Efficacy

Efficacy of Edaravone in ALS

Edaravone, marketed by Daiichi Sankyo, is a medication that has shown efficacy in the treatment of Amyotrophic Lateral Sclerosis (ALS), a progressive neurodegenerative disease. The drug was approved by the U.S. Food and Drug Administration (FDA) in May 2017 for use in patients with ALS based on clinical trials that demonstrated its ability to slow the decline in physical function. Specifically, a six-month clinical trial in Japan showed that ALS patients receiving edaravone experienced less decline in physical function compared to those receiving a placebo, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disability in ALS patients.

The exact mechanism by which edaravone exerts its effects in ALS is not fully understood. However, it is believed to be an antioxidant that helps relieve the effects of oxidative stress, which is thought to play a role in the death of motor neurons in ALS patients. By mitigating this stress, edaravone may help to preserve motor neuron function and slow the progression of the disease. It is important to note that while edaravone has been shown to slow the decline in physical function, it is not a cure for ALS and does not reverse damage already done by the disease.

Efficacy of Nuedexta in ALS

Nuedexta, a combination of dextromethorphan HBr and quinidine sulfate, is primarily approved for the treatment of pseudobulbar affect (PBA), a condition characterized by involuntary and uncontrollable laughing or crying that can occur in various neurological disorders, including ALS. While Nuedexta is not specifically approved for the treatment of ALS itself, it has been shown to be effective in controlling the symptoms of PBA in ALS patients. The efficacy of Nuedexta in treating PBA in ALS was demonstrated in clinical trials where patients with ALS showed a significant reduction in the frequency and severity of PBA episodes compared to placebo.

It is important to clarify that while Nuedexta has demonstrated efficacy in managing PBA symptoms associated with ALS, there is limited evidence regarding its direct impact on the progression of ALS or improvement in motor function. The primary role of Nuedexta in the context of ALS is to improve the quality of life by addressing the emotional lability that often accompanies the disease. Healthcare providers may consider the use of Nuedexta in ALS patients who are experiencing PBA, but its use for the neurodegenerative aspects of ALS remains off-label and requires further investigation.