2024 年 41 巻 3 号 p. 310-313
Amyotrophic lateral sclerosis (ALS) has been synonymous with incurable neurological diseases. To date, there is no treatment that patients can fully benefit from, but therapies are being developed that are expected to control the progression of the disease. The two treatments listed as Clinical Questions (CQs) in the ALS Clinical Guidelines 2023 are riluzole and edaravone, both of which are conditionally recommended. Along with attention to common side effects, information about the limited efficacy of the drug should be provided before its use.
Significant progress has been made since the development of the ALS Clinical Practice Guideline 2023. In June 2022, it was announced that the Phase III methylcobalamin trial in early ALS patients showed efficacy. In September 2022, AMX0035 (sodium phenylbutyrate/taurursodiol) was approved by the US FDA for the treatment of ALS. In December 2022, edaravone oral suspension was approved, eliminating the burden of hospital visits associated with intravenous infusions and the difficulties of securing a peripheral vein, thus improving the limitations of its use. In April 2023, the US FDA approved tofersen, an antisense oligonucleotide (ASO) for ALS patients who are SOD1 mutation positive. Although the tofersen Phase 3 trial did not show a significant difference in the primary endpoint, the significant suppression of the plasma neurofilament light chain (NfL) biomarker was the basis for approval. A method has been used to detect pathological phenotypes in motor neurons derived from iPS cells of ALS patients and to search for drugs that can ameliorate these phenotypes. Ropinirole and bosutinib, discovered by this method, have been tested in clinical trials.
Much progress has been made in the development of treatments for ALS in just the last two years, and more is expected in the future.
