Clinical trial aims to improve outcomes for children and young adults with primary brain tumors

Dr holding child's hand

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Children and young adults with primary brain tumors that have recurred or continued to grow after previous treatment may be eligible to participate in a new clinical trial at the NIH Clinical Center.

Central nervous system (CNS) tumors are the most common solid tumors among children and account for up to 25 percent of all childhood cancer cases. Although overall survival for children with tumors of the CNS has gradually improved, for some tumors, such as diffuse intrinsic pontine gliomas and high-grade gliomas, 5-year survival is less than 10 percent.  With few treatment options available at the time of recurrence or progression, Kathy Warren, M.D., of the Pediatric Oncology Branch aims to collect information that may help researchers learn more about treatments that could benefit children and young adults with high-grade glioma, medulloblastoma, ependymoma and diffuse intrinsic pontine glioma (DIPG).

This phase II, multicenter study will test pomalidomide, a drug that boosts the function of the immune system to fight tumors, to assess the efficacy, safety and tolerability of the drug, developed by Celgene.

The Center for Cancer Research is NCI’s internal cancer center, a publicly funded organization working to improve the lives of cancer patients by solving important, challenging and neglected problems in cancer research and patient care.  Highly trained physician-scientists develop and carry out clinical trials to create the medicines of tomorrow treating patients at the world’s largest dedicated research hospital on the campus of the National Institutes of Health in Bethesda, Maryland.  

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Summary Posted: 03/2018