2 Alberta toddlers receive life-saving drug thanks to angel donor and pharmaceutical lottery

Health Matters December 3: Two Edmonton-area children, suffering from spinal muscular atrophy Type 1 and in need of a $2.8 million life-saving Zolgensma treatment, have finally received it — thanks to a twist of fate. And the winner of the Mighty Millions Lottery grand prize is revealed. Su-Ling Goh reports.

Two Alberta families are celebrating early Christmas miracles this week.

On Thursday, two-year-old Kaysen Martin received the life-saving drug Zolgensma thanks to donations from around the world and an extraordinary gift from an anonymous donor.

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“We did it! You did it! We all as a community did it! Right now Kaysen is receiving Zolgensma,” the family shared in a post on Instagram on Thursday.

“We are so blessed that along with all the funds raised, an angel donor donated the rest of the money so that we could privately purchase Zolgensma for our sweet Kaysen!

“We are still in such a state of shock as every single day that passed after Kaysen’s second birthday we were slowly losing all hope. Well just as all hope was lost, an angel swooped upon us and made all our dreams and hopes come true for Kaysen.

“We are forever grateful and so thankful for everyone who stood by our side. Miracles do happen.”

Kaysen was diagnosed with spinal muscular atrophy Type 1 (SMA 1) when he was eight months old. It’s a progressive motor neuron disease that causes his muscles to weaken over time.

Most children with SMA 1 do not live to see their second birthdays.

Kaysen Martin, who has spinal muscular atrophy Type 1 (SMA 1), in Calgary Children's Hospital Dec. 3, 2020.

Kaysen Martin, who has spinal muscular atrophy Type 1 (SMA 1), in Calgary Children's Hospital Dec. 3, 2020.

Courtesy: Lana Martin

Kaysen has been taking Spinraza treatments every four months. But Spinraza is not a cure. It must be administered in hospital and it costs $375,000 a year.

His family asked Alberta Health to pay for a new one-time gene-therapy treatment called Zolgensma, which costs $2.8 million.

It is not funded by Health Canada.

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Kaysen’s Quest inspired Edmontonians, Albertans, Canadians — even celebrities like actor Ryan Reynolds — to donate.

A gofundme for Kaysen had raised over $1.4 million. Then, an anonymous donor contacted them through Cure SMA Canada and offered to pay for the rest.

“In late October, we got word that the angel donor wanted to top up our bank account to get Kaysen the medication,” his mom, Lana Martin, told Global News.

“It was just immediate tears but also shock,” she said. “It was coming down to the point where we were trying to wrap our head around it not happening for Kaysen.”

Kaysen Martin, who has spinal muscular atrophy Type 1 (SMA 1), with his mom and dad in Calgary Children's Hospital Dec. 3, 2020.

Kaysen Martin, who has spinal muscular atrophy Type 1 (SMA 1), with his mom and dad in Calgary Children's Hospital Dec. 3, 2020.

Courtesy: Lana Martin

The family was under the impression that Kaysen would have to be under two years in order to receive the drug. However, because the drug has been approved for use by weight (under 20 kilograms) in other countries, his doctor in Calgary was able to proceed.

It took several weeks to secure the private purchase of Zolgensma and have the transfusion approved in Alberta.

“It’s honestly a miracle,” Lana said. “A miracle.

“There’s good people out there who don’t want an ounce of credit for what they’ve done for Kaysen.”

Three other Alberta families have been trying to get the same costly treatment for their children.

Harper Hanki from Spruce Grove, Max Sych from Fairview, and Reign Johnston all have spinal muscular atrophy.

Harper — who is 16 months old — has Type 1. Max — who will turn two on Jan. 11 — has Type 2. Reign — who turns two on March 5 — also has Type 1.

Read more:
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The same anonymous donor also offered to pay for Harper Hanki’s treatment.

But about two weeks after the generous offer, Harper won a lottery being run by a pharmaceutical company to receive the multi-million-dollar treatment.

“We were already celebrating and then we got to celebrate even more,” Harper’s dad, John Hanki, said.

“The first thing we did was call the organization the donor was going through and said: ‘Could you please pass it on to the next family?’

“To find out they did pass it on to another kid is just unbelievable, especially one so close to home.”

In fact, the two families have known each other since Harper was diagnosed.

“Lana and I have fought for this for months, from the very beginning,” Harper’s mom, Amanda Hanki, said. “To see both our kids cross the finish line together was always a dream of ours and it’s so surreal.”

Harper Hanki with her dad John and mom Amanda. The Spruce Grove toddler has spinal muscular atrophy. Dec. 1, 2020.

Harper Hanki with her dad John and mom Amanda. The Spruce Grove toddler has spinal muscular atrophy. Dec. 1, 2020.

Courtesy: Amanda Hanki

Harper received her 60-minute IV gene-therapy treatment on Tuesday.

“This is new chapter for Harper,” John said. “This last year has been a struggle… Now we can look forward to everything else she can do.”

The couple said they’re looking forward to Harper’s first steps and watching her grow stronger every day.

“We’re really excited for the days, months and years to come,” Amanda said.

“We couldn’t be happier. This is exactly what we prayed. It’s truly a miracle that this happened.”

Harper Hanki, held by her dad John, receives her 60-minute IV treatment of Zolgensma, a gene therapy treatment for spinal muscular atrophy Type 1.

Harper Hanki, held by her dad John, receives her 60-minute IV treatment of Zolgensma, a gene therapy treatment for spinal muscular atrophy Type 1.

Courtesy: Amanda Hanki

Lana said they couldn’t have done it without the support of the community.

“Every dollar that was raised by the community did go towards the purchase of Zolgensma,” she said.

“I don’t know how to thank them. I don’t know how to put into words that the hope they’ve put into us that we’d lost.”

She hopes Kaysen has raised awareness about spinal muscular atrophy and helped pave the way for other kids like him. The Martins believe Health Canada’s approval of Zolgensma is “just around the corner.”

Read more:
Alberta family hopes to raise $2.8M to give toddler treatment for spinal muscular atrophy

Max’s family is also trying to raise money to get him the treatment. A GoFundMe page has been set up.

Reign’s and Kaysen’s families met when both babies were in Edmonton’s Stollery Children’s Hospital. There’s also a GoFundMe for Reign.

Read more:
Fighting for Reign: Alberta boy’s family says he needs $2.8M treatment for rare disorder

“The difference between the gene therapy and what he’s on now is that this gene therapy actually… replaces that dysfunctional gene that causes this neuromuscular disorder,” Durhane Wong-Rieger said on July 3.

Wong-Rieger is president and CEO of the Canadian Organization for Rare Disorders.

Max Sych's family is pushing to raise millions of dollars to cure his spinal muscular atrophy.

Max Sych's family is pushing to raise millions of dollars to cure his spinal muscular atrophy.

Supplied by Bowden Sych
Baby Reign has spinal muscular atrophy 1 (SMA), and his parents are hoping to get him a US treatment that's been deemed the world's most expensive therapy.

Baby Reign has spinal muscular atrophy 1 (SMA), and his parents are hoping to get him a US treatment that's been deemed the world's most expensive therapy.

Courtesy: GoFundMe

 

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