Northwestern scientists, clinicians and members of the ALS community gathered in the Feinberg Pavilion of Northwestern Memorial Hospital for the 14th annual Les Turner Symposium on ALS to celebrate progress and share the latest research in hopes of better understanding and treating the disease.
The daylong symposium, held in-person and virtually on November 4, featured sessions highlighting new research on amyotrophic lateral sclerosis, or ALS, a progressive neurodegenerative disease that deteriorates motor neurons in the brain and spinal cord, causing muscle weakness.
Currently, an estimated 350,000 people worldwide live with ALS, which has an average survival rate of three years.
“I’d like to thank everyone for your dedication, for coming together and trying to find effective cures for ALS,” said Hande Ozdinler, PhD, associate professor of Neurology in the Division of Neuromuscular Disease and one of the symposium’s organizers, as she welcomed attendees to this year’s symposium.
The symposium is sponsored by the Les Turner ALS Center at Northwestern Medicine, which aims to accelerate cutting-edge research and provides education to people living with ALS and their families. The Les Turner ALS Foundation established the center to bring ALS research, clinical and educational activities under one organization.
“This event is one of the most important gatherings for the ALS community and each year, it brings together leading scientists, clinicians, researchers and people living with ALS with a shared commitment to understanding and ultimately defeating this disease,” said Laura Freveletti, chief executive officer of the Les Turner ALS Foundation. “Together, we’re chipping away at ALS one discovery, one connection and one step at a time.”
“We’ve made enormous progress in clinical trial development and in getting interventions to patients as quickly as possible,” said Robert Kalb, MD, the Joan and Paul Rubschlager Professor of Neurology and director of the Les Turner ALS Center. “But there is still a gap between translating research into a potential cure for patients. Symposia such as these are a way to close that gap.”
Kalb, who is also chief of Neuromuscular Disease in the Ken and Ruth Davee Department of Neurology, went on to present the latest findings from his laboratory. Using genetic screening, Kalb and his collaborators identified components of the protein imbalance that is present in ALS and other neurodegenerative diseases and characterized how it led to misfolded proteins.
Lindsey Hayes, MD, PhD, associate professor of neurology at the Johns Hopkins University School of Medicine and Brain Science Institute, presented her work identifying the cellular and molecular mechanisms that regulate TDP-43. The dysregulation of the gene and proteins is a major pathological hallmark of ALS and an important therapeutic target.
“My lab is particularly interested in the mechanisms that regulate TDP-43 transport with the hopes that better understanding of the molecular regulation of these events will allow us to understand TDP-43 disruption in disease and to develop approaches to rescue that dysfunction,” Hayes said.
David Gate, PhD, assistant professor of Neurology, discussed the relationship between the immune system and ALS.
“What we’re interested in is how the genomic code in cells of the immune system in ALS patients might be different from that of healthy people,” Gate said. “Believe it or not, this really hasn’t been done before with this type of sophisticated assay.”
Gate’s research identified genetic differences in the immune cells of ALS patients, information which may help inform future treatments.
Marco Martina, MD, PhD, professor of Neuroscience, shared his research which explores how different neurons in the motor cortex behave electrically at an early stage, before ALS symptoms appear.
This year’s keynote address was delivered by Angela Genge, MD, professor of Neurology and Director of the ALS clinic at The Montreal Neurological Institute-Hospital.
Genge, an international leader in clinical trial design and development for rare neurological conditions, detailed her journey working with federal agencies to improve biomarker identification for ALS.
In her research, Genge advocates for standardizing patient quality-of-life measurements in ALS trials and including data about patient mobility and activity limitations. These factors, which are not captured in all clinical trials, are crucial to understanding treatment benefits for people living with ALS, Genge said.
“We need to be listening to the patient voice more in clinical trials,” Genge said. “My aim is to improve our use of patients themselves and to improve our use of science for biological biomarkers to make for more robust trials.”
Genge also implored scientists to consider using more agile clinical trial designs, in which more drugs are tested, and multiple analyses are conducted during shorter periods of time, to accelerate the discovery of new treatments.
Following scientific presentations and research poster presentations, a panel including Northwestern physicians, Les Turner ALS Foundation staff and people living with ALS answered questions about emerging ALS treatments, ALS care and community support.
