We are pleased to share our strategic impact goals from our latest strategic plan. We start with a cornerstone of our organizational commitment—research. At ALS Canada, our mission is shaped by the relentless challenges faced by people living with ALS, inspiring us to lead with pioneering research that transcends boundaries and ignites hope.
A world united in the pursuit of answers
We are Canada’s ALS organization, distinct in our comprehensive approach to investing, facilitating, contributing to and building capacity for groundbreaking research. Our commitment has always placed us at the forefront of the global fight against ALS, aiming to manage and ultimately eradicate this disease.
In research, your support catapults us to the top of scientific innovation. We are currently supporting various high-impact studies, expanding our research and clinical capacities, and playing a pivotal role in positioning Canada as a prime destination for promising ALS clinical trials.
These initiatives are more than just scientific endeavours—they provide real hope to families, giving their loved ones access to advanced treatments potentially capable of extending lives, slowing disease progression, and improving the quality of living.
Highlights of our current research initiatives:
Funding Cutting-Edge Projects: With over 250 awards supporting research since the national ALS Canada Research Program’s inception in Canada and internationally, we are driving discoveries that tackle ALS from multiple angles.
Empowering Through Clinical Trials:The Canadian ALS Research Network (CALS) represents a nationwide effort in the quest for cures through testing experimental therapies and sharing best practices for clinical research.
Collaborating for Greater Impact: ALS Canada works with researchers, clinicians, and organizations nationally and worldwide to ensure we maximize our impact toward new treatments that will help Canadians affected by ALS.
Each step forward in research is a step closer to a world free of ALS, made possible by your continued support. This journey towards groundbreaking discoveries is about achieving scientific milestones and crafting a legacy of hope and resilience that reverberates through families and communities across Canada.
Stay tuned for our next post, where we will delve into the support aspect of our mission, showcasing how your contributions are vital in providing care and resources to the people at the heart of our cause.
United, we are on a path to defeat ALS. Thank you for your unwavering support and belief in ALS Canada.
Meet the junior researchers behind the 2022 ALS Canada – Brain Canada Trainee Awards
After finishing his PhD in France, Dr. Ghazale moved to Canada to work with Dr. Carol Schuurmans at Sunnybrook Research Institute. Her team examines if neuronal reprogramming could serve as a potential treatment strategy for neurodegenerative diseases, including ALS.
ALS causes damage to neurons, affecting the body’s everyday ability to function. Dr. Ghazale’s work focuses on attempting to reprogram glial cells – a common and abundant type of brain cell – into new neurons. The team hopes these fresh cells could effectively integrate with existing cells in brains of people with ALS, improving their capacity and quality of life.
At the moment, Dr. Ghazale is testing the strategy in animal models. But with this influx of funding, he notes he can speed up his project timeline and do higher-tech experiments. The award will also help him refine his strategy to potentially translate this work to clinical applications.
“I know that this work could one day end up supporting people living with ALS and their families and help … ease what they’re going through,” he says. “This is what motivates me every day to keep working hard to improve therapeutic strategies.”
“We must continue to push the boundaries of ALS research,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “It’s through these uncharted territories that we will uncover the solutions and enable ourselves to envision a future where ALS no longer exists.”
Funding for the 2022 Postdoctoral Fellowship was made possible by Fondation Vincent Bourque, who generously contributed $82,500 to ALS Canada, which was matched by Brain Canada through the Canada Brain Research Fund (CBRF).
The CBRF is an innovative arrangement between the Government of Canada (through Health Canada) and Brain Canada Foundation, which increases Canadians’ support for brain research and expands the philanthropic space for funding brain research to achieve maximum impact. To date, Health Canada has invested more than $145 million in brain research through the CBRF which has been matched by Brain Canada Foundation and its donors and partners.
The ALS Canada-Brain Canada Trainee Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
Meet the students who received the 2022 ALS Canada – Brain Canada Doctoral Awards
PhD student Lucia Meng Qi Jadon (previously Liao) is the recipient of a $75,000 ALS Canada – Brain Canada 2022 Trainee Award. She will use the funding to investigate whether a newly discovered tag on TDP-43 might have an important role to play in ALS.
For her first six months of doctoral work in Dr. Dale Martin’s lab at the University of Waterloo, Lucia struggled. “I was trying to show something that hadn’t been shown before, so it was difficult,” she said.
But then – success. She detected a faint signal that confirmed she was on the right path. Thanks to her persistence, her work is opening up a new pathway for research.
In a way, the award was like that first signal, confirming her work as a researcher. “I put in all this work, and this award helps me know I’m good enough to be here,” she said. “It’s a huge encouragement that I am cut out for this.”
Lucia’s desire to understand the intricate, complex processes of human cells drives her passion for research.
“When you discover one process, it immediately opens up new possibilities. It’s like a puzzle you keep finding more pieces to,” she says.
Lucia’s project looks at one piece of the complicated ALS puzzle. Early in her PhD work, she confirmed that TDP-43 undergoes a modification called palmitoylation. This process may play a role in TDP-43 mislocation, one of the most common hallmarks of ALS.
She will use this funding to better define the properties of palmitoylation of TDP-43 and understand how it might be abnormal in ALS.
“I’m peeling back the layers, little by little,” she says.
“Ms. Jadon’s work will serve as a beacon, drawing the attention of like-minded scientists eager to explore new frontiers in ALS research,” says Dr. Viviane Poupon, President and CEO of Brain Canada.
Lucia says looks forward to sharing her findings to spark new collaborations within the ALS research community. “I want to establish the basics of this information I discovered and get this out there,” she said. “Then other researchers will know this is something they may want to look into.”
The ALS Canada-Brain Canada Trainee Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
Meet the students behind the 2022 ALS Canada – Brain Canada Trainee Awards
Charlotte Manser is the recipient of a $75,000 ALS Canada – Brain Canada 2022 Trainee Award. As a PhD student at the University of Ottawa, she investigates how ALS-linked genes might contribute to the loss of normal stress granule formation.
When our cells are stressed, they create “stress granules” to protect RNA, which are critical substances to cellular health. Charlotte explores how the abnormal accumulation of stress granules might contribute to the mislocation of the protein TDP-43, a common hallmark of ALS.
She has tested many genes that might be linked with both stress granule formation and ALS.
“I found two hits that I’m actively pursuing,” she says. “With these, we can try to uncover new mechanisms of disease or a new therapeutic target. They can help us better understand how the disease comes about.”
Determination motivated by loss
Charlotte’s work is motivated by her personal connection to the disease.
In 2013, her father died from ALS. Around that time, she switched her undergraduate major from forensics to neuroscience.
“It was a light bulb moment,” she says. “I knew this is what I wanted to do.”
She also became an active member of the community of families affected by ALS. Charlotte notes that it means a lot for this same community to recognize her work with this award.
“This work represents the hope of turning something awful, like grief, into something productive and positive,” she says. “My hope is that I can contribute something to the field so that it isn’t so bad for the next person.”
The ALS Canada-Brain Canada Trainee Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
Meet the junior researchers who received the 2022 ALS Canada – Brain Canada Trainee Awards
Donovan McDonald is the recipient of a $75,000 ALS Canada – Brain Canada 2022 Trainee Award. As a PhD student, he investigates how the function of tRNA could contribute to ALS disease processes.
