Promising data from the ongoing Phase I clinical trial Explorer has been announced by Blueprint Medicines Corporation. The trial is designed to evaluate the effects of the experimental drug avapritinib in the treatment of patients with systemic mastocytosis. The full article, which contains a more detailed report of the data, can be found here at PR Newswire.
Systemic mastocytosis (SM) is a form of mastocytosis that mainly affects adults. It is caused by an accumulation of mast cells in body tissues including the skin, bones, and organs. Mast cells are produced in the bone marrow and are involved in the immune system. People who are affected by SM may experience headaches, stomach pain, hives, and anaemia, amongst other symptoms. SM is also divided into four forms, each of which is associated with different typical effects. The four forms are indolent SM, SM with an associated hematologic non-mast cell lineage disorder, aggressive SM, and mast cell leukaemia. In the majority of cases of SM, the condition is caused by alterations to the KIT gene. The experimental treatment, avapritinib, functions by selectively inhibiting KIT and PDGFRA.
