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Updated: Aug 16, 2026
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
Published on: July 29, 2007
Therapeutic developments in amyotrophic lateral sclerosis
1Baylor College of Medicine, Houston Veterans Affairs Medical Center, 6550 Fannin, Suite 1801, Houston, Texas 77030, USA. elai@bcm.tmc.edu
Abstract:
There is currently no effective treatment for amyotrophic lateral sclerosis (ALS), a devastating disorder of the human nervous system that, due to motoneurone degeneration, causes progressive loss of muscle function and death. The relentless progression of ALS and the uniformly poor prognosis have been unhindered by a variety of therapeutic agents tested in previous clinical studies. Recently, two drugs, namely riluzole and recombinant human insulin-like growth factor-I (IGF-1), have been reported to benefit patients with ALS by improving survival or slowing disease progression. Several other drugs, such as gabapentin and various neurotrophic factors, are being investigated in on-going clinical trials. Therapeutic developments in ALS have been hampered by the fact that the precise cause of the disease remains unknown. In addition, there are considerable variations in disease related characteristics among patients, rendering accurate measurements of disease progression difficult. Advances in theories of pathogenesis, such as genetic factors, glutamate excitotoxicity, oxidative stress, autoimmune mechanism and cytoskeletal abnormality will help guide the development of future therapies. Newer approaches to therapy may include suitable glutamate antagonists, small molecules that augment neurotrophic factor function, and anti-oxidants. Combination therapy of effective agents should be considered.
Insights
Currently, no cure exists for amyotrophic lateral sclerosis (ALS). Riluzole and insulin-like growth factor-I (IGF-1) show promise in slowing disease progression and improving survival for ALS patients.
Area of Science:
- Neuroscience
- Neurology
- Pharmacology
Background:
- Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by motoneurone degeneration.
- Current treatments for ALS are largely ineffective, with a uniformly poor prognosis.
- Therapeutic development is hindered by unknown etiology and patient variability.
Purpose of the Study:
- To review current therapeutic strategies for amyotrophic lateral sclerosis (ALS).
- To discuss emerging drug candidates and novel therapeutic approaches.
- To highlight the importance of understanding ALS pathogenesis for future treatment development.
Main Methods:
- Literature review of clinical studies and ongoing trials for ALS treatments.
- Analysis of recent findings on riluzole and recombinant human insulin-like growth factor-I (IGF-1).
- Discussion of potential therapeutic targets based on advancing theories of ALS pathogenesis.
Main Results:
- Riluzole and IGF-1 have demonstrated benefits in improving survival or slowing disease progression in ALS patients.
- Gabapentin and neurotrophic factors are under investigation in clinical trials.
- Advances in understanding pathogenesis (genetics, excitotoxicity, oxidative stress) are guiding new therapies.
Conclusions:
- Future ALS therapies may include glutamate antagonists, neurotrophic factor enhancers, and antioxidants.
- Combination therapy approaches should be considered for enhanced efficacy.
- Further research into ALS pathogenesis is crucial for developing effective treatments.
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