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Diagnosis, Pathogenesis and Therapeutic Targets in Amyotrophic Lateral Sclerosis

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Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease of the motor system. The diagnosis is clinical, but additional investigations such as electromyography, transcranial magnetic stimulation and neuroimaging have demonstrated their usefulness in supporting diagnosis. Exhaustive research for the identification of molecular markers in the cerebrospinal fluid and plasma of ALS patients have been made; however, at present, there are no validated biomarkers for the disease.

Between 5 to 10% of the ALS cases have a positive familial history, up to now eleven genes have been identified as associated with the disease. The most studied gene encodes for cupper, zinc superoxide dismutase enzyme. The identified abnormal genes potentially allow the generation of experimental cell and animal models to study the mechanisms of the disease and to test potential therapeutic compounds.

The pathological characteristics of ALS include protein aggregation, proteasome inhibition, impaired axonal transport, mitochondria damage and apoptosis, oxidative stress, glutamate induced excitotoxicity, neuroinflammation and transcriptional dysfunction. Many compounds targeted to one or more of these mechanisms have been tested in multiple clinical trials. Nonetheless, nowadays only one drug, riluzole, has demonstrated a positive effect in the disease progression, but a number of recent compounds are promising in ALS therapy.

Keywords: AMPA; Amyotrophic lateral sclerosis; Apoptosis; CSF; Caenorhabditis elegans; Ceftriaxone; Celecoxib; Drosophila melanogaster; ELISA; Escherichia coli; Exosomes; FIG4; Magnetic resonance imaging; Memantine; Minocycline; NOGO A; Neuroinflammation; Parkinson's disease; SOD1; Saccharomyces cerevisiae; TARDBP; Transcranial magnetic stimulation; Transcriptional Dysfunction; UMN lesion; Verapamil; Xaliproden; axonal transport dysfunction; biomarkers; brefeldin-A-sensitive pathway; clinical trials; dextrometh-orphan; diagnosis; electromyography; experimental models; frontotemporal dementia; gabapentin; lamotrigine; liposarcoma; lower motor neuron; motor neurons atrophy; motor unit number estimation; pathogenesis; rHCNTF; riluzole; therapeutic compounds; tractography; upper motor neuron

Document Type: Research Article


Publication date: 2010-12-01

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  • CNS & Neurological Disorders - Drug Targets aims to cover all the latest and outstanding developments on the medicinal chemistry, pharmacology, molecular biology, genomics and biochemistry of contemporary molecular targets involved in neurological and central nervous system (CNS) disorders e.g. disease specific proteins, receptors, enzymes, genes. Each issue of the journal will contain a series of timely in-depth reviews written by leaders in the field covering a range of current topics on drug targets involved in neurological and CNS disorders. As the discovery, identification, characterization and validation of novel human drug targets for neurological and CNS drug discovery continues to grow; this journal will be essential reading for all pharmaceutical scientists involved in drug discovery and development.
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