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Updated: May 13, 2026
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ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
Published on: July 29, 2007
Bridging the Funding Gap in Drug Development for Amyotrophic Lateral Sclerosis
Pineal Iyassu Bareamichael1, Suma Babu2, Trang Hoang1,3
1Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD.
Neurology. Clinical Practice
|May 11, 2026
Summary
Developing effective treatments for Amyotrophic Lateral Sclerosis (ALS) requires overcoming challenges in preclinical models and clinical trials. Innovative funding and research ecosystems are crucial for accelerating ALS therapy development.
Area of Science:
- Neurodegenerative diseases
- Drug development
- Clinical trial design
Background:
- Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with limited therapeutic options.
- Current FDA-approved ALS drugs offer modest benefits, highlighting the need for improved treatments.
- High clinical trial failure rates and insufficient funding impede ALS drug development.
Purpose of the Study:
- To identify key challenges in Amyotrophic Lateral Sclerosis (ALS) drug development.
- To propose solutions for accelerating the development of effective ALS therapies.
- To suggest a sustainable research and funding ecosystem for rare neurodegenerative disorders.
Main Methods:
- Review of existing ALS drug development landscape, including approved therapies and clinical trial outcomes.
- Analysis of challenges in preclinical models, early-phase trials, and late-stage development costs.
- Examination of current federal initiatives and funding gaps for ALS research.
Main Results:
- Only three drugs (riluzole, edaravone, tofersen) have been approved since 1995, with limited efficacy.
- Significant hurdles include poor preclinical model translatability, underpowered trials, and high development costs.
- Existing federal initiatives face challenges in securing adequate funding for large trials and research networks.
Conclusions:
- Strengthening national registries, expanding adaptive trial platforms, and integrating research networks are essential.
- Innovative funding models, including public-private partnerships and revenue reinvestment, are needed.
- A coordinated, sustainable ecosystem can transform ALS therapy development and serve as a model for other rare neurodegenerative diseases.
