[A strategy to develop effective ALS therapy].

Area of Science:

• Neurology and Neuroscience
• Genetics and Molecular Biology
• Clinical Trial Research

Context:

• The early 1990s marked key discoveries in ALS, including the SOD1 gene mutation and the approval of riluzole, the first ALS treatment.
• Focus shifted towards improving patient quality of life through standardized care, multidisciplinary systems, and enhanced nutritional and respiratory support.
• Significant progress in basic science and translational research has led to numerous ALS drug candidates, though none have succeeded beyond riluzole.

Purpose:

• To review the historical progression of Amyotrophic Lateral Sclerosis (ALS) treatment and scientific understanding.
• To highlight advancements in patient care, including multidisciplinary approaches and specialized respiratory support.
• To critically assess the state of ALS drug discovery and propose strategies for future development.

Summary:

• Key 1990s discoveries include the SOD1 mutation in familial ALS and the introduction of riluzole. Patient care evolved with a focus on quality of life, multidisciplinary teams, and improved supportive measures.
• Despite over 25 drugs tested in clinical trials, riluzole remains the only approved medication, indicating a critical need for revised drug development strategies.
• The abstract emphasizes the necessity of a cohesive national effort, involving funding, regulatory oversight, and "outside the box" thinking from diverse experts to accelerate ALS drug discovery.

Impact:

• Improved patient care through multidisciplinary teams and enhanced supportive treatments has positively impacted quality of life for individuals with ALS.
• The slow adoption of care guidelines by caregivers underscores the need for better dissemination and integration of best practices.
• The current landscape of ALS research necessitates a paradigm shift in drug development, fostering collaboration and innovation to find effective treatments.