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Exp Neurol. 2014 Dec;262 Pt B:138-51. doi: 10.1016/j.expneurol.2014.07.001. Epub 2014 Jul 11.

Translating biological findings into new treatment strategies for amyotrophic lateral sclerosis (ALS).

Author information

  • 1KU Leuven, Laboratory of Neurobiology and Leuven Research Institute for Neuroscience and Disease (LIND), Leuven, Belgium; VIB, Vesalius Research Center, Leuven, Belgium.
  • 2KU Leuven, Laboratory of Neurobiology and Leuven Research Institute for Neuroscience and Disease (LIND), Leuven, Belgium; VIB, Vesalius Research Center, Leuven, Belgium; Neurology, University Hospitals Leuven, Leuven, Belgium.
  • 3KU Leuven, Laboratory of Neurobiology and Leuven Research Institute for Neuroscience and Disease (LIND), Leuven, Belgium; VIB, Vesalius Research Center, Leuven, Belgium. Electronic address: ludo.vandenbosch@vib-kuleuven.be.

Abstract

Amyotrophic lateral sclerosis (ALS) is characterized by the selective death of motor neurons in the motor cortex, brainstem and spinal cord. It is a neurodegenerative disorder with high genetic and phenotypic variability. In most patients, the cause of the disease is unknown. Until now, no treatment strategy has been discovered with the exception of riluzole which has a moderate effect on the disease process. While developing a new causal therapy targeting a specific disease-causing gene can have a huge effect on the disease process, only a limited number of ALS patients will benefit from such a therapy. Alternatively, pathogenic processes that are common in ALS patients with different etiology can also be targeted. The effect of such a modifying treatment will be smaller, but the target population will be larger as more ALS patients could benefit. In this review, we summarize the evidence for the involvement of different biological processes in the pathogenesis of ALS and will discuss how strategies influencing these processes can be translated into new therapeutic approaches. In order to further improve this translational step, there is an urgent need for a better understanding of the underlying mechanism(s), for new ALS animal models and for rigorous protocols to perform preclinical studies.

KEYWORDS:

Clinical trials; Motoneuron; Motor neuron disease; Neurodegeneration; Therapy

PMID:
25017368
DOI:
10.1016/j.expneurol.2014.07.001
[PubMed - indexed for MEDLINE]
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