Donovan came to Canada from the Bahamas. “People don’t always recognize the challenges that international students face,” he says. Funding opportunities, for example, can be far more limited.
Award programs like those provided by ALS Canada and Brain Canada help fill a critical funding gap for promising students like Donovan.
“Supporting international students in brain research is vital for fostering diverse perspectives, advancing global scientific collaboration, and accelerating breakthroughs in understanding the complexities of the brain,” says Dr. Viviane Poupon, President and CEO of Brain Canada.
“It’s not just about the money,” Donovan says. “It’s also having your research recognized. This is crucial to your development as a scientist.”
Donovan’s original project sought to understand basic biological processes around tRNA. This critical molecule acts as a master key within cells to help proteins form. But working alongside prominent ALS researchers at Western University, he realized how relevant his work could be to exploring “uncharted territory” in ALS research.
In particular, Donovan is exploring how tRNA dysfunction might contribute to ALS in relation to a protein called angiogenin. Researchers have linked mutations in angiogenin, which helps regulate tRNA, to genetic cases of ALS.
“Despite being known for a long time, very few studies look at angiogenin and its role in ALS. That’s where I come in,” Donovan says.
The young researcher hopes his work helps others recognize to the idea that tRNAs are important molecules to study in ALS.
“I want to unravel how tRNA dysfunction can help point to either development of or predisposition to ALS,” he says. The ALS Canada – Brain Canada Trainee Award will help him do just that.
The ALS Canada-Brain Canada Trainee Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
Meet Dr. Philip McGoldrick, recipient of the 2022 ALS Canada – Brain Canada Career Transition Award
Dr. Philip McGoldrick, a researcher at the Tanz Centre for Research in Neurodegenerative Diseases at the University of Toronto, is the 2022 recipient of a $250,000 ALS Canada – Brain Canada Career Transition Award.
This award helps launch talented early-career researchers, allowing them to set up their own labs, giving them the independence to establish their own research programs.
“This award enables me to continue something I’ve been passionately working on for several years,” he says. “The work I am doing is so exciting. It would have been a huge loss to not be able to continue it.”
Dr. McGoldrick was inspired to study neurodegenerative disease at a young age after seeing how these types of diseases affected family members. After moving to London (UK) for a PhD, ALS soon caught his interest.
“It was the most interesting one of the diseases being studied,” he says. He’s been working in ALS since 2008.
Dr. McGoldrick studies mutations in the C9orf72 gene – the most common genetic cause of ALS. His current projects explore how the loss of function that occurs when C9orf72 is mutated can affect a crucial biological process called nucleocytoplasmic transport. He and his co-researchers recently published an article on the topic in Cell Reports. He is also interested in how these insights could be helpful in understanding sporadic cases of ALS.
“This support will allow me to investigate a fundamental but understudied mechanism that may contribute to ALS,” he says. “My hope is that I can be part of a sustained effort on multiple fronts to understand a genetic cause of ALS, but also apply these findings to understand sporadic disease too.”
“This transformative program and collaboration with ALS Canada has the potential to inspire early-career researchers to explore innovative approaches, which could lead to a future without ALS,” says Dr. Viviane Poupon, President and CEO of Brain Canada.
The ALS Canada-Brain Canada Trainee Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS.
Meet the researchers behind the winning 2022 Discovery Grant projects
Could protecting the axon represent a promising treatment strategy for ALS?
Award: $300,000
Collaborators: Dr. Gary Armstrong, McGill University
Dr. Alex Parker, at the Centre de recherche du CHUM, Université de Montreal, is one of the first two recipients of the newly introduced three-year, $300,000 ALS Canada-Brain Canada Discovery Grant. Dr. Parker’s grant was funded in generous partnership with Dr. Jean-Pierre Canuel Fund – SLA Québec.
Dr. Parker’s project looks at possible targets for slowing neuronal degeneration in small worms used to model ALS, called C. elegans, which he has worked with since his days as a doctoral student at the University of British Columbia. He has been fascinated by neurodegeneration even as an early career researcher.
“I liked the idea of trying to model neurodegeneration in a simple system,” he says. “You can go fast and find things.”
He started his work looking at Huntington’s Disease before switching to ALS – a challenging disease that motivates him to work on problems not everyone wants to tackle.
“ALS is not like other diseases. It’s a hard problem – the disease window is short and aggressive,” says Dr. Parker. “For many people, it seems intractable. But we’re keeping at it – and I think we can make a contribution.”
For decades, researchers have used worm models to understand which genes help regrow neuronal axons after they are damaged. To date, these genes have not been studied for their role in the damage done by ALS.
Dr. Parker began this project by cataloging more than 100 of these genes – which either promote axonal regrowth or slow further damage – as potential targets for study in ALS models.
“We tested them all. And lo and behold, some of them actually stopped further neurodegeneration in an ALS model,” he says.
He then tested his “short list” on older worms that better simulate the cellular environment of ALS patients, who tend to be diagnosed in midlife or later. The result was two “extremely potent targets for slowing neurodegeneration,” he notes.
“We’re taking biology that’s known and rephrasing it for ALS,” Dr. Parker explains. The hope is to find drugs that can target these genetic pathways that reignite a cell’s ability to regenerate its own axons, effectively slowing disease progression.
He said the upgraded funding opportunity helped him think more ambitiously about the project and bring in collaborators that could accelerate the research.
Dr. Parker is working with Dr. Gary Armstrong at McGill University, an expert on zebrafish models. With the larger funding amount, Dr. Parker can move his hypothesis through different levels of validation, including zebrafish and iPSCs (human cell models), which could allow him to advance his findings to clinical trial on a much shorter timeline.
“The structure of the grant opened up the possibility of pushing this further and faster,” he says. “We’re already moving towards validation, and we just started.”
“This type of program is so important,” he adds. “It funds projects at the early stages, when it’s more challenging to get funding. This ALS Canada-Brain Canada Discovery Grant gave us the chance to really get our research going.”
“Supporting research at its earliest stages leads to transformative breakthroughs that have a lasting impact on human health and well-being,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “By funding early-stage research, we can address critical gaps in knowledge, focus on emerging challenges, and encourage researchers to explore unconventional approaches.”
The ALS Canada-Brain Canada Discovery Grant Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
To find out more about the 2022 ALS Canada-Brain Canada Discovery Grants, read the full press release here.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS.
“It’s always really exciting when you get a research grant, because you’re using your ideas that you’ve researched in the literature and created new experiments around,” he says. “After all that, it’s exciting to have other scientists review it and appreciate your ideas.”
This project represents his first grant for ALS research, but it builds on his decades of work studying the proteins involved in neurodegeneration, particularly in Parkinson’s disease.
“It’s a bit of a new direction for my lab, but it seems like a logical progression” says Dr. Shaw.
“At Brain Canada, we adopt the One Brain approach to research, knowing that every discovery has the potential to have an impact across a spectrum of brain diseases and disorders,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “Dr. Shaw’s work in both Parkinson’s disease and now ALS will improve our understanding of the complexity of the brain as a whole.”
