Possible treatment for a rare rare disease

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Possible treatment for a rare rare disease







A group of scientists from the Alberto Sols Institute for Biomedical Research, a joint center of the Spanish National Research Council (CSIC) and the Autonomous University of Madrid (UAM) (Spain) and in collaboration with the Oregon Health & Science University in Portland, have identified the thyroid hormone analogue "sobetirome" as a possible drug to treat Allan-Herndon-Dudley syndrome.



Allan-Herndon-Dudley syndrome is a rare disease caused by mutations in the MCT8 thyroid hormone transporter, which only affects males. Patients have a global developmental delay, profound intellectual disability, lack of language acquisition and severe neuromotor disorders, such as hypotonia or weak muscle tone, which evolves into spastic tetraplegia.



Patients with this disease also show alterations in the levels of thyroid hormones in blood, as they have high levels of thyroid hormone T3 that generates peripheral hyperthyroidism.



At the brain level, the lack of MCT8 in the blood-brain barrier (the barrier that separates circulating blood from the brain and regulates the passage of substances from one tissue to another), prevents thyroid hormones from being transported inside the brain, generating a cerebral hypothyroidism. Therefore, patients deficient in MCT8 present a paradoxical situation of peripheral hyperthyroidism and cerebral hypothyroidism.







(Image: CSIC-UAM)



At present unfortunately there is no effective treatment to alleviate the symptoms of the disease. And the design of a therapeutic strategy is complex, since an effective treatment should be able to correct peripheral hyperthyroidism and improve the neurological alterations derived from cerebral hypothyroidism.



The sobetirome is a synthetic drug that has a chemical structure distinct from the thyroid hormone T3, but that is able to bind to the thyroid hormone receptors and trigger the same type of responses in the cells.



"In our study, we treated Mct8 deficient mice with sobetirome, to evaluate if this analogue is capable of improving peripheral hyperthyroidism and of accessing the brain in conditions of Mct8 deficiency to generate similar responses to thyroid hormone T3," the authors explain.



Their results, published in Thyroid magazine, revealed that treatment with sobetirome in mice deficient in Mct8 drastically decreases T3 levels in the blood. And what is even more important, that the sobetirome is able to access the brain and regulate the expression of some genes that are normally regulated by T3.



"Therefore, our studies indicate that the sobetirome thyroid hormone analogue has the potential to improve peripheral hyperthyroidism and the neurological abnormalities characteristic of Allan-Herndon-Dudley syndrome," the authors conclude. (Source: UAM)

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