December 2025

NEUROSCIENCES

EXPLORING EMERGING THERAPEUTIC TARGETS IN NEURODEGENERATIVE DISEASE

In an interview with NeurologyLive, Robert G. Kalb, MD, director of the Les Turner ALS Center at Northwestern Medicine, explains his lab’s approach to identifying genetic therapeutic targets for neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

In the Kalb Neuronal Biology Laboratory, researchers use preclinical models, including genetically engineered mice, primary neuron cultures, induced pluripotent stem cell (iPSC)–derived neurons and C. elegans, to investigate mechanisms driving neuronal stress and protein misfolding involving SOD1, TDP‑43 and C9ORF72.

Through unbiased genetic screens, the team has identified suppressor genes that restore proteostasis and reduce toxicity. These findings inform a translational approach focused on targets that can be modulated with antisense oligonucleotides (ASOs) or tractable small molecules.

Key takeaway: Gene‑level interventions with promising preclinical results are emerging as viable strategies to alter disease biology toward and move toward clinical impact.
Robert G. Kalb, MD, Director of the Les Turner ALS Center, Chief of Neuromuscular Disease in the Department of Neurology, the Joan and Paul Rubschlager Professor and Professor of Neurology (Neuromuscular Disease) at Northwestern Medicine

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