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.
After receiving approval from both chambers of Congress earlier this month, President Trump signed the FY 2019 Defense and Labor, Health, and Human Services Appropriations conference report into law on Friday, September 28. This sustained federal funding will allow us to further develop scientific data, break through barriers, and forge a pathway to end NF.
WORKSHOP RECAP: The Growing Promise of Gene Therapy Approaches to Rare Diseases
On Aug 20-21, 2018 the National Center for Advancing Translational Sciences (NCATS) and FDA’s Center for Biologics Evaluation and Research (CBER) hosted a workshop on gene therapy in Bethesda, MD. Titled ‘The Growing Promise of Gene Therapy Approaches to Rare Diseases,’ the workshop was attended by experts from academia and industry, NIH and FDA staff, representatives from several rare disease foundations, and other stakeholders. The two-day event was filled with presentations and panel discussions on advancements and challenges in preclinical and clinical development of gene therapy products, vector manufacturing processes, public-private partnerships including with research foundations, business models, patient access especially in very low prevalence diseases, and reimbursement models.
International collaborative research led by Ludwine Messiaen, PhD, shows that while a three-base pair, in-frame deletion called p.Met992del in the NF1 gene has a mild phenotype for people with the genetic disorder neurofibromatosis type 1, or NF1, the mutation does cause complications. CTF has invested in Dr. Messiaen’s promising work by providing the Isaac and Sadie Fuchs Genotype-Phenotype Grant.
Ask Kate! is a new weekly YouTube series to answer your questions about neurofibromatosis! Kate Kelts, RN, BSN is the Patient Support Coordinator for the Children's Tumor Foundation, and each week she will answer questions submitted directly by viewers. If you have a question you'd like to see Kate answer, you can include it in the YouTube comments of the latest video, or email Kate at KKelts@ctf.org.
Click here to subscribe and click the bell to receive alerts when new videos are posted. Click "Read More" to view the first two videos in this exciting new YouTube series!