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‘So fortunate’: Alberta baby ‘wins’ drug lottery for $2.8M spinal muscular atrophy treatment

Click to play video 'Spruce Grove baby wins drug company lottery for $2.8M Zolgensma spinal muscular atrophy treatment' Spruce Grove baby wins drug company lottery for $2.8M Zolgensma spinal muscular atrophy treatment
Health Matters October 26: A Spruce Grove baby has won the lottery for the nearly $3 million drug she needs for her rare, fatal disorder. And a COVID-19 vaccine candidate from Oxford University appears promising for older adults. Su-Ling Goh reports. – Oct 26, 2020

A little girl from Spruce Grove, Alta., has won a lottery being run by a pharmaceutical company to receive a multi-million dollar treatment.

Harper Hanki, who is just over a year old, was diagnosed with spinal muscular atrophy Type 1 (SMA 1) when she was just seven months old.

Harper is missing the primary gene that delivers the protein neurons to all her muscles, including the heart, lungs and esophagus.

Click to play video 'Spruce Grove family gets help as it tries to raise money for girl’s medical treatment' Spruce Grove family gets help as it tries to raise money for girl’s medical treatment
Spruce Grove family gets help as it tries to raise money for girl’s medical treatment – Sep 26, 2020

Her parents, Amanda and John Hanki, had been desperately trying to raise money for treatment with the drug Zolgensma, which can cost $2.8 million.

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They had raised about $272,000, less than a tenth of what they needed, when they received a call this week from Novartis, the company that manufactures the drug.

Read more: Spreading hope for Harper: How an Alberta girl’s fight for a medical treatment is bringing people together

“We won the baby lottery,” Amber said. “The baby lottery is a lottery that the drug company has put on, and they have committed to giving away 100 free doses throughout the world. And we were selected as one of them.”

“We always crossed our fingers one day that this is how it’d end,” John said.

“Now we can kind of take a big breath and celebrate.”

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Today’s not like a regular Friday – it’s different. You see, today, we’re not starting off with five dollar Friday’s. We’re no longer spending long nights event planning. We don’t have to hire babysitters to work long hours organizing and researching fundraisers. We’ve committed to never post another GoFund Me for 2.8 million dollars and accept another automatic deposit for a medication that we cannot afford. This week we won the lottery to save Harper’s life. The one all SMA families dream of. We truly won the best lottery we could of ever asked to win! Harper will be getting Zolgensma in Calgary the first week of December. It’s the best thing that possibly could have happened in 2020. Today, we are celebrating and not holding back! How incredibly lucky are we! We’re not done yet. We have newborn screening to advocate for, governments to lobby and a drug that still needs Canada’s approval and funding. Today we are taking a moment to absorb the phone call that we got. We are going to celebrate this news and I hope you will too! Thank you for supporting our family. Thank you for sending me good vibes when the weeks felt heavy with grief. Thank you for being apart of this journey and being the hero that Harper needed! Stay tuned for our future plans which we will be discussing soon. This includes money, zolgensma and a few months of isolation in southern Alberta. Love Amanda, John and the whole Hanki Gang! #zolgensma #myheroharper #smatype1

A post shared by Harper Hanki (@myhero.harper) on

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The “baby lottery” is actually the company’s managed access program that Novartis is running to give away doses of the drug to applicants from all around the world who are under two.

Zolgensma will give Harper the gene she is missing, and although it won’t correct any loss of muscle movement so far, it will stop the spread of SMA.

“It’s awesome,” John said. “There’s still going to be a lot of work ahead for Harper. But this is going to put her on a trajectory to potentially walk one day. That’s what we’re hopeful for.”

John, Harper and Amanda Hanki in Spruce Grove on Oct. 25, 2020. Global News

The money the family has already raised will go to SMA research, her parents said, or possibly towards helping another Canadian baby with the disease.

“We’re so fortunate and we would love to see everyone benefit from it,” Amanda said.

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Harper will go to Calgary to get testing done at the beginning of November, and then is set to receive Zolgensma, also in Calgary, in December. Her family says they believe she will be one of the first Albertans to receive it.

“We know right now Harper is living on borrowed time, and we can’t wait to see what she does with it,” Amanda said.

Read more: B.C. baby randomly selected by drug company to receive life-changing $3 million treatment

Harper had previously been receiving Spinraza, a drug that is administered by injection every four months, with each dose carrying a price tag of $125,000.

Zolgensma is a one-time treatment. It has not yet been approved by Health Canada. 

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Spinal muscular atrophy (SMA) Type 1 is a rare disorder that affects about 1 in 6,000 babies and causes muscles to waste away. Most die of respiratory issues before their second birthday.

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–With files from Jessica Robb, Su-Ling Goh and Amy Judd, Global News