Hande Ozdinler
Associate Professor of Neuromuscular Disease in the Ken & Ruth Davee Department of Neurology Northwestern University
Seminars
Thursday 4th June 2026
Targeting the Cortical Component of ALS: Upper Motor Neurons, Circuit Complexity, and New Paths for Drug Development
11:30 am
- 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
Wednesday 3rd June 2026
Panel Discussion: What Happens Upstream of TDP-43 Misfolding? Shifting Focus from Restoring Function to Understanding Root Cause of Aggregate Pathology
10:00 am
- What are the earliest cellular events that trigger TDP-43 mislocalization and aggregation, and how do these upstream mechanisms vary across ALS subtypes?
- How do general protein homeostasis pathways, including nuclear import/export and stress response mechanisms, influence TDP-43 pathology?
- Which cryptic exons, epigenetic changes, or C9orf72-related mechanisms are likely contributors to disease initiation, and how should these inform target prioritization?
- How can we improve ALS model systems to better reflect the physiological context of TDP-43 misfolding and aggregate formation?
- Moving beyond restoring TDP-43 function, what strategies can drug developers adopt to intervene at the root cause of aggregate pathology?