Tagging proteins to “take out the trash”
When our cells age, our body has a mechanism to “tag” unwanted or problematic proteins so they can be removed. Unfortunately, the enzyme machinery for this process often does not work properly in ALS and other neurodegenerative diseases.
Dr. Shaw’s project seeks to identify which proteins might be responsible for tagging the unwanted or misfolded proteins for removal from cells in common types of ALS.
“And if what we can do is identify proteins that we think are regulating these processes,” he explains, “those enzymes could be potential targets for small molecule therapeutics.”
His transition from other neurodegenerative diseases to ALS research is aided by his collaborators, including long-time ALS researchers Dr. Martin Duennwald, also at Western University, and Dr. Elizabeth Meiering at the University of Waterloo.
Though Dr. Shaw studies the most intricate of biochemical processes, he says he is inspired by the possibility that his research could make a difference for people living with ALS.
“I’ve always wanted to do research that is going to make a tangible difference, that has the potential to better people’s lives and health,” he says.
“In the long run, we are attempting to identify the root cause of diseases like ALS. These types of experiments are really important in doing that,” he adds. “This project does have the potential, I think, to impact people’s lives.”
The ALS Canada-Brain Canada Discovery Grant Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
To find out more about the 2022 ALS Canada-Brain Canada Discovery Grants, read the full press release here.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS.
“I don’t usually do biomedical research – I usually do fundamental biology,” she says. “This project is really exciting because it allows my research to be more meaningful.”
Her winning 2022 proposal focuses on something Dr. Vera Ugalde has studied since her earliest work as a researcher: heat shock proteins (HSPs).
When a protein misfolds in our cells, they no longer do their jobs properly or may become toxic. Fortunately, our bodies don’t leave us hanging: they deploy HSPs, our cellular “paramedics,” to come refold the misshapen protein and help get the cell running again. This process supports protein homeostasis, critical for the balanced, healthy functioning of our cells.
Unfortunately? One type of cell is particularly bad at inducing HSPs in times of stress, even in healthy people: motor neurons.
By the same token, drugs that boost the role of HSPs in repairing damaged cells in other parts of the body don’t seem to work as well in motor neurons – the site of misfolded proteins in people living with ALS.
With the help of her 2022 Discovery Grant, Dr. Vera Ugalde will look closely at why motor neurons are so bad at making HSPs, exploring an entirely new mechanism that could explain the problem.
“Dr. Vera Ugalde’s research could pave the way for groundbreaking discoveries in motor neuron research,” says Dr. Viviane Poupon, President and CEO of Brain Canada.
Her hope is that by finding out the exact reason HSPs work poorly in motor neurons – and why current drugs don’t seem to help – she can help open a new pathway for future therapeutics.
“We have to do this”
The project started when Dr. Vera Ugalde began speaking with her McGill colleague, long-time ALS researcher Dr. Heather Durham, who is also an expert on HSPs.
The two realized quickly that Dr. Vera Ugalde’s technical expertise on protein homeostasis complemented Dr. Durham’s deep understanding of the pathways leading to cellular damage in ALS.
“When we started talking, we said, ‘we have to do this,’” Dr. Vera Ugalde says. “It’s a good synergy.”
The ALS Canada-Brain Canada Discovery Grant Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
To find out more about the 2022 ALS Canada-Brain Canada Discovery Grants, read the full press release here.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS.
Meet the researchers behind the winning 2022 Discovery Grant projects
What role does its sister protein play when restoring G3BP1 levels as a potential ALS treatment strategy?
Award: $125,000
Collaborators: Dr. Marlene Oeffinger, Institut de recherches cliniques de Montréal (IRCM)
Dr. Christine Vande Velde is a cellular biologist at the Centre de recherche du CHUM, Université de Montreal, and one of nine ALS Canada-Brain Canada Discovery Grant recipients for 2022. While she and her team spend their lab hours parsing out biological intricacies, her work is always motivated by something bigger: the lives of people affected by ALS.
Dr. Vande Velde just finished her six-year term on the ALS Society of Canada’s Board of Directors, which has given her face-to-face experiences with people living with ALS and their families.
“It’s been a fabulous experience that changed the way I think about what ALS Canada does. It has helped me always keep the mission front of mind,” she says.
For more than a decade, Dr. Vande Velde’s lab has been part of driving ALS research forward in Canada.
With this award, the research team will seek to understand the function of two “twin” proteins called G3BP1 and G3BP2. The proposal emerged from conversations between two trainees in her lab, a promising sign for the future of Canadian ALS research.
Conventional knowledge would say the two proteins are so similar, they’re identical. But other researchers have shown the prevailing narrative may be wrong, as previous studies reveal that when G3BP1 levels are reduced, G3BP2 cannot fully compensate for the loss.
G3BP1 tends to degrade when a protein called TDP-43 leaves its home in the nucleus – one of the most common hallmarks of ALS and other neurodegenerative diseases. Its twin, G3BP2, does not.
Dr. Vande Velde’s project asks a number of questions about what G3BP1 might do that its sibling can’t, if restoring G3BP1 function might be a therapeutic target for ALS, and more.
“In research, we can’t rely on overgeneralizations (like assuming two “twin” genes have the same function),” she says. “I’m excited to go deep into the biology to understand the nuances. In treatment, the details matter.”
“Regardless of the results, we will learn something about a target we think is druggable and relevant to the disease that we didn’t know before,” she says.
“Dr. Vande Velde is playing a pivotal role in shaping the future of ALS research in Canada. Her ability to think outside of the box makes her a valuable asset in the quest for innovative solutions and advancements,” says Dr. Viviane Poupon, President and CEO of Brain Canada.
“The more we understand the biology, the better we can interpret the results of any clinical trial,” says Dr. David Taylor, Vice-President, Research and Strategic Partnerships, ALS Canada. This is particularly important, as the research will help inform therapeutic strategies that Dr. Vande Velde is also working on in her lab.
At the end of the day, Dr. Vande Velde says she’s grateful to be part of such a robust research community – one that in recent years is producing real therapeutic results for patients.
“I don’t know anybody who works in ALS who’s not dedicated to the cause. Nobody does it as a hobby,” she says.
The ALS Canada-Brain Canada Discovery Grant Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
To find out more about the 2022 ALS Canada-Brain Canada Discovery Grants, read the full press release here.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS.
Meet the researchers behind the winning 2022 Discovery Grant projects
Can this routine and inexpensive procedure have a neuroprotective effect in ALS?
Award: $125,000
Collaborators: Dr. Minh Dang Nguyen, University of Calgary, and Dr. Deepak Kaushik, Memorial University of Newfoundland
Dr. Carlos Rodrigo Camara-Lemarroy is an early-career researcher and clinical neurologist based in Canada at the University of Calgary. He is a first-time recipient of an2022 ALS Canada-Brain Canada Discovery Grant.
In his clinical practice, Dr. Camara-Lemarroy has worked extensively with people experiencing neurodegenerative diseases – from multiple sclerosis (MS) to Parkinson’s disease and ALS.
“As a clinician, it has struck me that despite how much we know about the brain, we have so few therapies for patients,” he says. “Their stories just stick in my soul.”
Motivated by his many patients, Dr. Camara-Lemarroy’s primary research interest is to rapidly translate basic neuroscience research into novel therapies for neurodegenerative disorders. So far, his focus has been on MS. But he realized that many of the diseases he treats as a clinician have similar signatures.
