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

Abstract

Amyotrophic lateral sclerosis (ALS) is a rare progressive motor neuron disease that, due to its high complexity, still lacks effective treatments. Development of a new drug is a highly costly and time-consuming process, and the repositioning of approved drugs can represent an efficient strategy to provide therapeutic opportunities. This is particularly true for rare diseases, which are characterised by small patient populations and therefore attract little commercial interest. Based on the overlap between the biological background of cancer and neurodegeneration, the repurposing of antineoplastic drugs for ALS has been suggested. The objective of this narrative review was to summarise the current experimental evidence on the use of approved anticancer drugs in ALS. Specifically, anticancer drugs belonging to different classes were found to act on mechanisms involved in the ALS pathogenesis, and some of them proved to exert beneficial effects in ALS models. However, additional studies are necessary to confirm the real therapeutic potential of anticancer drugs for repositioning in ALS treatment.

Advancements and challenges in amyotrophic lateral sclerosis

“…These include drugs previously approved for cancer treatments (Bosutinib and Mastinib), HIV treatments (Dolutegravir, Abacavir, and Lamivudine), and rheumatoid arthritis (Baricitinib) ( Greig and Deeks, 2015 ; Isfort et al, 2018 ; Urits et al, 2020 ; Ketabforoush et al, 2023 ). Additional agents being investigated preclinically include autophagy inducers (Rapamycin), hormone antagonists (Tamoxifen and Imatinib), Alkylating agents (Cisplatin and Carboplatin), and immunomodulating drugs (Thalidomide and Lenalidomide) ( Mandrioli et al, 2023 ; Potenza et al, 2024 ).…”