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.
I am 66 years old. I have NF1 and it causes bouts of depression, but I deal with that. I am a fortunate man. I taught school for 35 years having earned my Bachelor’s and Master’s degrees. I am married with no children.
By Cristina Kline
“Cyclopes, one-eyed monster, Hunchback of Notre Dame!”
These are a few names I would hear throughout my school-age years. Kids can be mean, but being a kid myself at that time I didn’t understand. I played it tough. I pretended it didn’t bother me. I was strong. Those were just words but you know what? Words hurt. I’d cry in secret when nobody was watching. I wouldn’t tell my friends or family what I was going through and how it affected me. I had to be strong for them. I knew if I told my family they would suffer too. So, I kept it to myself. I kept it to myself for many years.
I was diagnosed with Neurofibromatosis Type 1 (NF1) when I was two years old. I am now 31 years old.