“At the end of the day, across these diseases, brain cells are dying,” he says. “We need to find a way to stop that.”
One of his current research questions surrounds ischemic preconditioning, i.e., the practice of briefly cutting off blood supply to prepare the body to better withstand disease.
In recent years, researchers have found that when they briefly cut off a patient’s blood flow (ischemia) with something as simple as a blood pressure cuff, the body will start to develop natural protective mechanisms, effectively conditioning itself to withstand greater injury.
Dr. Camara-Lemarroy and his team are asking whether ischemic preconditioning could help people with neurodegeneration better ward off the effects of their disease, effectively slowing progression.
Through his 2022 ALS Canada-Brain Canada Discovery Grant, Dr. Camara-Lemarroy is applying this concept in an ALS mouse model to see if there is any effect. Success in animal models could pave the way for the therapy to progress to clinical trials, and if ultimately found to be beneficial, it could lead to an accessible, inexpensive, and non-invasive treatment option.
He’s also leading a clinical trial using a similar concept in MS patients with funding from MS Canada.
“Dr. Camara-Lemarroy’s positive impact on MS research exemplifies the effectiveness of Brain Canada’s One Brain approach,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “This approach recognizes that every discovery has the potential to have an impact on a spectrum of brain diseases and disorders, as well as on our understanding of brain functioning.”
Both projects embody Dr. Camara-Lemarroy’s unique approach to therapeutic discovery.
“I’m trying to find therapies that don’t cost too much, are safe, that anyone can access, and that no one can put a patent on. We need therapies that can work for patients not just in the U.S. and Canada, but around the world. That’s the lens I use when designing studies,” he says.
In addition to working with Dr. Minh Dang Nguyen, also at the University of Calgary, Dr. Camara-Lemarroy is collaborating with Dr. Deepak Kaushik at Memorial University in Newfoundland, making it a cross-Canada collaboration.
“This research is truly novel,” says David Taylor, Vice-President, Research and Strategic Partnerships, ALS Canada. “And Discovery Grants are meant to fund novel ideas. It’s innovative ideas like Dr. Camara-Lemarroy’s that can open up new paths for discovery in ALS research.”
The ALS Canada-Brain Canada Discovery Grant Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
To find out more about the 2022 ALS Canada-Brain Canada Discovery Grants, read the full press release here.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS.
Meet the researchers behind the winning 2022 Discovery Grant projects
Could this new 3D cell culture model help researchers better predict disease progression in ALS?
Award: $125,000
Collaborators: Dr. Yasser Iturria-Medina, McGill University
When Dr. Thomas Durcan, director of The Neuro’s Early Drug Discovery Unit (EDDU), found out his team had been awarded a 2022 Discovery Grant, “It was a very happy day for us all.”
“All” includes his EDDU research associate, Dr. Mathilde Chaineau, and PhD student María José Castellanos Montiel, two of the key players on the winning proposal.
Dr. Durcan’s project uses an innovative model ten years in the making, thanks in large part to researchers at The Neuro (Montreal Neurological Institute-Hospital).
The model uses donor human blood cells that have been transformed into neurons, astrocytes, and microglia – all important cell types in the brain – to create 3D structures called spheroids. By better representing the multidimensional nature of the brain, the spheroids will help scientists discover new interactions and processes that may be happening in a diseased brain.
For the ALS Canada–Brain Canada Discovery Grant project, the team will use blood samples from people living with ALS to create spheroids that will offer new insights into how microglia and astrocytes interact together with neurons in a 3D ALS disease environment. They will then work with Dr. Yasser Iturria-Medina, a computational biologist at McGill University, to analyze what’s happening on the molecular level to generate new insights.
“Through this multimodal approach, we can hopefully find disease signatures that we would miss by just looking at the data in a simpler way,” says Dr. Durcan.
“From a scientific perspective, it’s very exciting for us to get these funds and to see this great project moving forward,” Ms. Montiel adds. “We’re going to build a model to study things that are not very well understood – we’re going to find out something new.”
“This is a very exciting collaboration,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “Dr. Durcan and his team’s innovative approach towards data analysis has the potential to revolutionize the ALS research landscape in Canada.”
Keeping patients at the heart of the work
This research project relies on the generosity of people to donate their blood samples, something all three team members expressed gratitude for.
“Patients are going through a very hard time with their family, but they still find time to consent to give us their samples,” Dr. Chaineau says. “Research is always teamwork, and I include the patients as part of that team.”
When we spoke to the researchers, all three had just attended the 2023 ALS Canada Research Forum, which brings people living with ALS, clinicians, families, and scientists together. It is an excellent opportunity for researchers to put faces to the research projects they dedicate their time to.
At this year’s event, the sense of hope was palpable, the team says.
“The patients being so positive pushed us in the right direction,” says Dr. Chaineau. “Every time we go to an ALS Canada Research Forum, we come back ready to go.”’
“There are people that rely on us to get up every day early in the morning and work and do what we do,” adds Dr. Durcan. “I think that’s what really keeps us motivated.”
Ms. Montiel agrees. “As scientists, it’s nice to know who we’re helping and that we’re giving back to the community.”
Meet the researchers behind the winning 2022 Discovery Grant projects
Could this new mouse model help to understand the potential role of retroviruses in ALS and lead to new treatments?
Award: $125,000
Collaborators: Dr. Jody Haigh, University of Manitoba, and Dr. Domenico Di Curzio, St. Boniface Hospital Albrechtsen Research Centre
When Dr. Renée Douville found out she had been awarded one of the 2022 ALS Canada–Brain Canada Discovery Grants, she was excited. Very excited.
“I screamed so loud, people down the hallway heard me,” she said.
Dr. Douville, a virologist at St. Boniface Hospital Research Centre and the University of Winnipeg, is one of nine grant recipients for 2022. The funding will help her build on nearly 15 years of research that has made her an expert in a niche area of ALS research: the role of endogenous retroviruses (ERVs).
She was awarded alongside her long-time collaborator, Dr. Domenico Di Curzio at St. Boniface Hospital Research Centre, as well as Dr. Jody Haigh at the University of Manitoba.
“It’s particularly exciting because we’ve been working on this project together for a long time, slowly but surely,” she said.
“Brain Canada is proud to support researchers who have demonstrated successful collaboration over many years, emphasizing the importance of teamwork and collaboration in advancing scientific knowledge,” adds Dr. Viviane Poupon, President and CEO of Brain Canada.
Unlike typical viruses, humans are born with dormant ERVs already in their DNA. Dr. Douville and others have shown that some people with ALS display elevated levels of a specific ERV protein, called ERVK, which led to the hypothesis that ERVs, if reactivated, can damage motor neurons.
Dr. Douville has already tested the effect of overexpressed ERVK in cell and fruit fly models. The results? Neuronal damage, similar to what we see in people living with ALS.
The ALS Canada-Brain Canada Discovery Grant will now help her and her collaborators understand the effect of elevated ERVK levels in a mouse model. If the team can further validate that elevated ERVK levels lead to ALS-like symptoms, it could open up new targets for treatment.
