How to create hope for people with ALS and their families.
It was also a globally important one because that protein could help lessen the tremendous hardship caused by amyotrophic lateral sclerosis (ALS). It might even be a path to a cure.
Often called Lou Gehrig’s Disease after the New York Yankees’ star who died from condition in 1941, ALS is a debilitating neurodegenerative condition that progressively impairs nerve cells for muscle control, leading to muscle wastage, paralysis and, ultimately, death. The average life expectancy of someone with ALS is only two to five years.
ALS is linked to a protein called TDP-43, which harms nerve cells. Proteins are like tiny machines inside our cells, each with a specific job. In ALS, the protein TDP-43 stops working properly and starts clumping where it shouldn’t. When that happens, the cell’s normal processes break down, and the nerve cell becomes damaged and eventually dies. Mike and his team discovered that the protein RGNEF can counter the effects of TDP-43 – significantly reducing the damage to nerve cells and even stopping it.
The research has been conducted on fruit flies and mice. Human trials will happen in the next three to five years.
The discovery was a pivotal moment for Mike, who has spent three decades with his team and collaborators pushing toward a major advance in ALS.
Mike is both a Schulich School of Medicine & Dentistry scientist and a physician who works with ALS patients. What matters most to him isn’t the discovery itself, but the impact it will have on the lives of those living with the disease.
“It’s about one word – hope. I've seen roughly 3,500 people with ALS. And every conversation centres around whether we have any hope at all that this disease can be cured or if we can slow it down in some way. Now, I sit down with families and say, ‘I don't have the cure for most forms of ALS right now, but we think we have it for a couple of them,’ and I couldn't do that before. Compared with 30 years ago, we have far more hope today, because it's realistic now.”
Mike's hopes for the future rest on three key requirements. One is collaboration.
The other essential factor is an openness to grow through experiences, good or bad.
“Someone once wrote, ‘you are the sum total of your experiences.’ I firmly believe that. If you're not constantly changing based on the experiences, then you're stagnant. If you ignore those experiences, I think you're a lesser person for it.”
And the final key to making a real difference? For Mike, it’s mentoring those who will carry the work forward.
“Mentorship is important in everything we do. I've had mentors who had no idea they've been mentoring me. I still talk to my research supervisor now, 35 years later. When I was doing my postdoc training, he said to me, ‘the best mentors are the ones who change the direction of what you're doing, and you have no idea they did it.’ It took me years to fully understand what that meant. But I like to think that's how I do it now with my colleagues and students.”
For Mike, every experiment, collaboration and mentee brings ALS research closer to a breakthrough — and with it, renewed hope for patients and families.