In the past few years, the number of The Neuro’s clinical trials involving patients with amyotrophic lateral sclerosis (ALS) has grown exponentially. As The Neuro marks ALS Awareness Month in June, eight ALS drug trials are actively underway and six more are planned.
Not so long ago, the outlook for effective ALS treatments seemed bleak, but today The Neuro is working in an era of hope.
“It’s an exciting time for ALS research,” notes Kristiana Salmon, assistant manager of the Clinical Research Unit (CRU) where she specifically manages the ALS research program. “There are so many drug studies underway targeting different mechanisms of action and symptom management. This is leading hopefully to disease-modifying therapies in the future.”
Results of one multi-centre drug trial in which The Neuro participated were presented in May at a meeting of the American Academy of Neurology. Forty-five patients at The Neuro took part in the trial, the largest number of any participating institution.
“One drug looked to improve muscle strength,” says Salmon. “In ALS, only the connection between muscles and the brain is dysfunctional. The muscles themselves are still functional, so still healthy muscles are a target that drugs can aim for. The drug seeks to make breathing easier and to maintain muscle function that’s used in daily activity.”
Another drug trial drug targeted a genetic mutation, the SOD1 mutation, found in two per cent of the ALS patient population.
“It’s a promising therapy that looks to modify the cause of the disease, which is a genetic cause that can, hopefully, be directly affected,” she says. “We screened our first patient in May for a Phase 3 trial, which aims to confirm the drug’s safety and effectiveness, and any risks that it might pose. In all, there will be 60 patients at 19 sites worldwide. We are one of only two sites in Canada. We have Canadian patients coming as far away as Vancouver and international patients coming from Israel and New Zealand. I think this is a testament to how the director of the CRU, Dr. Angela Genge, has made the CRU and the ALS Clinic into an international centre of excellence for both care and access as early as possible to innovative therapies.”
ALS research at The Neuro is also taking the lead in developing criteria for evaluating a patient’s ability to drive a car. Although ALS patients gradually lose control of their muscles, their ability to continue common daily routines for as along as possible is an important factor in enjoying a good quality of life.
“A grant to study driving came from ALS Canada from donations gathered in the Ice Bucket Challenge,” says Toni Vitale, a nurse clinician who cares for The Neuro’s ALS patients. “There are no national standards or consistent ways of evaluating drivers with ALS. Furthermore, it can take several months for drivers with ALS to get an appointment for a road-test evaluation, which is unacceptable in the case of someone with a neurodegenerative disease. Our funding helps subsidize private on-road evaluations. This way, we can demonstrate that more timely evaluations could allow patients to get the needed adaptations that will allow them to drive safely over a longer period of time.”
After the pilot study finishes next year, the intention is to share its findings with Canada’s more than 15 other ALS clinics in the hope that national standards might be adopted.
The Neuro’s ALS Clinic
About 250 ALS patients are actively observed and treated at The Neuro’s ALS Clinic. Between 60 and 80 new ALS cases are diagnosed here each year.
Angela Genge – Neurologist and Director, CRU
Rami Massie – Neurologist
Maxime Bérubé – Neurologist
Guy Rouleau – Geneticist
Toni Vitale – Nurse Clinician
Nathalie Magnan – Respiratory Therapist
Kendra Berry – Occupational Therapist
Maura Fisher – Physiotherapist
Caroline El-Tawany – Physiotherapist
Stephanie Palacco – Social Worker
Lana Kim McGeary – Spiritual Care Counsellor
Kalyna Franco – Speech Language Pathologist
Nancy Anoja – Genetic Counsellor
Ritsa Argyriou – Administrative Agent
Kristiana Salmon – Assistant Manager CRU
Natalie Saunders – Clinical Research Coordinator
Juliette Foucher – Clinical Research Coordinator
Roxana Nedelcu – Dietician
What is ALS?
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that occurs when motor nerve cells in the brain and spinal cord progressively die causing muscles to weaken until the patient becomes totally paralyzed. Death usually occurs within about five years following diagnosis. A small percentage of patients survive for ten years or longer. Fewer than one in patient in ten has a family history of ALS. There is no cure but much progress has been made in recent years to improve patients’ quality of life.
ALS is commonly known as Lou Gehrig’s disease after the famous American baseball player who died of it in 1941.