Metabolic Alteration in Presymptomatic and Symptomatic ALS Study (MAPS ALS Study)
症状前和症状性 ALS 研究中的代谢改变(MAPS ALS 研究)
基本信息
- 批准号:10644489
- 负责人:
- 金额:$ 22.96万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2023
- 资助国家:美国
- 起止时间:2023-05-01 至 2028-04-30
- 项目状态:未结题
- 来源:
- 关键词:ALS patientsAdultAffectAmyotrophic Lateral SclerosisAreaBiological MarkersBiometryBloodBody CompositionBody WeightBody mass indexC9ORF72Cessation of lifeClassificationClinicClinicalClinical Course of DiseaseCohort StudiesControl GroupsDataData SetDeglutition DisordersDevelopmentDiagnosisDiagnosticDifferential DiagnosisDiseaseDisease ProgressionEarly DiagnosisEarly InterventionEarly identificationEarly treatmentEnergy IntakeEnergy MetabolismEpidemiologyFamilyFatty acid glycerol estersFoundationsGenetic Predisposition to DiseaseGenetic RiskGoalsHeightIndividualKnowledgeLearningLipidsLongitudinal cohort studyMachine LearningMeasuresMentored Patient-Oriented Research Career Development AwardMetabolicMetabolic MarkerMetabolismMotor Neuron DiseaseMotor NeuronsMuscle WeaknessNatural HistoryNerve DegenerationNeurodegenerative DisordersNeuronal DysfunctionNewly DiagnosedOxidative StressParticipantPathogenesisPathogenicityPatternPlasmaPopulationPositioning AttributePrimary Lateral SclerosisProbabilityProductionProspective, cohort studyPublic HealthReportingResearchResearch PersonnelResearch TrainingRespiratory FailureRestSamplingSigns and SymptomsStressSymptomsTechniquesUnited StatesUniversitiesValidationVariantWeightalgorithm trainingbiobankbiomarker developmentclinical applicationcohortcomparison controldiagnostic biomarkerdrug developmentearly detection biomarkerseffective therapyfamilial amyotrophic lateral sclerosisfatty acid oxidationindexinginsightlipid mediatorlipid metabolismlipidomelipidomicslongitudinal, prospective studymachine learning algorithmmetabolic profilemitochondrial dysfunctionmutation carriernovelnovel diagnosticsphenotypic biomarkerpredictive markerpredictive toolspreventskillssporadic amyotrophic lateral sclerosissupervised learningtherapeutic targettotal energy expenditure
项目摘要
Project Summary/Abstract
Amyotrophic lateral sclerosis (ALS) is a devastating disease with upper and lower motor neuron dysfunction
and degeneration leading to progressive weakness and death due to dysphagia and respiratory failure. There
is an urgent need to develop effective treatment to stop or reverse the progression. However, this has proven
to be challenging due to an incomplete understanding of the pathogenesis and delay in the diagnosis of ALS.
There are data supporting biomarkers of mitochondrial dysfunction, energy expenditure (EE) and body
weight/composition as early indices of incident clinical disease. Identification of these markers could facilitate
earlier intervention to prevent or delay disease progression as well as provide information on therapeutic
targets in individuals at genetic risk for ALS. I hypothesize that 1) the plasma lipid mediators (lipidome) can
accurately differentiate ALS, and primary lateral sclerosis (PLS) subjects from controls 2) body
weight/composition and EE differ between C9orf72+ ALS, presymptomatic C9orf72 mutation carriers
(C9orf72+ Pre-ALS) participants and controls 3) certain metabolic profiles will predict symptom onset in
C9orf72+ Pre-ALS participants. In this proposed study, I will 1) investigate the plasma lipidome profile of ALS
and PLS subjects to identify lipid mediators as diagnostic biomarkers in motor neuron disease, 2) evaluate
body weight/composition and EE in C9orf72+ ALS and Pre-ALS to measure changes in energy metabolism
in advance of and through the course of disease, and 3) follow a cohort of C9orf72+ Pre-ALS participants
annually to track the emergence of ALS symptoms and signs to determine if certain metabolic profiles predict
symptom onset in this genetically predisposed population. Successful completion of this project would provide
key data to 1) establish the lipidome profile as a diagnostic biomarker for ALS and PLS, and 2) provide the
foundation for a prospective cohort study of C9orf72+ Pre-ALS individuals to determine if the lipidome profile
and metabolic markers (body composition, EE) could serve as a premonitory marker for the phenotypic
conversion from asymptomatic to symptomatic ALS. This K23 award will provide the support needed to
complete the proposed research and to further develop my expertise in three major scientific areas, 1)
expertise in lipidome data production and analysis, 2) expertise in metabolism and EE, and 3) mastery of
advanced statistical techniques for clinical applications. I will leverage the research training I receive in the
K23 to lead a longitudinal cohort study of pre-ALS participants to determine the relationship between
metabolic changes and clinical manifestations of ALS. My overarching goal is to identify early diagnostic
biomarkers and therapeutic targets for the development of an effective treatment to prevent or delay the
progression of ALS.
项目总结/文摘
项目成果
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