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The Press On Fund has made a 4-year, $1 million pledge in the first study to examine the efficacy of a drug that inhibits an enzyme certain pediatric brain tumors use to protect themselves from a natural immune response. This study will benefit children with recurrent brain tumors or newly diagnosed DIPG, a particularly aggressive brain tumor.

Drs. Theodore Johnson and David Munn, pediatric oncologists at the Medical College of Georgia and Georgia Cancer Center are the lead investigators on this exciting clinical trial; Press On and the CAM FUND previously invested $150,000 in an earlier phase of the study, which is exploring whether an inhibitor of the enzyme indoleamine 2,3 dioxygenase, or IDO, will allow for lower doses of chemotherapy and radiation, better quality of life during treatment, and still achieve improved survival for these children.

The idea is to unleash a natural immune response to attack the tumor. “We also have evidence that the addition of an IDO inhibitor to these children’s treatment will help us reduce the toxicity often associated with aggressive cancer treatment, improve quality of life during treatment and in some cases offer at least the potential for cure,” Johnson says. Standard therapies cure about 60 percent of children with these brain tumor types, and the investigators hope their new approach can help the 40 percent who have recurrent disease.
Adding the IDO inhibitor Indoximod to the mix helps standard treatments work better — even at a lower dose — by setting the immune system free to respond as it should.

For example, children in the study will be taking a low-dose, once daily chemotherapy pill known to have relatively modest side effects. In their first-in-humans phase 1 trial, which focused more on safety than efficacy, the investigators saw children finish high school, take their SATs and start and do well in college while on the protocol. “You wouldn’t be able to do that if you were getting high dose, intravenous chemotherapy,” Munn says.

The trial also will provide frontline treatment to 30 children with newly diagnosed diffuse intrinsic pontine glioma, or DIPG, a brain tumor for which there is no standard therapy considered curative. For these children, the trial pairs the Indoximod and radiation followed by maintenance therapy with chemotherapy and Indoximod, with the goal of improved overall survival compared with current approaches, which typically produce only a short-term response.