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Review
. 2022 Sep 28;12(10):1601.
doi: 10.3390/jpm12101601.

Status of ALS Treatment, Insights into Therapeutic Challenges and Dilemmas

Mohammed Khamaysa  1 , Pierre-François Pradat  1   2   3
Affiliations
Review

Status of ALS Treatment, Insights into Therapeutic Challenges and Dilemmas

Mohammed Khamaysa et al. J Pers Med. .

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.

Keywords: ALS trials platforms; gene targeted strategies; preclinical models; protein homeostasis inductors; riluzole.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
ALS mutations are involved in RNA metabolism, protein degradation, autophagy, or axonal transport.
Figure 2
Figure 2
Summaries the utility of biomarkers in drug development in ALS spans from preclinical models, with translational biomarkers, to real-life studies.
See this image and copyright information in PMC

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