Targeting the Cortical Component of ALS: Upper Motor Neurons, Circuit Complexity, and New Paths for Drug Development

• ALS is a dual‑neuron disease: upper and lower motor neurons are distinct populations, and ignoring the cortical component limits therapeutic success
• Upper motor neurons are central to ALS heterogeneity and progression, making their survival requirements critical for translational drug discovery
• NU‑9 is the first compound shown to improve upper motor neuron health, with IND progress and efficacy across ALS, FTD, and Alzheimer’s via shared neurodegenerative mechanisms
• Incorporating upper motor neuron efficacy into preclinical decision‑making enables smarter trial prioritisation and effective combination therapies, including synergy with riluzole and edaravone

Speakers:

Hande Ozdinler

Associate Professor of Neuromuscular Disease in the Ken & Ruth Davee Department of Neurology

Northwestern University