Abstract
Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disease that targets motor neurons (MNs) in the brain and spinal cord. It leads to gradual loss of motor signals to muscles leading to atrophy and weakness. Most patients do not survive for more than 3–5 years after disease onset. Current ALS treatments provide only a small delay of disease progression. Therefore, it is of utmost importance to explore new therapeutic approaches. One of the major hindrances in achieving this goal is poor understanding of causes of the disease. ALS has complex pathophysiological mechanisms in its genetic and sporadic forms. Protein aggregates are a common hallmark of ALS regardless of cause making protein pathways attractive therapeutic targets in ALS. Here, we provide an overview of compounds in different stages of pharmacological development and their protein pathway targets.
Highlights
Amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease is the most common motor neuron (MN) disease
Amyloid deposits from different proteins such as TDP43, Chromosome 9 open reading frame 72 (C9ORF72) dipeptide repeats (DPRs), phosphorylated high molecular weight neurofilament protein, rho guanine nucleotide exchange factor (RGNEF), and fused in sarcoma (FUS) have been detected in ALS MNs (Blokhuis et al, 2013)
We focus on proteins which form MN aggregates implicated in ALS pathology
Summary
Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disease that targets motor neurons (MNs) in the brain and spinal cord. It leads to gradual loss of motor signals to muscles leading to atrophy and weakness. Current ALS treatments provide only a small delay of disease progression. It is of utmost importance to explore new therapeutic approaches. Protein aggregates are a common hallmark of ALS regardless of cause making protein pathways attractive therapeutic targets in ALS. We provide an overview of compounds in different stages of pharmacological development and their protein pathway targets.
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