title: Biomarkers for immune intervention trials in type 1 diabetes.
abstract: After many efforts to improve and standardize assays for detecting immune biomarkers in type 1 diabetes (T1D), methods to identify and monitor such correlates of insulitis are coming of age. The ultimate goal is to use these correlates to predict disease progression before onset and regression following therapeutic intervention, which would allow performing smaller and shorter pilot clinical trials with earlier endpoints than those offered by preserved β-cell function or improved glycemic control. Here, too, progress has been made. With the emerging insight that T1D represents a heterogeneous disease, the next challenge is to define patient subpopulations that qualify for personalized medicine or that should be enrolled for immune intervention, to maximize clinical benefit and decrease collateral damage by ineffective or even adverse immune therapeutics. This review discusses the current state of the art, setting the stage for future efforts to monitor disease heterogeneity, progression and therapeutic intervention in T1D.
authors.affiliation: Cochin Institute, INSERM U1016, DeAR Lab Avenir, Paris, France; Paris Descartes University, Sorbonne Paris Cité, Faculté de Médecine, Paris, France; Assistance Publique Hôpitaux de Paris, Hôtel Dieu, Service de Diabétologie, Paris, France. Electronic address: roberto.mallone@inserm.fr.
authors.forename: Roberto
authors.initials: R
authors.lastname: Mallone
authors.forename: Bart O
authors.initials: BO
authors.lastname: Roep
mesh_terms: D001323:Autoantibodies / Q000276:immunology; D015415:Biomarkers / Q000378:metabolism; D002096:C-Peptide / Q000378:metabolism; D002986:Clinical Trials as Topic; D003922:Diabetes Mellitus, Type 1 / Q000276:immunology* / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D018450:Disease Progression; D006801:Humans; D007004:Hypoglycemic Agents / Q000627:therapeutic use; D007167:Immunotherapy; D007328:Insulin / Q000378:metabolism; D016381:Islets of Langerhans Transplantation; D013601:T-Lymphocytes / Q000276:immunology* / Q000378:metabolism / Q000473:pathology
chemical_list: D001323:Autoantibodies; D015415:Biomarkers; D002096:C-Peptide; D007004:Hypoglycemic Agents; D007328:Insulin
keywords: Autoantibodies; C-peptide; Endpoint; T cells