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Big advance against a vicious pediatric brain tumor

New Stanford research is providing the first advance in 35 years toward treatment of a vicious pediatric brain tumor. A study appearing today in Proceedings of the National Academy of Sciences describes development of the first ever in vitro and animal models of the tumor, diffuse intrinsic pontine glioma.

The new research tools are badly needed. DIPG has a horrific prognosis - the five-year survival rate is one percent. Without cell or animal models, scientists have been unable to learn how this tumor of school-aged children develops, so they've had great difficulty identifying and testing potential drug therapies. DIPG is impossible to remove surgically, as the tumor cells grow entwined with healthy cells in a part of the brain stem essential for life. Radiation merely buys patients a bit of extra time. And no effective drugs are known, though pediatric neurologist Michelle Monje, MD, PhD, who led the Stanford team, hopes her work will soon change that.

Monje's research has an unusual human element, as the press release I wrote about the new findings explains:

The advances come thanks to the parents of young cancer victims, who donated their deceased children’s brain tumors for research in the hopes of sparing other families the pain they had experienced. Because of its location in the brain stem, this cancer is rarely biopsied, so scientists have had few previous opportunities to examine the tumors.

A diagnosis of DIPG is “a death sentence for kids,” said John Jewett, whose son Dylan died of DIPG in 2009 at age 5. “No parent should have to hear that, but doctors can’t study the disease unless somebody makes a donation.” John and his wife, Danah, were the first parents to donate their child’s tumor to the Stanford team.

Dylan's tumor cells and those of a second DIPG patient were grown in culture, then injected into mice to produce a xenograft model of DIPG. The researchers have already used the cultured cells to identify a possible molecular target for anti-DIPG drugs, which they soon plan to test in the mouse model. And they're ready to share the their advances with scientific collaborators at other institutions, too.

Philip Beachy, PhD, the new study's senior author, sums up the whole team's feelings about the value of the new work for advancing DIPG treatment: “Just having this model makes possible a real attack on the problem."

Previously: Video profiles work of pediatric brain tumor researcher and New Stanford trial targets rare brain tumor

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