The work dovetails with Lighthouse 2, a clinical trial happening in Europe, Australia and New Zealand to see whether targeting retroviruses can benefit people living with ALS. The trial is unique because it is testing a drug already approved to treat people with HIV that has previously been shown to be safe in people living with ALS.
Dr. Douville had started her career at Johns Hopkins looking at ERVs in multiple sclerosis, but she soon realized they had a much stronger signature in ALS. She hasn’t looked back.
“Everything started through scientific curiosity,” she said. “But today, my work with ALS has really changed how I think about endogenous retroviruses. My focus is now more on therapeutics, and how we could treat the damage ERVs do to our bodies…. I’m actually really hopeful that our research will potentially lead to new treatments for patients.”
In addition to excitement, she said the other emotion she feels is gratitude.
“I’m just extremely thankful for all the fundraising that people with ALS and their families do to raise money for our research,” she said. “And I’m thankful to ALS Canada and Brain Canada for taking a chance on us, even though our research area might be further afield.”
The ALS Canada-Brain Canada Discovery Grant Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
To find out more about the 2022 ALS Canada-Brain Canada Discovery Grants, read the full press release here.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS. Interested in supporting projects like these? Donate now and make a difference today.
Meet the researchers behind the winning 2022 Discovery Grant projects
Could the study of neuromuscular junction proteins aid in the development of essential biomarkers?
Award: $300,000
Collaborators: Dr. Danielle Arbour and Dr. Roberta Piovesana at the Université de Montréal, and Dr. Robert Bowser, Barrow Neurological Institute
Dr. Richard Robitaille, at the Université de Montréal, received his first grant from ALS Canada ten years ago. Already a world-leading expert on the neuromuscular junction (NMJ), the funding was his first research foray into ALS and allowed him to find clinical applications for his expertise. His lab now spends nearly all their research efforts on the disease.
Dr. Robitaille’s work demonstrates how powerful Discovery Grants can be for moving ALS research forward.
This year, he received one of the first ever three-year, $300,000 versions of the Discovery Grants. The money will fund a project related to an upcoming clinical trial built upon the foundational research funded by his first Discovery Grant a decade ago.
“Both Brain Canada and ALS Canada are dedicated to supporting research across all phases of a scientific journey,” says Dr. Viviane Poupon, President and CEO of Brain Canada.
The team will use this year’s grant to advance efforts to validate a set of candidate proteins linked to the NMJ as potential biomarkers for ALS treatment and disease progression. Biomarkers are objective measures to track the presence and effect of a disease in the body.
While many neurodegenerative diseases affect the places where neurons connect with other neurons, ALS also impacts the synaptic connection between a nerve and muscle cells – the NMJ.
If successful, using NMJ-linked proteins as biomarkers would have many advantages: changes to the NMJ appear early in the disease, potentially leading to earlier diagnosis and intervention. All forms of ALS lead to changes at the NMJ, making it a possible universal biomarker for the disease. Finally, changes in the NMJ can be tracked using blood samples – something much less invasive for people than a lumbar puncture or tissue sample.
“ALS has a big need for biomarkers, and these are very original. There’s nothing like this that’s been explored in ALS,” says Dr. Robitaille.
In addition, Dr. Robitaille will explore whether the NMJ could also be a target for treatment.
“It’s really exciting to use our basic research to target something with clinical use,” he says.
Dr. Robitaille is working with Dr. Robert Bowser at the Barrow Neurological Institute in Arizona, an international leader in finding biomarkers for ALS.
Beyond the scientific side of his work, Dr. Robitaille is deeply involved with SLA Quebec, a strong partner of ALS Canada, which helps him stay connected to people living with ALS.
“I try to get as practical as possible,” he says. “Science is fun, but there’s an ultimate goal that we should also be useful as fast as possible. That’s the main driver.”
The ALS Canada-Brain Canada Discovery Grant Program has been made possible by the Canada Brain Research Fund (CBRF), an innovative arrangement between the Government of Canada (through Health Canada), Brain Canada Foundation and ALS Canada.
To find out more about the 2022 ALS Canada-Brain Canada Discovery Grants, read the full press release here.
ALS Canada’s mission is to improve the lives of people affected by ALS through support, advocacy, and investment in research for a future without ALS. Interested in supporting projects like these? Donate now and make a difference today.
The landscape of ALS research is ever-changing, as researchers continue to increasingly make developments towards a common goal shared by both the research community and those personally impacted by ALS – to improve quality of life for people living with ALS and to develop more effective therapies, as quickly as possible. At the three-day 2023 ALS Canada Research Forum, we were able to gather in-person for the first time since 2019, directly connecting the world of research to the community experience and enthusiastically convening around a collective commitment to the cause – learning, connecting, and collaborating for change.
This year’s event featured presentations from fundamental researchers, clinicians, and junior researchers from across the country. Things kicked off with an engaging presentation from Dr. Timothy Miller, an instrumental researcher in the development of tofersen (Qalsody), regarding novel therapeutic approaches to treating ALS and highlighting the genesis of the development and evolved trial design of the recently FDA-approved therapy.
The in-depth look into the inspiring discoveries of today sparked key discussion throughout the weekend between researchers and members of the ALS community – the connection serving as a reminder of why we do the work we do. As one participant had to say, “[…] it felt like everyone took away an impactful moment. Something that will help each of us push forward with replenished motivation to fight. Whether it be a personal one, a lived experience or contributions to the long, but not impossible task, of creating a world without ALS, we all have one common thought in mind.”
The Research Forum provided an opportunity for researchers, whether students, postdoctoral fellows, or seasoned investigators, to get together and ask questions, form new ideas, and seek avenues of collaboration. “Every year I feel a renewed sense of hope, inspired by the abundance of knowledge, passion, and dedication everyone brings together in one place in efforts to end ALS,” commented one participant.
Throughout the event, we heard 27 diverse presentations discussing translational and clinical research, fundamental research, and brand-new research being done by young investigators in the field. Each presentation provided not only direct informative take-aways, but simultaneously an opportunity for diverse discourse and questions between researchers, clinicians, and people affected by ALS. Members of the community affected by ALS have a vested interest in research, not only wanting to see treatments that change the reality of the disease but to talk to researchers, understand their work, and provide unique insights – expanding the bounds of what we can learn from each other. This remains a key aspect that the Research Forum continues to cultivate – offering a unique, and diverse avenue of conversation and knowledge exchange between all groups of participants. “I learned more about ALS over the past two days than I have in the past 20 years. I also met some of the most amazing and inspirational clinicians, researchers, sponsors, and of course, people living with ALS and their families,” said one participant.
If there must be only one take away from this year’s Research Forum, it was best said by one of our participants, “staying connected makes us stronger.” The ALS Canada Research Forum is an eye-opening Canadian hub for exceptional connection between all individuals tirelessly working towards and supporting changing what it means to live with ALS.
The ALS Canada Research Forum is made possible thanks to the generous support from platinum sponsor Mitsubishi Tanabe Pharma Canada Inc. (MTP-CA), gold sponsor Amylyx, silver sponsors QurAlis and Biogen Canada, and bronze sponsor Cytokinetics. We thank them for their support of knowledge sharing and capacity building between researchers, clinicians, and people affected by ALS.
