OTTAWA -- You could call it luck or fate, but the parents of Aidan Deschamps thank the scientists and doctors at CHEO. If their son had been born a week earlier, his story would be very different.
The Children's Hospital of Eastern Ontario began screening for Spinal Muscular Atrophy (SMA) just six days before Aidan was born. He is the first baby in Canada with the disorder identified through Ontario’s Newborn Screening program.
SMA is a debilitating and potentially deadly neuromuscular disease, according to CHEO. While officially considered a rare disease, it affects 1 in 10,000 and is the most common cause of death in childhood due to an inherited genetic reason.
Screening is done through a heel-prick test shortly after babies are born, meaning infants are diagnosed within days of birth, allowing treatment to start as soon as possible.
Filled with smiles, now 10-month old Aidan joined his parents during a virtual press conference Wednesday.
“Absolutely shocking,” Aidan’s father Adam Deschamps told CTV News Ottawa. “We are just so blessed to know that we live in a place that actually does testing.”
Aidan’s mom, Amanda Sully, said she is lucky they got pregnant and, “Unlucky to get the diagnosis, but lucky again to have the diagnosis so early that we have a possible treatment and a fighting chance.”
Sully and Deschamps received a call from Newborn Screening Ontario when Aidan was just 10 days old. Aidan was doing well at the time, so the call came as a surprise.
“They told us Aidan had screened positive for spinal muscular atrophy and had scheduled an appointment for us with Dr. Hugh McMillan, a neurologist at CHEO,” said Sully. “We had no idea what SMA was and what it might mean for our family.”
Until recently, there were no therapies for SMA. New therapies have since helped to get the genetic condition added to Ontario’s newborn screening program, according to a press release from CHEO.
“When I started at CHEO in 2010, we had nothing that could treat SMA,” said Dr. Hugh McMillan, a pediatric neurologist at CHEO, in the press release. “We managed symptoms as best we could to give children their best lives possible. Now, we actually have options for treatment and more therapies are on the near horizon. It’s very exciting. Being able to significantly improve the lives of kids like Aidan is unbelievably rewarding, as a physician and a researcher.”
In addition to his job as a pediatric neurologist, Dr. McMillan is also a clinical investigator at the CHEO Research Institute and an associate professor at the University of Ottawa.
“Because Aidan was treated before he had any symptoms of the disease, we can’t say for certain what the severity of his disease would have been. The very real hope is that Aidan has been treated early enough to maximize his developmental potential,” Dr. McMillan added.
Aidan was quickly started on a treatment called Spinraza at the age of three weeks before being granted access to and given a second treatment called Zolgensma when he was five weeks old.
Zolgensma is a gene replacement therapy and only needs to be given once. It is not approved for use in Canada but has been made available through the the pharmaceutical company’s managed access program, CHEO said.
“The neurological damage caused by the most severe forms of SMA starts very early in life and once the damage has occurred, it can’t be reversed. So the sooner a child with SMA accesses therapies, the better they do. This is why newborn screening is so important,” said Dr. Pranesh Chakraborty, Chief Medical Officer of Newborn Screening Ontario. “It helps us find babies with SMA as early as possible.”
The research on SMA was done by CHEO.
“The Newborn Screening Ontario labs here at CHEO, working alongside other CHEO clinicians and scientists, have demonstrated the power of combining compassionate clinical care and cutting-edge research, making it possible for children like Aidan to live their best life and have a different future,” says CHEO President and CEO Alex Munter.
Sully is thankful.
“You then take a step back and it’s not all about luck; it’s all about the hard work hard work of everyone out there that made this possible.”