Lithium carbonate in amyotrophic lateral sclerosis patients homozygous for the C-allele at SNP rs12608932 in UNC13A: protocol for a confirmatory, randomized, group-sequential, event-driven, double-blind, placebo-controlled trial

Journal article

Background and purpose: Amyotrophic lateral sclerosis (ALS) is among the most common motor neuron diseases in adults. Nevertheless, ALS remains fatal, despite decades of research and clinical trials, which has led to negative conclusions until recently in regard to four specific treatments. It is well known that we can learn from failures, and we consider that the time has come to present positive insight on this disease. Methods: We did a literature search using PubMed and Scopus for articles published in English from 1 January 2016, to 30 June 2022 dealing with “amyotrophic lateral sclerosis”, diagnosis, treatment, and biomarkers. Results: A comprehensive review of the literature on diagnosis, monitoring, and treatment of this condition showed convincing evidence that we are now able to diagnose earlier as well as to better monitor and treat ALS. Conclusions: Although ALS is often difficult to diagnose and remains incurable, there are many indications that an optimistic view of ALS management in the coming years is now realistic.

Authors: Willemse, SW; Roes, KCB; Van Damme, P; Hardiman, O; Ingre, C

• Publication status: Published
• Peer review status: Peer reviewed
• Publisher: BioMed Central
• Journal: Trials
• Volume: 23
• Issue: 1
• Pages: 978-978
• Article number: 978
• Publication date: 2022-12-05
• DOI: 10.1186/s13063-022-06906-5
• EISSN: 1745-6215
• ISSN: 1745-6215
• Language: English
• Keywords: Placebo-controlled study; Disease; Biology; Genotype; Amyotrophic lateral sclerosis; Randomized controlled trial; Oncology; Medicine; Clinical endpoint; Pathology; Interim analysis; Tolerability; Internal medicine; Adverse effect; Single-nucleotide polymorphism; Genetics; Placebo; Lithium carbonate; Double blind