Investigator Profile: Kimberly J. Riehle, MD, FACS, FAAP
Kimberly J. Riehle, MD, FACS, FAAP
Associate Professor, Division of Pediatric General Surgery
Liver Cancer in Children
Hepatocellular carcinoma (HCC) is the fifth most common cause of cancer death worldwide; developing effective therapies for HCC patients has been incredibly difficult due to a high variability in the genetic changes found in these cancers. Recently, however, a potentially causative mutation has been found in a subtype of HCC that occurs in children and young adults, fibrolamellar HCC (FL-HCC). FL-HCCs differ from other HCCs in that they arise in normal livers, whereas most HCCs develop in the setting of cirrhosis. Thus patients with FL-HCC are usually asymptomatic until their disease is very advanced; at that point cure is not possible. A newly discovered mutation in FL-HCC involves the active portion of a protein called protein kinase A (PKA), which was discovered decades ago and is critical to multiple diverse biologic processes. Dr. Riehle’s research focuses on understanding how this single mutation causes cancer in otherwise healthy patients. She has worked with investigators in the departments of pharmacology, pathology, and medicine to develop experimental model systems in which novel therapeutics can be tested, with the hope of developing a cure for these patients.