Serum miRNAs miR-206, 143-3p and 374b-5p as potential biomarkers for amyotrophic lateral sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS) is a fatal, neurodegenerative condition characteris loss of motor neurones and progressive muscle wasting. There is no diagnostic test fo therefore robust biomarkers would not only be valuable for diagnosis, but also the classification of disease subtypes, monitoring responses to drugs and tracking diseas progression. As regulators of gene expression, microRNAs (miRNAs) are increasingly for diagnostic and prognostic purposes in various disease states with increasing explo in neurodegenerative disorders. We hypothesise that circulating blood based miRNAs serve as biomarkers and use miRNA profiling to determine miRNA signatures from th serum of sporadic (sALS) patients compared to healthy controls and patients with dise that mimic ALS. A number of differentially expressed miRNAs were identified in each patient comparisons. Validation in an additional patient cohort showed that miR-206 a miR-143-3p were increased and miR-374b-5p was decreased compared to controls. A continued change in miRNA expression persisted during disease progression indicatin potential use of these particular miRNAs as longitudinal biomarkers in ALS

Tauroursodeoxycholic acid in patients with amyotrophic lateral sclerosis: The TUDCA-ALS trial protocol

Background: Amyotrophic lateral sclerosis (ALS) is a chronic neurodegenerative rare disease that affects motor neurons in the brain, brainstem, and spinal cord, resulting in progressive weakness and atrophy of voluntary skeletal muscles. Although much has been achieved in understanding the disease pathogenesis, treatment options are limited, and in Europe, riluzole is the only approved drug. Recently, some other drugs showed minor effects. Methods: The TUDCA-ALS trial is a phase III, multicenter, randomized, double-blind, placebo-controlled, parallel-group clinical trial. The study aims to enroll 320 patients in 25 centers across seven countries in Europe. Enrolled patients are randomized to one of two treatment arms: TUDCA or identical placebo by oral route. The study measures disease progression during the treatment period and compares it to natural progression during a no-treatment run-in phase. Clinical data and specific biomarkers are measured during the trial. The study is coordinated by a consortium composed of leading European ALS centers. Conclusion: This trial is aimed to determine whether TUDCA has a disease-modifying activity in ALS. Demonstration of TUDCA efficacy, combined with the validation of new biomarkers, could advance ALS patient care