Therapy in Amyotrophic Lateral Sclerosis (ALS)

an unexpected evolving scenario

Research output: Contribution to journalArticle

Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease resulting in increasing disability, being uniformly fatal. Since its approval in the 1990s, riluzole remained for long time the unique treatment, offering modest survival benefit. Most recently a second drug has been approved by the US Food and Drug Administration for treatment of ALS: edaravone. Significant advances have been made in the symptomatic management of the disease but more effective drug therapy targeting disease progression is still dreadfully needed, the success appearing almost a miracle. Recent discoveries related to genetics indicate divergent mechanisms of disease encouraging precision medicine leading to molecularly tailored interventions. The search for effective therapy still faces important challenges in the areas of both basic science and animal research, adequate translation of results into human clinical trials, inherent bias in human studies, and issues related to delays in clinical diagnosis. It is interesting to point out that ALS research may speed up drug development not only for this disease, but also for other more prevalent neurodegenerative diseases: the reverse is also conceivable.

Original languageEnglish
Pages (from-to)118-130
Number of pages13
JournalArchives Italiennes de Biologie
Volume155
Issue number4
Publication statusPublished - Dec 1 2017

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Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Riluzole
Precision Medicine
United States Food and Drug Administration
Drug Delivery Systems
Disease Management
Pharmaceutical Preparations
Disease Progression
Therapeutics
Clinical Trials
Drug Therapy
Survival
Research

Keywords

  • Journal Article

Cite this

Therapy in Amyotrophic Lateral Sclerosis (ALS) : an unexpected evolving scenario. / Silani, Vincenzo.

In: Archives Italiennes de Biologie, Vol. 155, No. 4, 01.12.2017, p. 118-130.

Research output: Contribution to journalArticle

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