Cancer and Blood Disorders
The Genitourinary Tumors Program at the Gill Center is one of only a handful of its kind in the country. Over the last decade, the program has become known for exceptional clinical care – particularly surgical expertise – and notable scientific advances in understanding the molecular abnormalities of these cancers.
Pediatric genitourinary cancers are uncommon, accounting for just 10% of all childhood cancers. At the Gill Center, our medical team treats every type of genitourinary cancer, including germ cell tumor (GCT), Wilms tumor and other cancers arising in the kidney, ureter, testis, ovary and urinary bladder.
Each month a team of specialists, including urologists, pathologists, general surgeons, radiologists and others, convene to develop and review treatment plans for new and existing patients. The most common treatment is a combination of surgery and chemotherapy. Given the prominence of surgery in these treatment plans, our staff intentionally pursues advances in non-invasive surgery. For example:
Germ cell tumors (GCT)
While treatment for most GCT is effective – better than a 90% cure rate – the toxicity of chemotherapy creates an incentive to develop molecularly guided therapies that could be an alternative to chemotherapy.
James Amatruda, M.D., Ph.D., is among a small handful of researchers worldwide leading the way on germ cell tumor research. For years, the genes controlling the growth of GCT remained unknown. Over the past several years, Dr. Amatruda and his colleagues have begun to unravel the molecular circuitry of GCTs. His group developed a novel zebrafish model of GCT, and identified a genetic mutation in Bone Morphogenetic Protein (BMP) receptor, a cell signaling pathway, as the cause of GCTs in the model. They then validated the same pathway is defective in GCTs occurring in children. The Amatruda lab has also defined other biochemical signaling pathways that contribute to GCT growth. Through work with Dr. Ted Laetsch of UT Southwestern and the CCBD, this has now led to clinical trial testing a novel combination of molecularly-targeted therapies for chemoresistant GCTs.
Wilms tumor is a cancer of the kidney that occurs in toddlers and young children. Working in the Amatruda lab, Kenneth Chen, MD, along with UT Southwestern collaborators Dr. Dinesh Rakheja and Dr. Joshua Mendell, discovered a new class of mutations that cause almost ¼ of all Wilms tumor. These mutations affect the cell's production of microRNAs, tiny RNA molecules that are key regulators of cell growth. This work creates exciting possibilities for new treatments for Wilms tumors, based on emerging microRNA-based therapies. Drs. Chen and Amatruda are continuing with highly translational projects in order to bring discoveries from the bench to the bedside.