Biomarker Identification by Conjoint Analysis of Metabolites and Gut Microbiome in ALS Patients and Animal Models to Monitor Disease Stages of ALS
Abstract
This grant proposes to develop a disease modifying treatment of Amyotrophic Lateral Sclerosis (ALS), targeting mitochondrial dysfunction as well as identify biomarkers to be used to screen potential ALS patients, differentiate diagnosed patients into subgroups and assess therapeutic efficacy. By combining the 2N Pharma (2N) teams expertise in neurodegenerative disease research and the expertise of our co-applicant, Dr. Barmada in the research area of ALS/FTD with our innovative conceptualization, 2N can bring a novel treatment and a novel biomarker concept into a clinical market. For over a decade, Dr. Nieland and collaborators have researched the connection between impaired energy metabolism and CNS diseases. Carnitine palmitoyl transferase 1 (CPT1) is a rate-limiting enzyme located in the outer mitochondrial membrane and is responsible for the conversion of long-chain acyl-CoAs into long-chain acyl-carnitines in the mitochondria. Based on findings reporting dysregulated metabolism in ALS patients and animal models of ALS, we hypothesize that inhibiting lipid metabolism by targeting CPT1 can halt progression or prevent induction of ALS.
Document Details
- Document Type
- Technical Report
- Publication Date
- May 01, 2023
- Accession Number
- AD1204420
Entities
People
- John Nieland