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 language | English |
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Article number | 1601 |
Journal | Journal of Personalized Medicine |
Volume | 12 |
Issue number | 10 |
Early online date | 28 Sept 2022 |
DOIs | |
Publication status | Published online - 28 Sept 2022 |
Bibliographical note
Publisher Copyright:© 2022 by the authors.
Keywords
- riluzole
- preclinical models
- protein homeostasis inductors
- ALS trials platforms
- gene targeted strategies