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Rare Blood Disease Drug Win Marks Fourth Approval for Blueprint Medicines
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FDA Approves Blueprint Medicines AYVAKIT™ (avapritinib) for the Treatment of Adults with Advanced Systemic Mastocytosis
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Three-quarters of patients with the rare blood disorder advanced systemic mastocytosis responded to treatment with avapritinib (Ayvakit), an interim analysis of a phase II trial found.
Among 32 patients treated with daily avapritinib in the PATHFINDER study, 19% had a complete remission (CR) with partial hematologic recovery, 31% had a partial response, and 25% achieved stable disease, with responses observed in all disease subtypes, reported Daniel DeAngelo, MD, PhD, of the Dana-Farber Cancer Institute in Boston. Avapritinib at a starting dose of 200 mg induced rapid, durable, and improving responses, DeAngelo said during his presentation at the American Association for Cancer Research virtual meeting. Reductions were seen in disease burden based on bone marrow mast cells, reduction in serum tryptase,
Session Time: April 13, 11:17-11:39 a.m. EST
Summary: Kaelin will highlight the important physiologic constraints a cancer tissue must operate within, focusing on the critical role hypoxia plays in not only tumor growth but progression, and how modulating the hypoxia machinery will limit tumor progression. The ability to target cancer at several molecular levels provides hope that our therapies will not only be more effective but also more durable. The lingering question is how to deploy these approaches by identifying which patients are most likely to benefit.
In addition to the exciting research presentations, Andrew Aguirre, MD, PhD, of the Dana-Farber Center for Gastrointestinal Oncology, has been named a 2021 AACR NextGen Star. Aguirre will present during the NextGen Stars Spotlight Session: Genetics and Epigenetics.
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There is no stronger risk factor for cancer than age. At the time of diagnosis, the median age of patients across all cancers is 66. That moment, however, is the culmination of years of clandestine tumor growth, and the answer to an important question has thus far remained elusive: When does a cancer first arise?
At least in some cases, the original cancer-causing mutation could have appeared as long as 40 years ago, according to a new study by researchers at Harvard Medical School and the Dana-Farber Cancer Institute.
Reconstructing the lineage history of cancer cells in two individuals with a rare blood cancer, the team calculated when the genetic mutation that gave rise to the disease first appeared. In a 63-year-old patient, it occurred at around age 19; in a 34-year-old patient, at around age 9.
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