シンポジウム4：世界で増加するALS治験：日本のドラッグロス回避と今後の戦略を考える
ALSの国際間での連携
2025 年 42 巻 3 号 p. 231-234
詳細
2025 年 42 巻 3 号 p. 231-234
On July 30, 2024, the Prime Minister's Office hosted the Drug Discovery Ecosystem Summit, where Prime Minister Kishida announced a five–year numerical target to eliminate uninitiated drug development by 2026. This initiative aims to revive Japan's pharmaceutical industry amidst growing dependency on overseas drugs, which has worsened the trade deficit. ALS is no exception to this “drug loss” issue.
ALS drug development has surged globally, with approximately 50 clinical trials ongoing. However, “pipeline pressure” due to excessive trials relative to patient numbers has become a concern in Western countries, prompting initiatives like NEALS, ENCALS, and TRICALS. Asia–Pacific established PACTALS in 2014, but regional trial infrastructure remains underdeveloped. While North America and Europe have embraced adaptive and platform trials, Japan has yet to participate. To address this, the Department of Neurology at Toho University launched an ALS clinic in 2017 and became Asia's first NEALS–certified site in 2020.
Japan's ALS registry, JaCALS, has contributed significantly to global research. However, only 2 of 11 ongoing Phase III trials listed in ClinicalTrials.gov include Japan. The lack of participation in adaptive trials exacerbates drug loss. Additionally, Japan's drug approval system and high trial costs deter venture firms.
To integrate with global trials, Japan is developing an English–language ALS clinical trial guideline. Efforts are also underway to harmonize ALSFRS–R standards and streamline trial site selection through ACT–PT. Strengthening international collaboration is essential for Japan to mitigate drug loss and enhance ALS drug development.
