Speaking before the House Energy and Commerce Committee of the U.S. House of Representatives on the implementation of the 21st 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.
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.
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.
The Children’s Tumor Foundation is an Associated Partner of the European-based Innovative Medicines Initiative
The Innovative Medicines Initiative (IMI), a jointly funded partnership between the European Union (EU) and the European Federation of Pharmaceutical Industries and Associations (EFPIA), will soon issue a call for an integrated research platform enabling patient-centric drug development.
In May 2018, the NF community gathered in Atlanta, GA for a long weekend of patient engagement training, leadership training, research updates, and patient and family support. We asked two first-time attendees to reflect on their experience.