Rare tumor research at NIH: Strategies to accelerate development of therapies

Wednesday, September 24, 2014 — Concurrent Symposia Session IV
12:00 p.m. – 2:00 p.m.	Classroom 4

Co-chairs

• Abby Sandler, NCI
• Karlyne Reilly, NCI

Program

NCI launched the Rare Tumors Initiative (RTI) in 2013 to foster closer collaborations between basic and clinical/translational researchers to develop therapies for rare tumors. Studying rare tumors poses challenges that apply to many diseases studied across NIH. These challenges include a paucity of human samples to study underlying mechanisms of disease, and difficulty accruing the number of patients needed to adequately power clinical trials. As biomedical research moves toward a precision medicine model, each individual becomes their own "rare disease" and new approaches to testing and validating therapies must be developed. At this symposia, we will report on the first two pilot studies of the RTI on desmoid tumors and malignant peripheral nerve sheath tumors, and highlight other research on rare tumors being done at NIH.

Development of MEK inhibitors for neurofibromatosis type 1 related plexiform neurofibromas
Brigitte Widemann, NCI

Desmoids: A rare disorder characterized by aggressive fibromatosis
Shivaani Kummar, NCI

Mosaic overgrowth disorders as a model system for cancer therapeutics
Leslie Biesecker, NHGRI

Modulators of therapeutic response in glioblastoma
Jayne Stommel, NCI

Headways and hangups in Diffuse Intrinsic Pontine Glioma
Kathy Warren, NCI

CDCA7L functions as a male-specific oncogene in astrocytoma; FARE award winner
Min-Hyung Lee, NCI

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