Therapeutic frontiers in ALS: iPSC-based drug discovery, cell therapy, and gene therapy—Advances through 2026
Satoru Morimoto, Chris Kato, Shinichi Takahashi, Hideyuki Okano · 2026
Read Therapeutic frontiers in ALS: iPSC-based drug discovery, cell therapy, and gene therapy—Advances through 2026 by Satoru Morimoto, Chris Kato, Shinichi Takahashi, Hideyuki Okano (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/PMC13292465.txt.
Description
Satoru Morimoto, Chris Kato, Shinichi Takahashi, and Hideyuki Okano review a 2026 treatment frontier for ALS built from three merging strategies. Patient stem cells now drive drug discovery, cell transplants aim to calm brain inflammation, and gene therapies silence toxic genes. The review weighs evidence strength and the field's progress through 2026, with attention to Japanese groups' leading roles.
The FDA's accelerated approval of tofersen in April 2023 was the first treatment aimed at a genetic cause of ALS. Stem-cell-derived candidates ropinirole and bosutinib finished early-phase trials. Regulatory T cell therapies remain promising but unproven at scale. RNA interference and AAV microRNA drugs entered first human trials in 2024 and 2025. A newer thread targets STMN2, downstream of TDP-43 failure, which could reach the roughly 90 percent of cases that are sporadic.
Catalog record
| CREATOR | Satoru Morimoto, Chris Kato, Shinichi Takahashi, Hideyuki Okano |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 44041 |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |