NF1 Clinical Trial of Selumetinib “Most Exciting” Says National Cancer Institute Director

Aug 16, 2018, Posted in Latest News, NF1, Science

Speaking before the House Energy and Commerce Committee of the U.S. House of Representatives on the implementation of the 21^st Century Cures Act, Norman (Ned) Sharpless, M.D., Director of the National Cancer Institute (NCI) was asked by Missouri Congressman Billy Long, “Can you tell us about the most exciting thing that is being supported in the Cancer Moonshot?”

Sharpless focused his answer on the NCI’s Rare Tumor Initiative and the clinical trial presented in June at the ASCO Conference by Andrea Gross, MD of the NCI about the selumetinib (MEK inhibitor) clinical trial treating inoperable NF1 plexiform neurofibromas.

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Selumetinib Granted Orphan Drug Designation by the European Medicines Agency

Aug 16, 2018, Posted in Latest News, NF1, Science

AstraZeneca and Merck/MSD have announced that the European Medicines Agency (EMA) has granted orphan designation to selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1). This follows the announcement earlier this year that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib.

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Leading the Way: Children’s Tumor Foundation Annual Report

Aug 7, 2018, Posted in Latest News

We’re proud to announce that our latest annual report, Leading the Way, is now available to read online. This report of the Foundation’s activities during the 2017 fiscal year highlight progress in research, awareness, patient support, and the accomplishments of a vibrant NF community in the fight to end NF. The financial pages offer transparency regarding CTF’s expenditures, which have earned the Foundation a four-star rating from Charity Navigator once again. And of course, none of this would be possible without the support of all of you – the thousands of donors who give to ensure that NF research and programs continue. 

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‘Pig Models’ Advance the Study and Treatment of NF1

Aug 2, 2018, Posted in CTF in the News, NF1, Published, Science, Synodos

Synodos for NF1 Research Published in Leading Journal

Children’s Tumor Foundation research led by its Synodos for NF1 team has demonstrated the ability to replicate NF1 manifestations in pigs, as reported in JCI (Journal of Clinical Investigation), a leading peer-reviewed publication of the American Society for Clinical Investigation. These manifestations include café-au-lait spots, neurofibromas, axillary freckling, and learning and memory neurological deficiencies. The significance of this advancement is that it allows researchers and clinicians, for the first time, to test and study potential NF treatments in large animal models that mimic human manifestations of neurofibromatosis.

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