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Metabolic Changes in Lymphangioleiomyomatosis Patients Treated with Sirolimus and Hydroxychloroquine.

08:00 EDT 9th July 2019 | BioPortfolio

Summary of "Metabolic Changes in Lymphangioleiomyomatosis Patients Treated with Sirolimus and Hydroxychloroquine."

Lymphangioleiomyomatosis (LAM), a destructive lung disease that primarily affects women, is caused by loss-of-function mutations in TSC1 or TSC2, leading to hyperactivation of mechanistic/mammalian Target of Rapamycin 1 (mTORC1). Rapamycin (sirolimus) treatment suppresses mTORC1, but also induces autophagy, which promotes the survival of TSC2-deficient cells. Based on the hypothesis that simultaneous inhibition of mTORC1 and autophagy would limit the availability of critical nutrients and inhibit LAM cells, we conducted a Phase I clinical trial of sirolimus and hydroxychloroquine for LAM. Here, we report the analyses of plasma metabolomic profiles from the clinical trial.

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Name: Chest
ISSN: 1931-3543
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Medical and Biotech [MESH] Definitions

A chemotherapeutic agent that acts against erythrocytic forms of malarial parasites. Hydroxychloroquine appears to concentrate in food vacuoles of affected protozoa. It inhibits plasmodial heme polymerase. (From Gilman et al., Goodman and Gilman's The Pharmacological Basis of Therapeutics, 9th ed, p970)

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A derivative of sirolimus and an inhibitor of TOR SERINE-THREONINE KINASES. It is used to prevent GRAFT REJECTION in heart and kidney transplant patients by blocking cell proliferation signals. It is also an ANTINEOPLASTIC AGENT.

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