Based on significant growth and productivity as a program in development during the last funding period, Neuro-oncology (NO) was recently elevated from an HICCC Program in Development to a Full Program. The broad goal of the NO Program is to optimize diagnostic and therapeutic strategies for brain tumors by applying the expertise of a multidisciplinary team towards translating knowledge of brain tumor systems biology, genetics and cell biology. This goal is motivated by the recognition that molecular/genetic insight into brain tumors has the potential for development of individualized patient-specific treatment strategies. To advance its goal, the NO Program is organized around three interactive themes: 1) expanded novel tumor target discovery based on greater understanding of regulatory networks, cellular transformation and micro-environmental influences;2) improved translational therapeutic studies through development of preclinical animal models, high throughput drug screening and novel drug delivery strategies;and 3) refinement of a multidisciplinary clinical center with advanced tissue banking and database collection to facilitate molecularly stratified clinical trials. Major strengths of the program include expertise in systems biology with demonstrated translational application, innovative stem cell/progenitor cell biologists focusing on therapeutic target discovery in brain tumors, and innovative leaders in the fields of animal models of glioma and advanced drug delivery strategies with demonstrated abilities to translationally exploit therapeutic testing. Additionally, the Program maintains a robust brain tumor tissue bank specifically designed to facilitate the translation basic science discoveries into preclinical models and comprehensive clinical trials with correlative molecular pathological studies. The Program has fostered these goals through organization of collaborative investigators, recruitment of new members, support of intra-programmatic meetings and use of shared tissue bank/database resources. The NO program consists of 20 Program members (14 full) representing 9 departments within the College of Physicians and Surgeons and combining a spectrum from basic science to clinical proficiency. During the last budget period (July 1, 2012 - June 30, 2013), the NO Program received a total of $4.7M (direct costs) in cancer-relevant grant support, including $1.5M (direct costs) in NCI funding, $1.9M (direct costs) in other peer reviewed cancer-related support and $1.2M (direct costs) in cancer-relevant non-peer reviewed funding. There were a total of 173 Program publications from 2008 to the present, of which 32% were intra-programmatic and 20% were inter-programmatic and/or collaborative with investigators in other institutions;12% were published in journals with Impact Factor >10, and 5% in journals with Impact Factor > 20.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Center Core Grants (P30)
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Subcommittee G - Education (NCI)
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Columbia University (N.Y.)
New York
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Bassuk, Alexander G; Sujirakul, Tharikarn; Tsang, Stephen H et al. (2014) A novel RPGR mutation masquerading as Stargardt disease. Br J Ophthalmol 98:709-11
Li, Yao; Wu, Wen-Hsuan; Hsu, Chun-Wei et al. (2014) Gene therapy in patient-specific stem cell lines and a preclinical model of retinitis pigmentosa with membrane frizzled-related protein defects. Mol Ther 22:1688-97
Wert, Katherine J; Sancho-Pelluz, Javier; Tsang, Stephen H (2014) Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa. Hum Mol Genet 23:514-23
Shen, Sherry; Sujirakul, Tharikarn; Tsang, Stephen H (2014) Next-generation sequencing revealed a novel mutation in the gene encoding the beta subunit of rod phosphodiesterase. Ophthalmic Genet 35:142-50
Palomero, Teresa; Couronné, Lucile; Khiabanian, Hossein et al. (2014) Recurrent mutations in epigenetic regulators, RHOA and FYN kinase in peripheral T cell lymphomas. Nat Genet 46:166-70
Higuchi-Sanabria, Ryo; Pernice, Wolfgang M A; Vevea, Jason D et al. (2014) Role of asymmetric cell division in lifespan control in Saccharomyces cerevisiae. FEMS Yeast Res 14:1133-46
Lam, A T; Curschellas, C; Krovvidi, D et al. (2014) Controlling self-assembly of microtubule spools via kinesin motor density. Soft Matter 10:8731-6
Olszak, Torsten; Neves, Joana F; Dowds, C Marie et al. (2014) Protective mucosal immunity mediated by epithelial CD1d and IL-10. Nature 509:497-502
Murtomaki, Aino; Uh, Minji K; Kitajewski, Chris et al. (2014) Notch signaling functions in lymphatic valve formation. Development 141:2446-51
Nong, Eva; Lee, Winston; Merriam, Joanna E et al. (2014) Disease progression in autosomal dominant cone-rod dystrophy caused by a novel mutation (D100G) in the GUCA1A gene. Doc Ophthalmol 128:59-67

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