Development of Bone Morphogenetic Protein Inhibitors to Treat Blood and Bone Disorders

National Center For Advancing Translational Sciences

Investigators

Abstract

Bone morphogenetic protein (BMP) is a protein essential for normal bone growth and other functions in the body. Gene defects that cause too much BMP activity can lead to two diseases: fibrodysplasia ossificans progressiva (FOP) and anemia of inflammation (AI). FOP is a rare, inherited and deadly disease in which muscles and tendons turn to bone, making movement difficult. AI is a more common condition in which too much BMP activity in the liver causes anemia. No completely effective treatments exist for either disease. The investigators are developing a drug that inhibits BMP activity and could be used to treat FOP and AI. This projects aim is to prepare the drug for testing in humans. The team is collaborating on the completion of the following studies: - Synthesis of Good Manufacturing Practice (GMP) and non-GMP material - Formulation development - Pharmacokinetic/absorption, distribution, metabolism, and excretion (PK/ADME) studies - Investigational New Drug (IND)-directed toxicology