I am an ALS scientist. For years, I have been spending my days conducting research in the lab. We are always trying to find the next big discovery, and today I want to tell you something: the biggest discovery of my career almost didn’t happen. But it did, thanks to ALS Canada. And thanks to their generous donors.
Years ago, we were studying the tiny junction between the nerve cells in our spinal cord and our muscles. For years, conventional research had told us this junction wasn’t relevant for ALS research. It was neglected and overlooked.
Due to the long-held belief that paralysis happens in ALS because the motor neurons die, many of my colleagues were surprised when I started applying for funding to help us research this tiny nerve junction further. ALS Canada saw the potential in this research when nobody else would, and they took a risk. With the support of donors, ALS Canada offered me a research grant so I could continue the investigation. On behalf of my research team and the ALS community who serves to benefit from this discovery, thank you to everyone supported these efforts.
Because of that ALS Canada research grant, we now know that there is significance to this research that other scientists had once dismissed. We have proven these tiny nerve junctions don’t simply die when people develop ALS, which is what scientists believed before. We now know that’s not the case! This research unlocked the most important first step, and now that we know “what,” we are working hard to understand “why” this happens.
What started as scientific curiosity became a breakthrough discovery thanks to ALS Canada’s supporters. When I started, there was only one researcher helping me with this work part-time. Now, I have an entire lab of researchers dedicating nearly all of their time to this work. The momentum is building, and I can truly feel the excitement in the lab every day.
Thank you to all who support ALS Canada. Because you do, my important research is possible. Without your support, I may not have been able to continue. But because of you, I still can.
I can’t wait to see where it goes next.
Sincerely,
Dr. Richard Robitaille
ALS Canada funded researcher
Professor, Department of Neurosciences, Université de Montréal
Member of ALS Canada Scientific and Medical Advisory Council
One of the most important safeguards in our bodies is the blood-brain barrier (BBB). Created by tightly interlocked vessel wall cells, the barrier prevents viruses and other microscopic malcontents that could do much harm to our central nervous system from entering our central nervous system.
While the BBB is exceptional at keeping us safe, it poses a problem for people with diseases of the brain: it bars many therapeutics from getting to the places in the brain where they can be most effective.
Dr. Agessandro Abrahao is one of a multi-disciplinary team at Sunnybrook Research Institute that has been grappling with this problem for years.
Thanks to an ALS Canada-Brain Canada Discovery Grant, the team is closer than ever to solving the problem of the BBB using sophisticated, non-invasive technology that has the potential to revolutionize drug delivery for ALS.
Opening the blood-brain barrier
Dr. Abrahao, a neurologist, joined Sunnybrook in 2015 as a fellow under the supervision of his mentor, Dr. Lorne Zinman, the Sunnybrook ALS Clinic director. They began working with Dr. Kullervo Hynynen, Sunnybrook’s vice-president of research and innovation, who had discovered something extraordinary. Using focused ultrasound, you can use sound waves to create “micro-gaps,” like small windows, in the BBB. These gaps remain open for about 24 hours, during which drugs delivered to the bloodstream can “leak” into a target area.
Drs. Abrahao and Zinman wondered: could the focused ultrasound method be applied to the motor cortex? If so, drugs for diseases like ALS could potentially be delivered directly to the areas damaged by the disease, greatly increasing their effectiveness.
Dr. Hynynen’s answer was encouraging: they’d already successfully tested it in animals. It was time to see if it worked in humans.
“Let’s make history”
In 2018, a small cohort of people with ALS volunteered to undergo the procedure for a safety trial, funded by a previous ALS Canada Project Grant. They put on a helmet outfitted with up to a thousand small ultrasound elements in it. Then, using an MRI, the medical team directed the sound waves to the affected area of the brain with surgical precision.
Before the procedure began, the first participant in the trial said, “Let’s make history.”
And they did: the study successfully proved it was possible to safely breach the BBB in the motor cortex.
“The beauty of this technology is that we’re not doing an invasive, open surgery,” said Dr. Abrahao. “We’re using acoustic energy to puncture these small blood vessels and allow the medications to leak to specific regions of the brain. Then, they close up all on their own.”
A trial paved with gold
After this encouraging victory, Drs. Abrahao, Zinman, and Hynynen, Sunnybrook neurosurgeon Dr. Lipsman, and the team – which includes other ALS experts, neurologists, physicists, neurosurgeons, radiologists, and engineers – are now using funding from their latest Discovery Grant to run a Phase 2 clinical trial. Similar to the first trial, it will test using MRI-guided focused ultrasound to create microgaps in the BBB. But this time, there’s an added element (literally): gold.
The trial is partnering with a pharmaceutical company to test the delivery of gold nanoparticles to the brain in people with ALS. Research suggests that gold may be able to improve the metabolism in motor neurons that are degenerating in the brain, helping those cells survive longer. These gold nanoparticles are also currently being tested in ALS as an oral drug in the HEALEY Platform Trial.
This Phase 2 trial will further confirm the safety of the procedure, as well as its efficacy for delivering an actual therapeutic to motor neurons.
Dr. David Taylor, Vice President of Research for the ALS Society of Canada, is optimistic about the potential impact on drug delivery in ALS. “ALS is a disease of both upper and lower motor neurons and most experimental treatment delivery in clinical trial is focused more heavily on accessing the lower motor neurons of the spinal cord. By non-invasively targeting the motor cortex, MRI-guided focused ultrasound, if successful here, could open up avenues not only for new therapies, but also to enhance the efficacy of currently tested treatments in ALS. That prospect is very exciting.”
Brain Canada Chief Research and Programs Officer, Dr. Catherine Ferland agrees. “The potential is very exciting as it has implications for therapies that extend beyond ALS. The impact of this study will be significant for other neurological diseases where the BBB prevents drug entry, and it will inspire solutions that can make a big difference in the research landscape.”
Funding that makes an impact
Since 2014, ALS Canada’s partnership with Brain Canada has resulted in more than $24 million being invested in leading-edge ALS research that has helped further understanding of the disease. The Discovery Grant Program is designed to fuel innovation that will accelerate our understanding of ALS, identify pathways for future therapies and optimize care to improve quality of life for people and families affected by this devastating disease. In 2022, nine projects awarded through the 2021 Discovery Grant Program will benefit from $1.125 million in funding.
The Discovery Grant Program has been made possible with the financial support of Health Canada, through the Canada Brain Research Fund, an innovative arrangement between the Government of Canada (through Health Canada) and Brain Canada, and of the generosity of provincial ALS Societies, ALS Canada donors and community-based efforts, including 40 per cent of net proceeds from the Walk to End ALS.
Please consider making a donation to help create a future without ALS.
Neurological imaging has advanced by leaps and bounds in the past two decades. Researchers can now “see” details of biological processes at a level never previously imagined.
But we still aren’t using imaging to its full potential, says Dr. Freimut Juengling, a nuclear oncologist, neuroscientist and director of the PET/MR centre at the University of Alberta. He notes that brain scientists have some of the most sophisticated technology available for diagnosis, but he insists that there is still more that can be done to use imaging to advance treatment.
With help from an ALS Canada-Brain Canada Discovery Grant, Dr. Juengling and his team are pushing the boundaries of medical imaging and exploring new pathways for ALS treatment in the process.
