New Target Found to Attack an Incurable Brain Tumor in Children

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Restoring the tumor suppressor gene p16 slows tumor growth in vitro

A study reveals that a tumor suppressor gene p16 is turned off by a histone mutation (H3.3K27M), which is found in up to 70 percent of childhood brain tumors called diffuse intrinsic pontine glioma (DIPG). This insight suggests that restoring p16 is a promising therapeutic strategy. The authors have demonstrated that this can be accomplished in vitro using a drug that is approved for treatment of adult leukemia and other cancers. Histone is a protein that acts like a spool for DNA, helping to package the six-foot long DNA strand into the tiny nucleus of every cell. Histones also help regulate which genes turn on and off, a process that goes awry when there is a histone mutation. “Using a genetic mouse model of DIPG, we found that the histone mutation turns off p16, which is a gene that acts like a break on dividing cells,” says senior author Oren J. Becher, MD, from Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago.

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