Repurposed Rheumatoid Arthritis Drug Baricitinib Shows Promise in ALS Treatment
核心洞察
The TRIALS program is repurposing FDA-approved drugs to expedite the introduction of therapies for ALS (搜索) patients, leveraging existing safety data.
Research indicates baricitinib, an FDA-approved drug for rheumatoid arthritis (搜索), may act on novel drug targets in neurons relevant to ALS (搜索).
A new tool is being developed to predict ALS (搜索) progression, utilizing cryptic exons as a proxy for TDP-43 pathology, a hallmark of ALS.
The Sean M. Healey & AMG Center for ALS (搜索) is actively exploring repurposed drugs to identify effective therapies for amyotrophic lateral sclerosis (搜索) (ALS). The Therapeutic Repurposing in ALS (TRIALS) program focuses on leveraging existing FDA-approved drugs to accelerate the availability of new treatments for ALS patients.
Baricitinib's Potential in ALS
Recent studies from the Albers Lab at Massachusetts General Hospital indicate that baricitinib, a drug approved for rheumatoid arthritis (搜索), may have potential in treating ALS (搜索). Mark Albers, MD, PhD, notes that comprehensive studies suggest baricitinib likely acts on other drug targets in neurons. The lab is now developing more potent, brain-penetrant molecules targeting these pathways.
Predicting ALS Progression
Researchers are developing a machine learning-based predictor of disease progression in ALS (搜索), incorporating cryptic exons as a proxy for TDP-43 pathology, which is present in 97% of ALS cases. This tool will be applied using gene expression profiles of ALS from the New York Genome Center and Target ALS (搜索). The Consortium of ALS Research Laboratories (CARL) has developed an informatics platform to harness this data. The TRIALS predictor will be made open source, allowing researchers worldwide to identify new drug candidates by studying gene expression changes induced by FDA-approved drugs in cell culture models of ALS.
Screening Chromatin Modifier Drugs
A recent screen of chromatin modifier drugs has been completed to identify new drug targets and pathways. These data will be integrated into the TRIALS predictor to enhance its ability to discover potential treatments. This approach involves analyzing gene lists comprehensively to identify new drug targets and pathways relevant to ALS (搜索).