Literature DB >> 26701289

Effect of Triiodothyroacetic Acid Treatment in Mct8 Deficiency: A Word of Caution.

Soledad Bárez-López1,2, Maria Jesus Obregon1, Raquel Martínez-de-Mena1, Juan Bernal1,2, Ana Guadaño-Ferraz1,2, Beatriz Morte1,2.   

Abstract

BACKGROUND: Monocarboxylate transporter 8 (MCT8) is a thyroid hormone-specific cell membrane transporter. Mutations in the MCT8 gene lead to profound psychomotor retardation and abnormal thyroid hormone serum levels with low thyroxine (T4) and high triiodothyronine (T3). Currently, therapeutic options for patients are limited. Triiodothyroacetic acid (TRIAC) has potential therapeutic value. The aim of this study was to evaluate the effects and efficacy of therapeutic doses of TRIAC on Mct8-deficient mice (Mct8KO).
METHODS: Wild-type (Wt) and Mct8KO mice were treated with 30 ng TRIAC/g of body weight/day, given in drinking water, from postnatal day 21 to 30. TRIAC, T4 and T3 levels in plasma, as well as T3 and TRIAC content in the cerebral cortex and striatum were measured by specific radioimmunoassays. The activities of deiodinases 1 and 2 were measured in liver and cortex. The effect of TRIAC treatment in the expression of T3-dependent genes was measured in the heart, cerebral cortex, and striatum.
RESULTS: Plasma TRIAC concentration were the same in Wt and Mct8KO animals after treatment. TRIAC treatment greatly decreased plasma T4 in Wt and Mct8KO mice, and reduced T3 to normal levels in the Mct8KO mice. Deiodinase 1 activity and gene expression in the liver increased, while it did not have any effect on the expression of Serca2a in the heart. TRIAC treatment did not induce the expression of T3-dependent genes in the cerebral cortex or striatum, but further decreased expression of Flywch2 in the cortex and Aldh1a1 and Flywch2 in the striatum. Direct measurements of TRIAC and T3 content in the cortex and striatum revealed a decrease in T3 after treatment with no significant increase in the level of endogenous TRIAC.
CONCLUSIONS: Therapeutic doses of TRIAC in Mct8KO mice restored plasma T3 levels but severely decreased T4 levels. TRIAC has a direct effect on deiodinase 1 in the liver and does not have an effect on gene expression in the heart. The increase in the plasma TRIAC levels after treatment is not sufficient to increase TRIAC levels in the brain and to promote the expression of T3-dependent genes in brain cells. Instead, it leads to a state of brain hypothyroidism with reduced T3 content.

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Year:  2016        PMID: 26701289     DOI: 10.1089/thy.2015.0388

Source DB:  PubMed          Journal:  Thyroid        ISSN: 1050-7256            Impact factor:   6.568


  8 in total

1.  Sobetirome and its Amide Prodrug Sob-AM2 Exert Thyromimetic Actions in Mct8-Deficient Brain.

Authors:  Soledad Bárez-López; Meredith D Hartley; Carmen Grijota-Martínez; Thomas S Scanlan; Ana Guadaño-Ferraz
Journal:  Thyroid       Date:  2018-06-29       Impact factor: 6.568

Review 2.  Oligodendroglial Lineage Cells in Thyroid Hormone-Deprived Conditions.

Authors:  Min Joung Kim; Steven Petratos
Journal:  Stem Cells Int       Date:  2019-04-30       Impact factor: 5.443

3.  Intracerebroventricular administration of the thyroid hormone analog TRIAC increases its brain content in the absence of MCT8.

Authors:  Soledad Bárez-López; Carmen Grijota-Martínez; Xiao-Hui Liao; Samuel Refetoff; Ana Guadaño-Ferraz
Journal:  PLoS One       Date:  2019-12-06       Impact factor: 3.240

Review 4.  Thyroid Hormone Hyposensitivity: From Genotype to Phenotype and Back.

Authors:  Giuditta Rurale; Emery Di Cicco; Monica Dentice; Domenico Salvatore; Luca Persani; Federica Marelli; Cristina Luongo
Journal:  Front Endocrinol (Lausanne)       Date:  2020-01-24       Impact factor: 5.555

Review 5.  Thyroid hormone-dependent oligodendroglial cell lineage genomic and non-genomic signaling through integrin receptors.

Authors:  Rahimeh Emamnejad; Mary Dass; Michael Mahlis; Salome Bozkurt; Sining Ye; Maurice Pagnin; Paschalis Theotokis; Nikolaos Grigoriadis; Steven Petratos
Journal:  Front Pharmacol       Date:  2022-09-05       Impact factor: 5.988

6.  Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial.

Authors:  Stefan Groeneweg; Robin P Peeters; Carla Moran; Athanasia Stoupa; Françoise Auriol; Davide Tonduti; Alice Dica; Laura Paone; Klara Rozenkova; Jana Malikova; Adri van der Walt; Irenaeus F M de Coo; Anne McGowan; Greta Lyons; Femke K Aarsen; Diana Barca; Ingrid M van Beynum; Marieke M van der Knoop; Jurgen Jansen; Martien Manshande; Roelineke J Lunsing; Stan Nowak; Corstiaan A den Uil; M Carola Zillikens; Frank E Visser; Paul Vrijmoeth; Marie Claire Y de Wit; Nicole I Wolf; Angelique Zandstra; Gautam Ambegaonkar; Yogen Singh; Yolanda B de Rijke; Marco Medici; Enrico S Bertini; Sylvia Depoorter; Jan Lebl; Marco Cappa; Linda De Meirleir; Heiko Krude; Dana Craiu; Federica Zibordi; Isabelle Oliver Petit; Michel Polak; Krishna Chatterjee; Theo J Visser; W Edward Visser
Journal:  Lancet Diabetes Endocrinol       Date:  2019-07-31       Impact factor: 32.069

7.  The Protein Translocation Defect of MCT8L291R Is Rescued by Sodium Phenylbutyrate.

Authors:  Doreen Braun; Ulrich Schweizer
Journal:  Eur Thyroid J       Date:  2020-07-08

Review 8.  MCT8 Deficiency: The Road to Therapies for a Rare Disease.

Authors:  Carmen Grijota-Martínez; Soledad Bárez-López; David Gómez-Andrés; Ana Guadaño-Ferraz
Journal:  Front Neurosci       Date:  2020-04-28       Impact factor: 4.677

  8 in total

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