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.
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.
Care of Adults with NF1, a new resource, has been published by the American College of Medical Genetics and Genomics (ACMG). It offers voluntary guidelines for medical management based on published reports and the expertise of the authors, who are leaders in NF1 medical care.
We are sharing this news with you as a resource to manage your own care. You can get this article free of charge at Springer Nature: https://rdcu.be/MFHJ
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.