December 2025

NEUROSCIENCES

THE FUTURE OF ALS RESEARCH: BIOMARKERS, GENETICS AND TRIAL INNOVATION

Over the last decade, amyotrophic lateral sclerosis (ALS) research has undergone a profound transformation. Advances in trial design, biomarker integration and genetic therapies are reshaping how clinicians approach this complex disease.
In this NeurologyLive Q&A, Ryan Donaghy, MD, shares his perspective on what these changes mean for neurologists and clinical practice.

Key shifts in ALS research
  • Multicenter networks: Trials moved from isolated centers to global platforms, improving patient access and accelerating timelines
  • Adaptive platforms: HEALEY ALS trial model enabled multiple therapies to be tested under one infrastructure, reducing setup time and increasing efficiency

​Biomarkers: Changing the game
  • Neurofilament: Early detection of biological changes before clinical symptoms; pivotal in tofersen approval
  • Longitudinal biobanking: Serial CSF and blood collection linked to clinical data speeds understanding of disease mechanisms

Genetics: The future of care
  • Genetic profiling: Expected to become standard for all ALS patients
  • Personalized trials: Combining genetic insights with biomarkers enables precision medicine

Practical considerations for clinics
  • Genetic counseling: Clinicians must guide patients on family implications and treatment decisions
  • Infrastructure: Intrathecal delivery requires partnerships with procedural teams

Lessons from other disorders
  • Breakthroughs in spinal muscular atrophy and Duchenne show the power of targeted genetic therapies and standardized measures — principles now shaping ALS trials
Ryan Donaghy, MD, Assistant Professor of Neurology (Neuromuscular Disease) at Northwestern Medicine

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