Calling Mastocytosis patients & loved ones to action: Start Avapritinib trial ASAP

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Indolent and smoldering systemic mastocytosis are rare conditions caused by the accumulation of mast cells in the body’s organs, including the bone marrow and skin. Mast cells are an important type of immune cell that normally play a role in the body to help fight infection and trigger the allergic response. In systemic mastocytosis, mast cells are inappropriately released, causing many symptoms that can negatively impact daily life. People with indolent and smoldering systemic mastocytosis often experience severe symptoms which can include rashes, GI problems, fatigue, “brain fog,” severe allergies, risk of anaphylaxis, bone pain, osteoporosis and more.

Systemic mastocytosis is classified into five main subtypes – indolent SM, smoldering SM, aggressive SM, SM with associated hematologic neoplasm, and mast cell leukemia. Across all these subtypes, up to 95% of people have a specific mutation (genetic alteration) in a gene called KIT. The mutation, known as KIT D816V, plays a central role in the activation and accumulation of mast cells in people with systemic mastocytosis. Avapritinib (BLU-285) developed by Blueprint Medicines is an investigational medicine that was specifically designed to block the KIT D816V mutation.

PIONEER is a phase 2 clinical trial that will compare the safety and effectiveness of  avapritinib against a placebo in people with indolent or smoldering systemic mastocytosis (SM) whose symptoms aren’t adequately controlled with standard therapies. The trial's sponsors (BLUEPRINT medicines & St. Michael's Hospital) been holding off the trial for months now. PIONEER has been approved and is running successfully in 7 other countries: the Netherlands, the UK, Spain, Italy, Norway, the US, Korea, Belgium. People with a serious system-wide disease - Mastocytosis are being prevented from getting treated because the trials' sponsors can't get their act together. Please sign this petition if you want PIONEER to start as soon as possible.