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The new strategy could help fight the deadliest brain tumors in children

News from March 4, 2019 — A new study, published in Nature Communications, found that inhibiting an enzyme called ACVR1 slowed tumor growth and prolonged life in animal models of diffuse pontoglioma (DIPG), the deadliest brain tumor in children, and that no drugs have been approved to treat DIPG. So this study offers hope for developing potential new therapies.




"Our results are encouraging and suggest that it is possible to test this enzyme inhibitor clinically." Dr. Oren Becher, the study's corresponding author and an associate professor at the Stanley manler children's institute at Ann and Robert lowry children's hospital in Chicago and the feinberg school of medicine at northwestern university. "Until then, we need to evaluate the effects of different inhibitors in animal models to ensure that the safest and most effective medicine are delivered to children in clinical trials."

In 2014, Becher's lab was involved in finding that 25% of DIPG patients had ACVR1 mutations, which cause the enzyme to overactivate. In the present study, Dr. Becher and his colleagues have shown for the first time that mutations in this enzyme are associated with a histone mutation in 20% of tumors in an animal model (h3.1k27m). These mutations are crucial for initiating tumorigenesis.

Histones act as a sort of DNA axis, helping to assemble the DNA into the nucleus of each cell. Histones also switch genes on and off, a process that can go wrong when histones are mutated. "Our future work will explore why simultaneous mutations of histone and ACVR1 lead to DIPG and how to facilitate this process." "Dr. Becher said. "Exploring this process more deeply will make it easier and easier for us to develop drugs to treat children with DIPG."

There is no medicine is approved for the treatment of DIPG. So this study offers hope for developing potential new therapies.
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