Status of ALS Treatment, Insights into Therapeutic Challenges and Dilemmas

Mohammed Khamaysa, Pierre-François Pradat

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Abstract

Amyotrophic lateral sclerosis (ALS) is an extremely heterogeneous disease of motor neurons that eventually leads to death. Despite impressive advances in understanding the genetic, molecular, and pathological mechanisms of the disease, the only drug approved to date by both the FDA and EMA is riluzole, with a modest effect on survival. In this opinion view paper, we will discuss how to address some challenges for drug development in ALS at the conceptual, technological, and methodological levels. In addition, socioeconomic and ethical issues related to the legitimate need of patients to benefit quickly from new treatments will also be addressed. In conclusion, this brief review takes a more optimistic view, given the recent approval of two new drugs in some countries and the development of targeted gene therapies.
Original languageEnglish
Article number1601
JournalJournal of Personalized Medicine
Volume12
Issue number10
Early online date28 Sep 2022
DOIs
Publication statusPublished online - 28 Sep 2022

Keywords

  • riluzole
  • preclinical models
  • protein homeostasis inductors
  • ALS trials platforms
  • gene targeted strategies

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