LEUCALS: Meet the Labs working together to fight ALS
May 19, 2023
Ludo Van Den Bosch (left), Philip Van Damme (center), and Sandrine Da Cruz (right), ALS researchers at the VIB-KU Leuven Center for Brain & Disease research
ALS (or Amyotrophic Lateral Sclerosis) became a household name in 2014 when the Ice Bucket Challenge (see an example from the Neurobiology lab below) took the internet by storm, making a massive impact in raising awareness of and accelerating the fight against the disease.
In this devastating disorder, motor neurons – cells in the central and peripheral nervous system that transmit signals to make muscles work – degenerate, leading to muscle weakness and wasting that gets worse over time. Despite significant increases in research funding and efforts, effective treatments for ALS are still lacking. Most people die between 2-5 years of disease onset.
Leuven Center for ALS Launch
This May – which also marks International ALS Awareness Month – signals the start of the Leuven Center for ALS (or “LEUCALS”). The launch event provided a platform for this crucial initiative to introduce itself and share its vision and mission within the ALS research community.
Philip Van Damme is a neurologist, professor, and researcher at UZ Leuven and the VIB-KU Leuven Center for Brain & Disease Research. He opened the event by introducing LEUCALS' vision, mission, and goals. "LEUCALS is a collective of top ALS research labs that are dedicated to advancing the understanding of ALS and finding new treatments for this devastating disease," Philip explains. "It aims to bring together both clinical and preclinical research groups in order to stimulate collaboration as well as accelerate research into mechanisms, biomarkers, and treatments.”
Meet some of our Labs working on ALS
Philip has worked closely with Ludo Van Den Bosch – group leader of the Lab of Neurobiology at the VIB-KU Leuven Center for Brain & Disease Research – on ALS research for over 20 years. Earlier this year, Ludo won the prestigious Generet Award, a one-million-euro prize to fund research into rare diseases. At the LEUCALS launch event, Ludo shared more about a potential new therapeutic strategy for ALS: the inhibition of the enzyme HDAC6. Both Philip and Ludo work in close collaboration with ALS patient organization ALS Liga België, whose CEO and Chairwoman Evy Reviers also spoke at the launch.
The latest ALS researcher to join the VIB-KU Leuven Center for Brain & Disease Research is Sandrine Da Cruz, who started her Lab of Neurophysiology in Neurodegenerative Disorders as recently as three years ago, in 2019. Sandrine has been working on understanding the complex mechanisms of neurodegeneration, with the goal of working towards therapy treatment in ALS. I asked Sandrine what she’s most excited about the LEUCALS project: “The LEUCALS inauguration symposium was a real success, full of vibrant science. The multidisciplinary, highly synergistic research that was presented is really exciting, and I am sure it will foster great collaborations and advance our progress towards therapy development to combat ALS.”
The Future of ALS Research and Therapy
The LEUCALS launch ended on a hopeful note for the future of ALS research and treatment. We heard from Professor Don Cleveland – Breakthrough Prize awardee and one of the event's two highly distinguished keynote speakers (not to mention Sandrine Da Cruz’s former postdoctoral research supervisor!), the other being Professor Dame Pamela Shaw – who shared the inspiring story behind the recently FDA-approved ALS treatment, which started in his lab alongside researchers Tim Miller, Richard Smith, Melissa McAlonis Downes, and Frank Bennett (Ionis Pharmaceuticals).
"In 2004, we had an idea: can we develop a designer DNA drug which – when introduced into the nervous system – can lower expression of a gene whose expression is implicated in disease?" Don explains. You could feel the anticipation amongst the LEUCALS attendees across the auditorium. "Everyone told us this wouldn't work. All the cell biology and pharmacology textbooks at the time said that DNA drugs were too big and too heavily charged to enter cells.”
After a slight pause, Don grinned characteristically: "Turns out the cells of the nervous system hadn't read those textbooks.” The result is Tofersen: an antisense oligonucleotide-targeting SOD1-based drug. This is a landmark achievement, as the first approved drug in existence for this ALS familial form.
I asked Ludo Van Den Bosch to share some final thoughts in light of the groundbreaking news: "The launch event of LEUCALS was perfectly timed in view of the accelerated approval by the FDA of Tofersen. Apart from a new therapy for a relatively small group of ALS patients, Tofersen proves that ALS is a curable disease. This is another promising step towards a world without ALS, the mission of the labs participating in LEUCALS.”
Pictures from the LEUCALS launch. ©Robert Stevens