B.C. family nearing finish in race to raise $2.8 million to treat baby's rare disease

·3 min read

Update: The family announced in an Instagram post Saturday evening that their fundraising efforts surpassed $3 million.

A family in Surrey B.C. hopes to raise enough money to pay for a possibly life-changing but pricey therapy for their baby boy.

One-year-old Aryan Deol struggles with muscle weakness and limited mobility. His parents say he relies on the assistance of a breathing tube to eat and a machine to breathe, and he cannot sit up on his own or hold his head up.

He was diagnosed with Type 1 spinal muscular atrophy, also known as SMA, a rare recessive neuromuscular disease that results in muscular atrophy and respiratory issues for mostly children.

Aryan's parents said doctors advised them to research possible treatments.

'Maybe he'll be able to do everything'

"Maybe he'll be able to do everything, he'll be able to live a normal life," said the boy's mother Harpreet Kaur Deol.

A relatively new therapeutic drug called Zolgensma is available in the United States — at a cost of more than $2 million US. So far, Deol has been taking Spinraza, a medicine approved in Canada.

Channel Punjabi, a Vancouver-based radio station, hosted a fundraiser on Friday, which also marked Aryan's first birthday, to help collect funds to pay for the new drug.

Harman/CBC
Harman/CBC

Aryan's parents say they are thankful for the community's support.

Since March, an online fundraiser has collected more than $2.7 million. The family hopes to reach $2.8 million.

The family says donations started off slowly but took off after celebrity Neeru Bajwa, a Canadian-born Indian actress, director and producer, began supporting the cause.

Getting treated

Aryan's mother said Zolgensma would provide the therapy required for her son's body to begin producing proteins needed to eat, breath and walk on his own.

Zolgensma has been approved by the U.S. Food and Drug Administration (FDA) but Health Canada has not yet approved it.

An online statement from Novartis Pharmaceuticals Canada Inc. in June announced the filing of an application for AVXS-101, known elsewhere in the world as Zolgensma, for approval by Health Canada. It states the company anticipates a decision will be made by the end of the year.

The FDA's prescribing information sheet indicates the gene therapy is a one-time dose used to treat patients under age two. It warns of acute serious liver injury as a possible adverse effect of the infusion.

In addition to the Deols, other families in Canada are also holding out hope that they too can find a way to get Zolgensma for their children diagnosed with SMA.

Gaganpreet Singh Deol, Aryan's father, said he has seen success stories coming out of the U.S. from families of children who have tried the drug there.

The family hopes an equivalent drug will be allowed for use north of the border soon, so their son can be treated in Canada. They are staying optimistic despite the circumstances.

"I don't want to go negative. I want to be positive," Gaganpreet said.

He believes his son will get the therapy he needs and his condition will improve soon.