Can Some Anticancer Drugs Be Repurposed to Treat Amyotrophic Lateral Sclerosis? A Brief Narrative Review

Area of Science:

• Neuroscience
• Oncology
• Pharmacology

Background:

• Amyotrophic lateral sclerosis (ALS) is a rare, progressive motor neuron disease with limited effective treatments.
• Drug development is costly and time-consuming, making drug repositioning an efficient strategy, especially for rare diseases with small patient populations.
• Biological similarities between cancer and neurodegeneration suggest repurposing antineoplastic drugs for ALS treatment.

Purpose of the Study:

• To review existing experimental evidence on the use of approved anticancer drugs for ALS treatment.
• To explore the potential of antineoplastic drugs as therapeutic opportunities for ALS.

Main Methods:

• This narrative review systematically examined studies investigating anticancer drugs in the context of ALS.
• Evidence was gathered on various classes of anticancer drugs and their mechanisms of action relevant to ALS pathogenesis.

Main Results:

• Approved anticancer drugs from different classes target mechanisms implicated in ALS pathogenesis.
• Several antineoplastic agents have demonstrated positive effects in preclinical ALS models.
• The review highlights the potential of these drugs for repositioning.

Conclusions:

• Anticancer drugs show potential for repurposing in ALS treatment due to their action on shared biological pathways.
• Further investigation is required to validate the therapeutic efficacy of these repositioned drugs for ALS patients.
• Drug repurposing offers a viable strategy to accelerate therapeutic development for rare diseases like ALS.