Ongoing research into pediatric brain tumours @  Johns Hopkins University

Master Gene Regulatory Pathway Revealed as Key Target for Therapy of Aggressive Pediatric Brain Cancer - neuroinnovations

Working with cells taken from children with a very rare but aggressive form of brain cancer, Johns Hopkins University scientists have identified a genetic pathway that acts as a master regulator of thousands of other genes which spur cancer cell growth and resistance to anticancer treatment.

AT/RT mostly strikes children 6 and younger, and the survival rate is less than 50 percent even with aggressive surgery, radiation and chemotherapy, treatments that can also disrupt thinking, learning and growth. AT/RT accounts for 1 percent of more than 4,500 reported pediatric brain tumours in the U.S., but it is more common in very young children, and it represents 10 percent of all brain tumours in infants.

The team state that the study also identified new ways in which to treat AT/RT with experimental drugs already being tested in pediatric patients. Because few outright genetic mutations, and potential drug targets, have been linked to AT/RT, the researchers turned their attention to genes that could regulate thousands of other genes in AT/RT cancer cells. Proof Of Concept experiments in fruit flies had already suggested a gene known as LIN28 could be important in regulating other genes involved in the development of brain tumours. Specifically, the LIN28 protein helps regulate thousands of RNA molecules in normal stem cells, giving them the ability to grow, proliferate and resist damage.

These factors provide stem cells with characteristics that cancer cells also have, such as resistance to environmental insults. These help tumour cells survive chemotherapy and radiation.   These proteins also help stem cells move around the body, an advantage cancer cells need to metastasize.

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