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Targeting the LIN28 Pathway in ATRT

The Johns Hopkins University School of Medicine
Eric Raabe, MD, PhD
Grant Type: 
'A' Award Grants
Year Awarded: 
Type of Childhood Cancer: 
Atypical Teratoid/Rhabdoid Tumor (AT/RT), Brain Tumors
Project Description: 


Atypical teratoid rhabdoid tumor (ATRT) is one of the most aggressive pediatric brain cancers. We do not understand why it is so aggressive. Unlike many cancers, it has only one recurrent mutation (in the INI1 gene). Our group has identified in ATRT increased expression of a gene called LIN28, which can regulate many other genes. When we suppress LIN28, ATRT cancer cells die.

Project Goal

This proposal will investigate why LIN28 is so important to ATRT cancers, and how LIN28 might work with INI1 mutation to make ATRT grow. We have also found drugs that target genes that are turned on by LIN28, and we will investigate if combinations of these drugs will kill ATRT cells. Together our experiments will teach us why ATRT is so bad, and how to kill ATRT cancer cells.


"Funding from Alex's Lemonade Stand will help us better understand atypical teratoid/rhabdoid tumors and find new treatments to help patients who currently have no curative therapeutic options."   --Eric Raabe