The Johns Hopkins University School of Medicine

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Baltimore, MD 21205

United States

Targeting the LIN28 Pathway in ATRT

Brain Tumors, Atypical Teratoid/Rhabdoid Tumor (AT/RT)

Background

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.

Using a novel epigenetic therapy to drive differentiation of atypical teratoid rhabdoid cells and facilitate immunotherapy

Atypical Teratoid/Rhabdoid Tumor (AT/RT)

Atypical teratoid rhabdoid tumor (ATRT) is the most common malignant brain tumor of babies. We use very intensive chemotherapy, including stem cell transplant and even radiation, but we only cure a minority of patients. Those we do cure have life-long development and learning problems. We desperately need new therapies. Our current treatment does not address the root cause of ATRT, which is due to loss of function of a gene that helps cells decide when they should grow and when they should differentiate or mature.

Alex's Lemonade Stand Foundation for Childhood Cancer