Researchers at The University of Texas MD Anderson Cancer Center , in collaboration with BostonGene, conducted the largest and most comprehensive molecular analysis of renal medullary carcinoma (RMC), a rare and aggressive form of kidney cancer, leading to the identification of TROP2 as a promising therapeutic target.
What is the key discovery in this research and why is it significant?
By evaluating 25 patient samples, the researchers identified overexpression of TROP2 and several other cell-surface proteins in RMC, as well as upregulation of the Hippo pathway. These findings led to the exploration of sacituzumab govitecan, a TROP2-targeted antibody-drug conjugate.
In a small cohort of four heavily pretreated RMC patients, sacituzumab govitecan resulted in one partial response and two patients with stable disease, with a median progression-free survival of 2.9 months.
“Identifying TROP2 as a therapeutic target in renal medullary carcinoma marks a pivotal step toward precision treatment for one of the most aggressive and underserved cancers,” said principal investigator Pavlos Msaouel, M.D., Ph.D. , associate professor of Genitourinary Medical Oncology.
What does this mean for future treatment options?
Renal medullary carcinoma is a fast-moving, treatment-resistant kidney cancer primarily affecting young individuals with sickle cell trait, highlighting the urgent need to understand its molecular basis to guide therapeutic development.
This study, which also characterized the RMC tumor microenvironment, supports further investigation of drugs targeting TROP2 as treatment options for this patient population.
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This study was supported in part by the National Cancer Institute (P30 CA016672), and by a strategic alliance between MD Anderson and BostonGene. For a full list of collaborating authors, disclosures and funding sources, see the full paper in Cell Reports Medicine .
Cancer Cell
30-Oct-2025