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    Home > Medical News > Latest Medical News > ASH . . . Rare immune disease precision anti-cancer therapy 2 years of data positive

    ASH . . . Rare immune disease precision anti-cancer therapy 2 years of data positive

    • Last Update: 2020-05-31
    • Source: Internet
    • Author: User
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    SM comes in several forms, including inert SM, invasive SM, and late SMSM is a cloning blood disease characterized by the accumulation of fat cellsFat cells are immune cells that produce histamines and other media that mediate inflammation and allergic reactionsIn SM patients, high levels of these media released by hyperblastated cells can lead to varying degrees of symptoms, including pain, nausea, rash, fever, fatigue, etcIn severe cases, multiple organ systems in SM patients experience debilitating symptoms that can be life-threateningThe D816V mutation of the KIT protein is the main driver of almost all SM diseasesCurrently, there are no approved targets for this mutation therapy on the marketAvapritinib is a type 1 inhibitor that targets kinase-activated conformations and inhibits a variety of protein kinases that carry mutations in the KIT and PDGFRA genesBlueprint Medicines was originally developed to treat advanced gastrointestinal mesothelioma (GIST) and advanced SMIn June 2017, avapritinib was recognized by the FDA As a Breakthrough Therapy for the treatment of non-reprecision or metastatic GIST patients with PDGFR alpha D842V mutationsPreviously, the FDA also qualified avapritinib orphan drugs for the treatment of GIST and hyperblastomaIn June last year, Keystone Pharmaceuticals acquired the clinical development and promotion interests of avapritinib in Chinathree different doses of avapritinib compared to placebo resulted in a significant lyser trypsin level significantly lower than baseline (Photo: Source: Supplied source, 2)phase 2 trial called PIONEER was a randomized, double-blind, placebo-controlled clinical studyTrial data as of November 12, 2019 showed that patients in the three different dose-based treatment groups of avapritinib had significantly and rapidly reduced serum trypsin levels compared to baselines and were maintainedThe data did not change at 12 weeks in patients in the placebo groupThe decrease in serum trypsin levels is part of the clinical remission assessment criteria for SM patients based on the IWG-MRT-ECNM standard Blueprint Medicines expects to begin screening patients in the second part of the PIONEER trial in the first half of 2020 "s PIONEER trial data for the treatment of patients with inertial SM further demonstrate the potential for avapritinib to become a new treatment option for all SM subtypes," said Dr Andy Boral, Chief Medical Officer, Blueprint Medicine References: , Blueprint Medicines Announces Initial Data From Phase 2 PIONEER Trial Trial of Avapritinib In Patients With Indolent Mastocytos Schilling Activity at All Levels, Retrieved December 9, 2019, from https:// 2? Symptoms, Lloyd's With Standard Therapy Origind December 9, 2019, from https:// Original title: ASH , Precision Anti-Cancer Therapy Phase 2 Data Actively, Treatment of Rare Immune Diseases
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