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Updated: May 3, 2026

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
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Opportunities for improving therapy development in ALS

Lucie Bruijn1, Merit Cudkowicz,

  • 1ALS Association , Washington, D.C.

Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration
|January 30, 2014
PubMed

Insights

To improve amyotrophic lateral sclerosis (ALS) therapy development, future clinical trials should target specific patient subgroups with biomarkers and incorporate innovative designs with patient advocate involvement.

Area of Science:

  • Neurology
  • Clinical Trials
  • Biomarkers

Background:

  • Stakeholders convened in May 2013 to discuss challenges in amyotrophic lateral sclerosis (ALS) therapy development.
  • The meeting involved The ALS Association and The Northeast ALS Consortium (NEALS).

Purpose of the Study:

  • To summarize issues and propose new directions for improving ALS therapy development.
  • To provide recommendations for future clinical trial designs in ALS.

Main Methods:

  • A round-table discussion among stakeholders was convened.
  • Issues and potential new directions were identified and summarized.

Main Results:

  • Future phase II clinical trials in ALS should target specific patient subgroups based on likely involvement in pathogenesis.
  • Trials should include biomarkers to monitor clinical progression and pharmacodynamic engagement.
  • Innovations in trial structure, design, and patient advocate involvement are recommended.

Conclusions:

  • Targeted therapies with biomarkers are crucial for advancing ALS treatment.
  • Improving clinical trial design and patient engagement can enhance therapeutic development for ALS.
Keywords:
Amyotrophic lateral sclerosisbiomarkersclinical trialstherapy

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