Recent Advances in ALS Research: Perspectives for Personalized Clinical Application

Chen Benkler, Daniel Offen, Eldad Melamed, Tamar Amit, Silvia Mandel, Moussa B.H. Youdim, Orly Weinreb*

*Corresponding author for this work

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review


Treatment of amyotrophic lateral sclerosis (ALS) has been fueled, in part, by frustration over the shortcomings of the symptomatic drugs available, since these do not impede the progression of this disease. Currently, over 150 different potential therapeutic agents or strategies have been tested in preclinical models of ALS. Unfortunately, therapeutic modifiers of murine ALS have failed to be successfully translated into strategies for patients, probably because of differences in pharmacokinetics of the therapeutic agents, route of delivery, inefficiency of the agents to affect the distinct pathologies of the disease or inherent limitations of the available animal models. Given the multiplicity of the pathological mechanisms implicated in ALS, new therapies should consider the simultaneous manipulation of multiple targets. Additionally, a better management of ALS therapy should include understanding the interactions between potential risk factors, biomarkers and heterogeneous clinical features of the patients, aiming to manage their adverse events or personalize the safety profile of these agents. This review will discuss novel pharmacological approaches concerning adjusted therapy for ALS patients: iron-binding brain permeable multimodal compounds, genetic manipulation and cell-based treatment.

Original languageEnglish
Title of host publicationNew Strategies to Advance Pre/Diabetes Care
Subtitle of host publicationIntegrative Approach by PPPM
PublisherKluwer Academic Publishers
Number of pages40
ISBN (Print)9789400759701
StatePublished - 2013

Publication series

NameAdvances in Predictive, Preventive and Personalised Medicine
ISSN (Print)2211-3495
ISSN (Electronic)2211-3509


  • Amyotrophic lateral sclerosis
  • Genetic manipulation
  • Iron chelating-multifunctional drugs
  • Neurotrophic factors
  • Personalized medicine
  • Stem cells


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