July 2026

NEUROSCIENCES

SCIENTISTS UNCOVER CAUSE OF NEURONAL HYPEREXCITABILITY, IDENTIFY NEW TARGET FOR ALS TREATMENT

In a recent study published to Nature Neuroscience, Northwestern Medicine scientists identified a mechanism behind neuronal hyperexcitability in ALS and developed a gene-targeted therapy to potentially reverse it. The team included Evangelos Kiskinis, PhD, and Robert Kalb, MD.

What Clinicians Should Know
  • Dysfunction of the protein TDP-43, a hallmark of most ALS cases, disrupts an ion channel that normally helps regulate neuronal activity.
  • This disruption causes neuronal hyperexcitability, a common feature of ALS that has been associated with worse clinical outcomes.
  • Using patient tissue and stem cell-derived human neurons, investigators identified how TDP-43 alters the molecular processes that control neuronal firing.
  • Then, the team developed an antisense oligonucleotide to correct the underlying RNA defect and restore normal neuronal activity.
  • The therapy is moving toward preclinical testing with the goal of advancing to human studies.
Evangelos Kiskinis, PhD, Associate Professor of Neurology and Neuroscience at Northwestern Medicine
Robert Kalb, MD, the Joan and Paul Rubschlager Professor and Director of the Les Turner ALS Center at Northwestern Medicine

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