Researcher Seeks to Understand the Inner Workings of Rhabdoid Tumors

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Alex Kentsis, MD, PhD


Project: Tumorigenesis by Endogenous DNA Transposons in Rhabdoid Tumors

Alex Kentsis
  • Alex Kentsis, MD, PhD
  • Pediatric Oncologist / Investigator
  • Sloan-Kettering Cancer Center

Rhabdoid tumors are one of the most lethal childhood cancers. Rhabdoid tumors affect mainly infants and young children and can develop in the kidneys, liver, soft tissue, and brain. Most children diagnosed with a rhabdoid tumor that cannot be completely removed through surgery do not have effective treatment options.

Alex Kentsis, MD, PhD, a pediatric oncologist and investigator at the Memorial Sloan Kettering Cancer Center is interested in understanding the genetic make-up of these tumors, focusing on DNA sequences called transposons that can potentially move within cell genomes. Almost half of the human genome is made up of DNA derived from ancient transposons, but their activity in tumor cells is not understood. Dr. Kentsis has found that, in rhabdoid tumors, some of these transposons appear to be mobile and potentially contribute to tumor growth and survival in response to chemotherapy. Dr. Kentsis is using specially developed methods to analyze the mobile DNA in the rhabdoid tumor genome to more fully understand how it impacts the tumor’s biology. He is then using this information to study human malignant rhabdoid tumors in genetically-engineered mice and zebrafish. Dr. Kentsis hopes that if he can successfully block tumor DNA transposition in animal models, this approach can eventually be translated into new therapies for children with rhabdoid tumors. Dr. Kentsis also hopes that his study of mobile DNA in rhabdoid tumors will lead to a deeper understanding of genetics of other tumors.

As of the conclusion of his CureSearch-funded project, Dr. Kentsis has identified new promising therapeutic options for malignant rhabdoid tumor:

  • 1 novel drug target identified for malignant rhabdoid tumor
  • 1 new transgenic mouse model developed
  • 1 drug in preclinical testing for treatment of rhabdoid tumors

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