Treatment Approaches for Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that impairs motor neuron function, causing progressive muscle weakness and paralysis. ALS is extremely rare, affecting only around 30,000 people in the United States. However, it has a very low survival rate; only 10% of patients survive 10 years after disease onset. While the cause is typically unknown, 5-10% of ALS cases are caused by genetic mutations in the SOD1 and C9orf72 genes. Currently, several FDA-approved therapies for ALS exist. These drugs aim to partially correct the SOD1 mutation, or block cellular and neurological pathways related to motor neuron damage. In clinical studies to date, these drugs either unsuccessfully treat ALS, or are only able to prolong a patient's lifespan up to several months. Scientists are exploring novel therapies that target the root cause of ALS, whether it be damaged neurons, genetic factors, or muscle dysfunction. These therapies include gene therapy, monoclonal antibody therapy, and stem cell therapy. While further research is needed, current data show significant promise, as these treatments directly target the underlying mechanisms of ALS, rather than focusing on select symptoms like conventional approaches, ultimately making new approaches to ALS therapy more sustainable for the future. This paper will discuss and evaluate current FDA-approved therapies for ALS and explore the potential efficacy of novel treatments.