A decade after the ALS Ice Bucket Challenge swept the internet, tremendous progress has been made toward understanding the devastating neurodegenerative disorder that steadily robs patients of muscle control. However, effective treatments remain elusive — and so does a cure. 

But Dr. Erik Pioro believes we’re closer than ever to solving the riddle of amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig’s disease. Recently appointed to the ALS Society of BC Professorship in ALS Research at UBC, he’s developing a new research program that spans basic science and clinical research to uncover life-changing therapies that could slow or halt the disease’s progression.

We spoke with Dr. Pioro in his lab at the Djavad Mowafaghian Centre for Brain Health (DMCBH), where he also leads Vancouver Coastal Health’s Amyotrophic Lateral Sclerosis and Related Disorders (ALSRD) Clinic, about how the program and his research will benefit people with ALS in British Columbia and beyond.

What inspires you to pursue ALS research?

Throughout my career, I have had the honour of getting to know many people with ALS who, in the face of this incredibly relentless and severe disease, never cease to inspire me with their bravery, hope and resilience. My passion really stems from both the profound human impact of the disease and the scientific challenge it represents.

ALS is a complex puzzle that affects the entire nervous system, and we still don’t have a solid understanding of what causes it and how to stop it. Putting that puzzle together, so we can help patients, is what drives me.  

What do we know about the causes of ALS?

That’s one of the biggest questions we’re working to solve. There are some instances that have a genetic or hereditary component, but in 85 to 90 per cent of cases, there’s no apparent rhyme or reason why a person develops ALS. The disease strikes unexpectedly, oftentimes affecting people in extraordinarily good health. So, the million-dollar question is, why?

The current thinking is that it is a combination of genetics, environment and lifestyle. We’re exploring how factors like pollution, injury and neuronal stress may contribute to the disease in genetically susceptible individuals. If we can understand the root causes, then we’ll have a better shot at preventing, treating and curing ALS.

How is your research bringing us closer to solving these challenges?

Our research spans the entire spectrum — from basic science in the lab, to translational research identifying biomarkers, to clinical research with patients and finding effective treatments. What’s incredibly powerful is the integration between our UBC research program and clinical care in the ALSRD Clinic. We want to give every patient in B.C. the opportunity to participate in cutting-edge research that could help us understand how to slow the progression of this disease.

“This is an incredibly exciting time in ALS research — advances in artificial intelligence, machine learning, neuroimaging and genetics are rapidly transforming our understanding of this devastating disease.”

– Dr. Erik Pioro

With this integration, we’re building a robust clinical trial program that will give ALS patients in B.C. access to the latest medical breakthroughs and novel treatments. It also means we’re able to collect clinical data and patient samples that provide real-world and highly individualized insights into our research.

As one example, we can use patient samples to generate induced pluripotent stem cells (iPSCs) and essentially recreate a patient’s ALS in a petri dish. It’s a huge step toward precision medicine that allows us to model a patient’s individual disease and understand its triggers, how it progresses and how best to treat it.  

How are emerging technologies reshaping ALS research?

This is an incredibly exciting time in ALS research, with advances in artificial intelligence, machine learning, neuroimaging and genetics rapidly transforming our understanding of this disease. And because UBC is home to many of the top researchers in each these fields, we’re able to bring that expertise together to tackle questions from completely new perspectives.

For example, we can use AI to analyze neuroimaging data from MRI and PET scans, revealing previously unidentified changes in brain structure and chemistry associated with ALS and other neurodegenerative disorders. By combining the power of these technologies, we hope to accelerate the discovery of biomarkers that will enable earlier diagnosis, more accurate tracking of disease progression and quicker selection of novel effective therapies.

What does the future hold for ALS research in B.C.?

The progress we’ve made in understanding ALS over the past few years is truly encouraging, but it’s only the beginning. By leveraging the expertise here at UBC, with the support of patients and families, the ALS Society of BC and PROJECT HOPE, as well as the Province of British Columbia and health system partners, I am optimistic that we will make significant strides toward developing effective treatments that noticeably slow down, and ultimately, stop ALS.

My goal is to firmly establish UBC as a global leader in ALS research, which will benefit all Canadians living with ALS and related disorders, as well as those afflicted by the other neurodegenerative diseases.