A Phase III trial involving a promising stroke drug will receive a $6.6-million injection from Brain Canada – yet another endorsement for a Canadian medication that takes aim at the country's third leading cause of death.
The intravenous medication, called NA-1, is the $30-million, private venture of a company called NoNO Inc. Its development, nearly 20 years in the making, is unusual: It is neither in bed with a large pharmaceutical company nor has it relied on the largesse of the public purse. The brainchild of neurosurgeon Mike Tymianski of Toronto Western Hospital, the drug will be administered by paramedics to 518 stroke patients in a field program called FRONTIER, which rolls out in ambulances in the GTA, Peel Region and Vancouver next January.
The hope is that NA-1 – designed to contain the damage to the brain in the event of a stroke – will be the country's most meaningful contribution to modern medicine since insulin.
"This is potentially a huge Canadian success story," said Mark Bisby, a science adviser for Brain Canada. "Strokes cost the country three to four billion dollars a year, and if we can put a dent in that, it will be money well invested."
Last May, Toronto's Sunnybrook Health Sciences Centre waived consent for FRONTIER, meaning the hospital's research ethics board approved health-care professionals to apply the drug without patient consent. On July 31, St. Michael's Hospital in Toronto granted FRONTIER deferred consent, meaning researchers can collect information needed to perform the study without any change in the care procedures before a patient or family member consents. In previous trials using animals and a small population of humans, NA-1 has demonstrated positive results without any serious side effects.
NA-1, a neuroprotectant, belongs to a thus-far-unsuccessful classification of drugs designed to help or protect brain cells from the effects of stroke. Over the past two decades, more than a thousand of them have failed to reach market.
While skeptical about the drug's prospects as recently as six months ago, team members and NoNO investors are sounding increasingly optimistic about their medication's unique proposition. NA-1 tries to control the activity of PSD-95, a potentially malevolent protein that, in the event of stroke, turns brain cells into pools of lethal toxins.
The announcement of the grant, which will be made Friday at St. Michael's by federal Health Minister Rona Ambrose, is earmarked for FRONTIER – the $10-million, Phase III trial headed by Laurie Morrison, the Robert and Dorothy Pitts Chair in Acute Care and Emergency Medicine at St. Michael's. Brain Canada will contribute $3.3-million over three years with a matching grant from private contributors, including NoNO and the Canadian Stroke Network. "It's much larger than our standard research grant," Dr. Bisby said.
In Canada, someone has a stroke every seven minutes, which beyond its social cost takes a massive physical, financial and emotional toll on patients and family alike. It's especially important to treat the event with dispatch. A stroke kills two million brain cells a minute, and the chances of a positive outcome decreases 10 to 20 per cent every half-hour that it goes untreated.
Hoping to intercept the damage long before a patient reaches the hospital, FRONTIER's paramedics will administer NA-1 with the remote guidance of a neurologist. (The experiment will be double-blinded, meaning a placebo will also be used). NA-1 then buys the brain enough time for the patient to reach a nearby stroke centre, where ideally neurologists can treat the patient with tPA, the only stroke drug on the market. Also called Brain-O, tPA is a clot-buster with a daunting list of medical circumstances that disqualify most patients from receiving it.
FRONTIER will be a gauntlet that will involve the education of thousands of health-care professionals. The research itself won't be easy, either. "The challenge now," Dr. Tymianski said, "is to do as rigorous a study as possible in the chaotic environment of an ambulance."