Good news at the Rebecca Run
The Families of SMA (Spinal Muscular Atrophy) and supporters gathered for the annual Rebecca Run for SMA, at Fairy Lake Park on July 8 – where there was good news.
Not only did event sponsor ESSO make another big donation to the grassroots fundraising effort, there was an announcement by Health Canada: Spinraza, the first viable treatment developed for SMA approved by the American FDA last December, has now been approved for use in Canada.
SMA is a genetic disease, that results in the atrophy or wasting of the muscles due to the loss of motor neurons in the spinal cord and brain. As a result, infants diagnosed with Type 1 – the most severe form of SMA – are unable to lift their head, sit up unassisted, or crawl, and have difficulty in breathing and swallowing. SMA is the leading killer of children under the age of two.
Other forms of SMA lead to gradual loss of mobility, and eventually, death. There is no cure, and until now, no treatment, for the condition that affects 1 in 6,000 to 10,000.
Louise Smith organized the first Rebecca Run in 2002, in honour of her niece, Rebecca Grace Van Fraassen, diagnosed with SMA. Smith's goal: to “find a cure in her lifetime.”
Spinraza isn't a cure, Smith says, but the clinical trials suggest “babies diagnosed with SMA today will not suffer symptoms.” Studies found that infants given the drug, developed by Biogen, met their milestones – able to lift their head, able to crawl and stand – and had a longer life.
“We did it,” Smith says, but quickly adds, “We're not done. The next step is the cost.” The cost of the new drug is estimated at $750,000 for the first year alone, $375,000 for every year afterwards for the rest of the patient's life. “Our next fight is to have it funded by OHIP.”
Hundreds came out to support the fundraiser and Run or Walk for SMA. Winner of the 5K run was Bradford resident Fernando Carvalho, who finished the course in under 17 minutes.
He has attended the Rebecca Run nearly every year. “It got me into running,” Carvalho says. Initially, he knew nothing about the Charity, nothing about SMA, but came out to Fairy Lake because it was one of the few local Runs at the time.
It was after he met Smith, that he made running in the Rebecca Run an annual tradition. “She's a good person, it's a good charity,” Carvalho says.
Rebecca Van Fraassen, now 16 and in a wheelchair, attended the Run with her new service dog, to watch the 5K Run/ Walk, 1K Kids' Fun Run, and 3K Walk, Run or Roll through Fairy Lake Park, and listen to the announcement that followed.
There could be more good news, down the road. A gene therapy treatment for SMA is “in the pipes,” in Phase 2 trials, and showing promise, Smith says. “That will be a cure.”