UW-Madison researchers have created an animal model for studying Wilms Tumor by using Wilms Tumor cells from patients. The researchers reprogrammed the patient tumor cells into induced pluripotent stem cells. When the iPSCs were transplanted into the kidney capsules in mice, tumors developed, and the tumors metastasized to the lung and liver. The tumors also expressed gene markers indicating their blastemal lineage. This model displays characteristics similar to those observed in physiological Wilms Tumor.
The inventors also tested some drugs against their new model and found that the gold standard WT treatment was more effective in killing the tumor cells in their model as compared to other WT cell lines. They screened additional molecules and showed that a generic molecule, Cytarabine, presently prescribed for various leukemias also showed tumor cell killing efficacy against their cell model. Another molecule effective against their cell model was Panobinostat approved for treating multiple myeloma in 2015. That drug was pulled from the US market in 2022.