- Mastocytosis is a disease characterized by an abnormal accumulation of mast cells, a type of white blood cell, located in peripheral tissues and organs. Mast cells store components that mediate inflammatory and allergic responses.
- There are several subtypes of the disease, and outcomes can vary significantly by subtype. While the exact number of patients suffering from all forms of mastocytosis, including urticaria pigmentosa, is not known, it is estimated that about 3,000 patients are newly diagnosed each year in the United States and about 30,000 patients live with the disease in the United States.
- Approximately 94% of adult patients with systemic mastocytosis are reported to have a mutation in KIT ( D816V) which leads to unregulated KIT activation. The KIT receptor, which is widely expressed on mast cells, stimulates signaling pathways that control cell growth, differentiation and survival, and this mutation is believed to enable mast cells to proliferate in the absence of normal activation signals. In 2017 Midostaurin, which targets various kinases including KIT was approved in the U.S. to certain types of mastocytosis. Imatinib is an approved therapy for adult systemic mastocytosis patients lacking the KIT D816V mutation.
Living with Systemic Mastocytosis Webinar
Deciphera Pharmaceuticals hosted a webinar for the Systemic Mastocytosis (SM) community about Living with Systemic Mastocytosis, and Deciphera Pharmaceuticals’ ongoing clinical trial of DCC-2618, an investigational drug being testing in people with smoldering and advanced forms of Systemic Mastocytosis.
This webinar is provided for educational purposes only, is not medical advice and is not intended to replace the advice of a medical professional. This webinar contains forward-looking statements including, without limitation, concerning our clinical development program for SM, which are subject to a number of risks, including those identified in our SEC filings. This webinar was recorded on October 16, 2018 and we disclaim any obligation to update the information contained in it.
Our Fight for Patients – Systemic Mastocytosis
A great majority of patients with systemic mastocytosis are reported to have a specific KIT mutation enabling mast cells to proliferate. Our goal at Deciphera is to inhibit that KIT mutation thereby returning cell signaling to a normal activation state.
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