PEGylated talazoparib for pediatric malignant rhabdoid tumor therapy

Malignant rhabdoid tumors (MRT) were initially described in 1978 as a rare and highly malignant childhood cancer that often starts in the kidneys, but may be found in other soft tissues such as the brain. Such tumors are typically seen in infants and toddlers, and carry a common genetic change – loss of the SMARCB1 gene which normally helps to control how healthy cells grow, divide, and die. The current standard therapies include surgery, chemotherapy, and radiation; however, it is generally ineffective as only about 10% of children who are diagnosed before 6 months of age survive. With funding from CURE Childhood Cancer, Dr. Raushan Kurmasheva from the University of Texas at San Antonio will test several promising drug combinations in novel preclinical models including some created by this group.  In particular, they are focused on leveraging knowledge that MRTs typically lack the SMARCB1 gene. Upon successful completion of the proposed project, we anticipate moving quickly to COG (Children’s Oncology Group) or POETIC (Pediatric Oncology Experimental Therapeutics Investigators’ Consortium) for clinical trials.