Northwestern University hosted the 14th annual Les Turner Symposium on ALS, gathering scientists, clinicians, and members of the ALS community to share progress and research updates in understanding and treating amyotrophic lateral sclerosis (ALS). The symposium, held both in-person and virtually, focused on the progressive neurodegenerative disease that affects motor neurons, leading to muscle weakness.
Latest Research Unveiled
Hande Ozdinler, PhD, associate professor of Neurology, welcomed attendees, emphasizing the dedication to finding effective cures for ALS. Currently, approximately 350,000 people worldwide are living with ALS, with an average survival rate of three years.
Robert Kalb, MD, director of the Les Turner ALS Center, highlighted the progress in clinical trial development but noted the gap between research and potential cures. His lab's findings on genetic screening identified components of protein imbalance in ALS and other neurodegenerative diseases, characterizing how this leads to misfolded proteins.
Lindsey Hayes, MD, PhD, from Johns Hopkins University, presented her work on the cellular and molecular mechanisms regulating TDP-43, a major pathological hallmark of ALS and a key therapeutic target. Hayes stated her lab's interest in understanding TDP-43 transport mechanisms to rescue dysfunction in disease.
David Gate, PhD, assistant professor of Neurology, discussed the relationship between the immune system and ALS, focusing on genetic differences in immune cells of ALS patients. Marco Martina, MD, PhD, professor of Neuroscience, shared his research on the electrical behavior of different neurons in the motor cortex at an early stage of ALS.
Keynote on Clinical Trial Innovation
Angela Genge, MD, professor of Neurology and Director of the ALS clinic at The Montreal Neurological Institute-Hospital, delivered the keynote address. As an international leader in clinical trial design for rare neurological conditions, Genge detailed her work with federal agencies to improve biomarker identification for ALS.
Genge advocated for standardizing patient quality-of-life measurements in ALS trials, including data on patient mobility and activity limitations. She emphasized the importance of the patient voice in clinical trials and the need for more robust trials using both patient input and scientific biomarkers.
Agile Clinical Trial Designs
Genge also urged scientists to consider more agile clinical trial designs, testing more drugs and conducting multiple analyses in shorter periods to accelerate the discovery of new treatments. Following the presentations, a panel including Northwestern physicians, Les Turner ALS Foundation staff, and people living with ALS addressed questions about emerging ALS treatments, care, and community support.
