A baby who can’t crawl: A cure is on the way, but this Milton baby’s time is running out – InsideHalton.com

Posted: January 14, 2020 at 6:49 am

"We're moving close to a cure," says Wyatt's doctor, Mark Tarnopolsky, who is director of the neuromuscular and neurometabolic clinic at McMaster University Medical Centre in Hamilton.

Children with spinal muscular atrophy lack a certain protein that is critical for the maintenance and function of specialized nerve cells, called motor neurons, which control muscle movement throughout the body.

Without the protein, the motor neurons die and the muscles can't move. If they don't move, they shrink and weaken. The child suffers debilitating effects that inhibit even their ability to breathe and swallow.

Until recently, most children with this type of spinal muscular atrophy died of respiratory failure by their second birthday.

But in 2018, a new drug called Spinraza was approved for use in Canada. It prevents the rapid nerve degeneration from occurring, if it is given early enough in the child's life.

At $125,000 a dose, it's very expensive. Luckily, the Ontario government pays for it.

Wyatt has had four doses since he was diagnosed six months ago.

He can clap his hands together and kick a little bit, Dannon said.

"He is such a happy little guy," she said. "Every moment I spend with him, I love."

Wyatt's aunt and parents have started a GoFundMe page to help pay for some of his expenses. The family has already raised about $74,000 through this and other fundraisers.

They've poured their hopes into a brand new drug called Zolgensma. It's a gene therapy does the same thing as Spinraza, but it's a single dose that lasts 25 years.

"With a longing we didn't know was possible, we hope one day Wyatt can dance to the beat of his favourite song, send our hearts racing as he climbs a high tree or wrestle with his dad on the floor," the family says on its GoFundMe page.

But that's unlikely to happen.

The drug is very expensive, at $2.8 million Canadian for a dose. It's not available in Canada yet.

More significantly, Tarnopolsky thinks the benefits would be very limited for a child like Wyatt. That's because he's already too old.

Nerve degeneration happens very quickly for the tiny number of children, perhaps several hundred in Ontario, who have this disease. It drops "like a stone" shortly after birth, Tarnolpolsky said.

And once the nerve function is gone, it doesn't come back.

The studies have shown it's much better for children to receive either Spinraza or Zolgensma if they get it when they're two or three months old. The oldest child in one of the Zolgensma studies was eight months old.

He would like to see newborns routinely screened for this disease so it can be treated right away.

Michael Harris, the Waterloo regional councillor who was formerly MPP for Kitchener-Conestoga riding, has met many families like the Vaseys.

"They'll do anything" for a chance for their child to get better," he said.

When he was MPP between 2011 and 2018, Harris championed the cause of families with rare diseases.

He toured the province to hear from them, tried to have an all-party committee of MPPs to discuss the issues, and lobbied for them to have access to government funds for the expensive medication they need. (It's expensive because so few patients need it, and there are few buyers to share the high cost of research, development and clinical trials.)

Working for these families was "the only time I came to tears in my entire career," he said. "I think of them all the time."

One look at Wyatt helps explain why.

He is cheerful all the time despite the long uphill road ahead for him, his family said.

Now that he has learned to clap his hands together, "all he loves to do now is clap," said Dannon.

"You would never know any of the challenges (that he faces) exist for him."


Twitter: @DamatoRecord


Twitter: @DamatoRecord

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A baby who can't crawl: A cure is on the way, but this Milton baby's time is running out - InsideHalton.com

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