From bench to bedside
Effective therapies for ALS are still elusive. One pathway that could be promising is the BDNF/TrkB signaling pathway, which sees changes in some ALS patients.
Researchers actually tested BDNF/TrkB-related treatments for ALS in the 1990s, but unfortunately the therapy didn’t live up to its potential. However, since that time, some researchers have considered whether this pathway deserves a second look as there have been questions around the effectiveness of the delivery method used in those studies.
The University of Alberta is home to a state-of-the-art piece of equipment called a PET/MR that offers the possibility of answering new insights into how BDNF/TrkB actually functions. PET/MR is a powerful tool that can “watch” chemical interactions as they happen in a patient’s body, allowing for a much sharper picture of what’s really going on inside someone.
Last year, a team of researchers at the University of Alberta developed a new PET tracer (a specific solution injected into the blood before a PET scan that allows researchers to watch chemical interactions happen in real-time) to investigate the BDNF/TrkB pathway using the PET/MR. Then, a team at McGill used the tracer to capture information about the pathway in non-ALS patients.
With the Discovery Grant, Dr. Juengling will use the technology to examine the pathway in people with ALS, comparing the results with those from participants without ALS and, hopefully, mining critical insights into how this pathway might be used to better understand the disease and possibly provide treatment.
“It was really exciting to get the grant,” says Dr. Juengling. “It’s enabling research that’s from bench to bedside. What everybody is dreaming of is getting to bedside diagnostics, and possibly therapeutics. This grant is a big boost into this possibility.”
“Once we get the whole infrastructure working, this may relate to other neurodegenerative diseases as well,” he added.
“This study is going to utilize modern techniques in a way that will really help us figure out whether or not we should be taking another look at the BDNF/TrkB pathway in ALS,” said Dr. David Taylor, Vice President of Research for the ALS Society of Canada.
New ideas from oncology imaging
Dr. Juengling started as a neurosurgeon when the first neuro-imaging machines using PET were being introduced. At the time, he was asked to train colleagues on how to interpret the neuro-images – and he never went back.
“I’m an imaging guy,” he said. “It’s fascinating to look into the living brain. I just got caught by it.”
Just last year, he decided to move to Alberta from his home country in Switzerland because his oncologic imaging expertise was requested to run the PET/MR program at the Cross Cancer Institute in Edmonton. Here, colleagues had developed the new tracer, initially designed for tumor research. When he had a closer look at the molecular structure, he immediately saw its potential for ALS using PET/MR. Getting a grant to start neuro-research with the machine within the Cancer Institute was an unexpected surprise, and a perfect starter for a new neuro program.
“I’m very grateful that ALS Canada and Brain Canada offer this type of grant,” he said. “Because getting funding for the first step of novel research is always the hardest.”
Link with CAPTURE ALS a huge benefit
For Dr. Taylor, the other major advantage of this research is its link with CAPTURE (Comprehensive Analysis Platform To Understand, Remedy, and Eliminate) ALS.
CAPTURE ALS is a national platform that provides the systems and tools necessary to collect, store, share, and analyze substantial amounts of information about ALS, creating the most comprehensive biological picture of people with ALS ever.
Data from the PET tracer study will contribute a new type of data to the platform.
“The PET tracer is still evolving technology and isn’t used much in ALS research – but we should be doing more with it,” said Dr. Taylor. “This study is well-designed and will hopefully help set the stage for more PET imaging studies to be done in the future with ALS.”
Funding that makes an impact
Since 2014, ALS Canada’s partnership with Brain Canada has resulted in more than $24 million being invested in leading-edge ALS research that has helped further understanding of the disease. The Discovery Grant Program is designed to fuel innovation that will accelerate our understanding of ALS, identify pathways for future therapies and optimize care to improve quality of life for people and families affected by this devastating disease. In 2022, nine projects awarded through the 2021 Discovery Grant Program will benefit from $1.125 million in funding.
The Discovery Grant Program has been made possible with the financial support of Health Canada, through the Canada Brain Research Fund, an innovative arrangement between the Government of Canada (through Health Canada) and Brain Canada, and of the generosity of provincial ALS Societies, ALS Canada donors and community-based efforts, including 40 per cent of net proceeds from the Walk to End ALS.
Please consider making a donation to help create a future without ALS.
History and context
Canada has a rich history in ALS clinical trials. Though an ALS research community developed here alongside the rest of the world in the late 1970s and 1980s, Dr. Andrew Eisen at Vancouver General Hospital is widely regarded as the first ALS clinical trialist in the country, leading a double blind, placebo-controlled study of lamotrigine in the early 1990s. At that time, most clinical trials were performed at a single site and were academically driven, which means the clinician conceives the idea, designs the study, and often pays for it with a limited research grant.
However, as the 1990s progressed, an informal network of Canadian ALS clinicians began collaborating and participating in trials that included testing treatments in partnership with pharmaceutical companies. In industry-supported trials, the company designs the study, often in conjunction with clinicians, and pays for the sites to perform the testing. Pharmaceutical company funding allowed for larger, multi-centre, and multi-national clinical trials that could provide more robust statistical analysis to determine if something works or not. Unfortunately, through the 2000s, our still nascent understanding of ALS limited the number of companies willing to invest in ALS clinical trials and only a handful were developing treatments at any one time.
The network of ALS clinics to support clinical trials across Canada was formalized in 2008 as the Canadian ALS Research Network (CALS) through a collaboration with ALS Canada and under the initial leadership of Dr. Lorne Zinman at Sunnybrook Hospital in Toronto. The establishment of CALS was bolstered by a collaborative clinical trial of lithium chloride with the Northeast ALS Consortium (NEALS) in the United States, a study that ultimately failed, but visibly solidified the network for wider engagement in clinical trials run by pharmaceutical companies. By 2012, Cytokinetics had selected 12 sites across Canada to participate in the BENEFIT-ALS clinical trial of tirasemtiv, and CALS clinics have since participated in more than 20 different industry-sponsored studies over the past decade.
Where we are today
ALS Canada plays an important supportive role in bringing new experimental and proven treatments to Canada. By establishing early relationships with industry who are invested in clinical development of new treatments, ALS Canada helps to facilitate connections with Canadian ALS clinical trial sites and strives to continually keep Canada on every company’s radar. Whether we are advocating for a promising experimental treatment to be tested in Canada during the next trial phase or creating relationships to keep Health Canada top of mind should a company demonstrate efficacy in a trial elsewhere, the work is always ongoing to advocate for Canadians to have faster access to effective treatments.
As of 2022, Canada joins countries like the United Kingdom and Italy as top tier clinical trial sites behind the United States for testing the most promising experimental therapeutics from Phase 1 to Phase 3. This comes not only from having world-class clinics and ALS specialists, but also a long-term collaborative investment in infrastructure that supports trial access and capabilities. Canada has a large ALS research community with strong and varied expertise that can support enhancements like biomarker studies, CAPTURE ALS— a cross-Canada platform for biomarker discovery and better understanding of the differences between people living with the disease that is designed to support collaboration with pharma —and the Canadian Neuromuscular Disease Registry, which has become a very exciting resource for post-marketing real world evidence analyses.
