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Evaluation of anti-insulin receptor antibodies as potential novel therapies for human insulin receptoropathy using cell culture models.

Title: Evaluation of anti-insulin receptor antibodies as potential novel therapies for human insulin receptoropathy using cell culture models.
Authors: Brierley, Gemma V; Siddle, Kenneth; Semple, Robert K
Publisher Information: Springer Nature; //doi.org/10.1007/s00125-018-4606-2
Publication Year: 2018
Collection: Apollo - University of Cambridge Repository
Subject Terms: Diabetes; Donohue syndrome; Insulin receptor; Insulin resistance; Insulin signalling; Monoclonal antibodies; Rabson–Mendenhall syndrome; 3T3-L1 Cells; Adipocytes; Animals; Antibodies; Antigens; CD; CHO Cells; Cricetulus; Glucose; Humans; Hypoglycemic Agents; Insulin; Mice; Mutation; Phosphorylation; Receptor; Signal Transduction
Description: AIMS/HYPOTHESIS: Bi-allelic loss-of-function mutations in the INSR gene (encoding the insulin receptor [INSR]) commonly cause extreme insulin resistance and early mortality. Therapeutic options are limited, but anti-INSR antibodies have been shown to activate two mutant receptors, S323L and F382V. This study evaluates four well-characterised murine anti-INSR monoclonal antibodies recognising distinct epitopes (83-7, 83-14, 18-44, 18-146) as surrogate agonists for potential targeted treatment of severe insulin resistance arising from insulin receptoropathies. METHODS: Ten naturally occurring mutant human INSRs with defects affecting different aspects of receptor function were modelled and assessed for response to insulin and anti-INSR antibodies. A novel 3T3-L1 adipocyte model of insulin receptoropathy was generated, permitting conditional knockdown of endogenous mouse Insr by lentiviral expression of species-specific short hairpin (sh)RNAs with simultaneous expression of human mutant INSR transgenes. RESULTS: All expressed mutant INSR bound to all antibodies tested. Eight mutants showed antibody-induced autophosphorylation, while co-treatment with antibody and insulin increased maximal phosphorylation compared with insulin alone. After knockdown of mouse Insr and expression of mutant INSR in 3T3-L1 adipocytes, two antibodies (83-7 and 83-14) activated signalling via protein kinase B (Akt) preferentially over signalling via extracellular signal-regulated kinase 1/2 (ERK1/2) for seven mutants. These antibodies stimulated glucose uptake via P193L, S323L, F382V and D707A mutant INSRs, with antibody response greater than insulin response for D707A. CONCLUSIONS/INTERPRETATION: Anti-INSR monoclonal antibodies can activate selected naturally occurring mutant human insulin receptors, bringing closer the prospect of novel therapy for severe insulin resistance caused by recessive mutations.
Document Type: article in journal/newspaper
File Description: Print-Electronic; application/pdf
Language: English
Relation: https://www.repository.cam.ac.uk/handle/1810/276693
DOI: 10.17863/CAM.23988
Availability: https://www.repository.cam.ac.uk/handle/1810/276693; https://doi.org/10.17863/CAM.23988
Rights: Attribution 4.0 International ; https://creativecommons.org/licenses/by/4.0/
Accession Number: edsbas.CB687F41
Database: BASE