What patients and families need to know
The Children's National Research Institute
- Ph.D., Molecular Genetics, Northwestern University, Chicago, IL (2004-2010)
- B.S., Biology, Indiana University, Bloomington, IN (2000-2004)
Assistant Research Professor of Pediatrics, George Washington University School of Medicine and Health Sciences
Dr. Christopher Heier's goal is to develop next generation treatments and biomarkers for diseases in children. His particular focus is on genetic diseases of muscle (such as Duchenne muscular dystrophy), but by targeting the underlying molecular biology of disease Dr. Heier believes we can help a broad array of human illnesses. For example, his lab is currently dissecting molecular pathways involved in steroid signaling. This has helped to develop a new anti-inflammatory and heart-protective drug named vamorolone (previously named VBP15) to the point that it is now in clinical trials for Duchenne muscular dystrophy. Early work indicates this drug has the potential to replace prednisone as the standard of care for muscular dystrophy, by providing a drug that selectively targets the same receptors to be more effective while also being much safer. Through a better understanding of drug pathways, Dr. Heier's laboratory is also developing serum-based biomarkers reflective of drug activities in patients. Ultimately, this approach has the power to greatly improve the treatment of a large number of diseases featuring chronic inflammation and/or cardiomyopathy.