Despite all of this, there are still things we can do to improve Canada’s standing as a primary destination for the most promising clinical trials. Pharmaceutical and biotechnology companies who wish to test their treatment in ALS have many important considerations when deciding which countries and clinics they want to work with to run their clinical trials. One of the key considerations is the capacity to recruit participants, as the faster this happens, the better; for them and everyone. When meeting with industry, ALS Canada highlights the value of our world-class clinicians and clinics, the CALS Network and our organization’s role as a central facilitator, but we are only able to look to past recruitment numbers and reputation to extrapolate the demand on the part of Canadians to contribute to research. Moving forward, having a tangible indication of this enthusiasm to relay will only strengthen the desire for companies to do their research here and to consider Canada a primary market for their treatment once proven.
It’s also not lost on ALS Canada that there are disparities in clinical trial access depending on where someone lives. The Clinical Research Fellowship is offered annually to support a young neurologist to focus their efforts on ALS care and research, building capacity in ways that can also support underserved areas to access clinical trials. Dr. Kerri Schellenberg, the current Chair-Elect of the CALS Network and the first recipient of this fellowship in 2007, has brought clinical trials and multidisciplinary care to Saskatchewan for the first time. With additional support the collaboration of ALS Canada and CALS could also pursue creative ways of advocating for trials nationwide such as virtual trials or supporting clinics differently to enhance trial capabilities.
Take Action
This ALS Awareness Month, ALS Canada is asking you to raise your hand in support of more opportunities to participate in ALS research and a desire to see Canada as a “must have” destination for industry in their future trials.
Share this blog post and our social media posts this June to raise awareness within your community about clinical trials and the work ALS Canada does as it relates to ALS clinical trials.
Our understanding of ALS is evolving at a rapid pace, and the resulting experimental treatments now being developed, based on stronger ALS science, are expected to have a greater potential for efficacy. By helping us create Canada as a clinical trials destination, our clinicians will have more opportunities to host the most promising studies. Further investment in clinical development in Canada will also support longstanding efforts to start, maintain and bring back clinical trials to underserviced regions of the country such as British Columbia, Manitoba, Nova Scotia, and more. Building capacity nationwide will enhance opportunities for all Canadians and has always been an important goal for both the CALS Network and ALS Canada.
Help us strive towards a goal of equitable, affordable, and timely access to the most promising experimental and proven ALS treatments available.
TDP-43 is a protein that lives in the nucleus of our cells. But in at least 97 per cent of ALS patients, TDP-43 somehow finds its way out of the nucleus – also called “mislocalization” – and starts to “clump” up in the cell. There are still a number of questions to answer about this process: is it the loss of TDP-43 in the nucleus of motor neurons that leads to symptoms? Or is it the presence of these unusual clumps in the wrong place that causes trouble? Maybe both?
Because it is such a common phenomenon in patients, many current ALS studies are dedicated to finding out the whats and whys of this mislocated protein.
That includes Dr. Maxime Rousseaux, a neuroscientist at the University of Ottawa. He and his team are using an ALS Canada-Brain Canada Discovery Grant to dig deeper into the biology of TDP-43 mislocalization, particularly how a process called SUMOylation might be involved. What they find could lead to a better understanding of one of the most common biological markers of ALS – and new avenues for treatment.
The key question: why cells go from healthy to not healthy
One of Dr. Rousseaux’s primary research focuses is understanding how and why proteins end up in the wrong place in cells involved in neurodegenerative diseases such as Parkinson’s. But this new focus on ALS was inspired by a doctoral student in his lab, Terry Suk, who received a Trainee Award in 2019 supported by ALS Canada and Brain Canada.
“Terry has really been the driving force behind this research, intellectually and experimentally,” said Dr. Rousseaux.
Terry came to the Rousseaux lab in 2018 with a nagging question in his mind: how do cells go from being healthy to not healthy?
“It’s a bit of an obsession at this point,” Terry said.
Under Dr. Rousseaux’s guidance, Terry had been studying SUMOylation, a standard biological process that regulates different functions in a cell. Very few researchers, however, have looked at how SUMOylation might work in relation to TDP-43. Terry wondered: is there a link between TDP-43 mislocalization and SUMOylation that hasn’t been seriously considered yet?
This question led to their Discovery Grant, which is powering a number of experiments to explore the connection between SUMOylation and TDP-43. In one experiment, they are blocking TDP-43 SUMOylation in mice to see if ALS-like symptoms develop. Another will explore ways to monitor SUMOylation in human samples (as opposed to animal models), which could lead to a new biomarker for ALS.
“Dr. Rousseaux and his team are trying to better understand the biology to see if it’s relevant in helping researchers find a way to treat the disease,” said Dr. David Taylor, Vice President of Research for the ALS Society of Canada. “Every little bit we can uncover is a step in the right direction for people living with ALS.”
A reconnection at the right time
One of Dr. Rousseaux’s key collaborators on the project is Dr. Martin Duennwald, a cell biologist at Western University – who also happened to be one of Terry’s undergraduate honors thesis advisors. The researchers connected after Dr. Duennwald heard Terry give a presentation on an earlier iteration of the research at an ALS Canada Research Forum.
“He messaged me saying, ‘This is really cool. Can we talk about it?’” Dr. Rousseaux said.
Now they are working together on a high-potential project that could illuminate key biological mechanisms potentially involved in ALS.
“We appreciate when a Discovery Grant can fund this type of cross-institutional collaboration,” said Dr. Catherine Ferland, Chief Research and Programs Officer at Brain Canada. “When resources and talent come together like this, the potential for impact becomes much greater.”
Motivated by an inspiring community
Dr. Rousseaux said he is excited that the Discovery Grant is allowing him to focus a larger portion of his lab time and resources on ALS – something he has found rewarding so far.
“I am extremely inspired by how organized and motivated the ALS community is,” he said, noting that despite being relatively new to ALS research, the community has embraced him wholeheartedly. “I have felt incredibly welcomed to the community, and am so grateful for its willingness to fund us, even at this early stage of research.”
“It just makes me want to work that much harder,” he added.
Funding that makes an impact
Since 2014, ALS Canada’s partnership with Brain Canada has resulted in more than $24 million being invested in leading-edge ALS research that has helped further understanding of the disease. The Discovery Grant Program is designed to fuel innovation that will accelerate our understanding of ALS, identify pathways for future therapies and optimize care to improve quality of life for people and families affected by this devastating disease. In 2022, nine projects awarded through the 2021 Discovery Grant Program will benefit from $1.125 million in funding.
The Discovery Grant Program has been made possible with the financial support of Health Canada, through the Canada Brain Research Fund, an innovative arrangement between the Government of Canada (through Health Canada) and Brain Canada, and of the generosity of provincial ALS Societies, ALS Canada donors and community-based efforts, including 40 per cent of net proceeds from the Walk to End ALS.
Please consider making a donation to help create a future without ALS.
We use cookies and other technologies to understand how you use our site and to improve your experience and provide you with valuable content. By continuing on this website, you consent to the use of these cookies. For more information and to opt-out of cookies, visit the cookie section of our privacy policy.
This site is registered on wpml.org as a development site. Switch to a production site key to remove this banner.
