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NF NEWS V ol ume I V, 2013 T h e N e w s l e t t e r o f t h e C h i l d r e n ’ s Tu m o r F o u n d a t i o n Successful Benefit Galas Raise Funds for NF Research On October 26th, “The beNeFit: A Celebration to Beat NF” was held in Detroit and on November 7th, the Children’s Tumor Foundation’s Annual Benefit Gala was held in New York City. These events raised over $3 million for NF research. Detroit held its inaugural Children’s Tumor Foundation gala with the theme “A Glamorous Gatsby Affair.” Over 900 guests decked out in their favorite 1920s attire filled Cobo Center’s brand new Grand Ballroom, overlooking the Detroit River. The Great Gatsby himself would have been proud. Joined by local dignitaries, sports stars, and business leaders, Quicken Loans and Rock Ventures Founder and Chairman Dan Gilbert and Children’s Tumor Foundation President John Risner welcomed guests with heartfelt words and stressed the urgency of defeating NF. Featuring silent, live, and research award auctions, The beNeFit raised more than $2 million toward finding a cure for NF, of which every dollar will be invested efficiently and effectively. NF Hero Victor Chukwueke The highlight of the night was Victor Chukwueke, who was born in a small Nigerian village and endured nine major surgeries to remove the tumors on his face as a result of NF. Brought to the United States by missionary nuns, the teenager overcame Continued on page 15 The beNeFit: A Celebration to Beat NF Accelerating the Delivery of Treatments to the Clinic Launched in 2008 and executed in two phases, the Children’s Tumor Foundation Neurofibromatosis Preclinical Consortium (NFPC) was a collaboration of six research centers, each using genetically-engineered animal models of NF1 and NF2 tumors with the common goal of rapidly advancing promising therapies to clinical trials. The NFPC for NF1 completed its second phase in June/July 2013. The NF2 group will conclude activities by the end of 2013. During the past five years (2008-2013), the Consortium collaborated to perform and complete 52 preclinical trials across different models. Seven trials are ongoing and will be completed in the next few months. These studies identified 16 drugs or drug combinations with promising efficacy. As a direct result, ten clinical trials have been started and four additional ones are under evaluation or discussion to start soon. This is a HUGE success considering that typically only 2-5% of preclinical molecules enter into clinical development. The preclinical group has focused not only on finding novel targets for clinical trials, but also on testing optimal compounds and dosing schedules to better inform the clinic, and improving combinations with drugs that already give some positive anti-tumor effects. The only drawback with the current molecules is that they are often affecting similar mechanisms or pathways. That is why it is extremely important to continue this research, and the Foundation is pleased to no longer be alone in funding this effort. For NF1, the Foundation, in conjunction with the Neurofibromatosis Therapeutic Acceleration Program (NTAP) at Johns Hopkins University, has agreed to continue funding the Consortium for the next three years (2013-2016). The new Consortium is now called the NF Therapeutic Consortium (NFTC). The objective of this consortium is to find promising NF-relevant molecules that are in development or on the market for other indications, test the concept in the animal models, and get the molecules to the clinic. For NF2, the current NF Preclinical Consortium will terminate at the end of the year. A follow-up consortium is currently under construction.

Children's Tumor Foundation Newsletter, Volume IV

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