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