Status of ALS Treatment, Insights into Therapeutic Challenges and Dilemmas

Overview

Journal J Pers Med

Date 2022 Oct 27

PMID 36294741

Authors

Mohammed Khamaysa

Pierre-Francois Pradat

Affiliations

Soon will be listed here.

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.

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