I remember the day I couldn’t see out of my left eye. I became obsessed with waving my hand in front of my face, searching for the sweet spot where I could make it disappear. Earlier that year I suffered through a bout of Idiopathic Uveitis. Believing it had returned, I went to the ER expecting to be given a script for steroid eye drops and then be on my way. Wrong! I was told that my optic nerve was swollen and apparently that’s a big deal; I was admitted.
The Children’s Tumor Foundation NF Endurance Team gives participants the opportunity to run, bike, or swim in endurance events across the country. We asked one of our athletes, Dawn Lowell, why she races with us and what it means to her family.
Every year, on the last day of February, we recognize Rare Diease Day, a time for the entire rare disease community to come together to raise awareness. NF affects 1 in 3,000 people overall; NF2 is even more rare (1 in 25,000) and schwannomatosis even more rare (1 in 40,000). These days, there is a lot of excitement and hope for the MEK clinical trial for inoperable NF1 plexiform tumors (click here to read about the trial). Over 70% of patients had a dramatic response and saw their tumors shrink 20% or more. This is unprecedented and really makes us hope that this drug will soon be approved by the FDA as the first-ever treatment for NF1. But what about NF2? Even though it's the MEK trial making headlines in NF these days, there is a lot going on also for NF2. In recognition of Rare Disease Day, we're sharing these stories about two adults living with NF2 and their experiences in the Avastin (Bevacizumab) trial.
By Salvatore La Rosa, PhD, Children’s Tumor Foundation VP, Research and Development
These days there is a lot of excitement and hope for the MEK clinical trial for inoperable NF1 plexiform tumors (click here to read about the MEK trial). Over 70% of patients had a dramatic response and saw their tumors shrink 20% or more. This is unprecedented and really makes us hope that this drug will soon be approved by the FDA as the first ever treatment for NF1. But what about NF2? What is the status in the clinic for this condition? Well, I would like to focus on four drug trials that are ongoing and that promise to bring great news for our patients in the short term.
This month marks the eighth consecutive Cupid’s Undie Run to benefit the Children’s Tumor Foundation. Cupid’s has grown in leaps and bounds since 2010, when participants raised $10,000 to now, in 2017, when they’ve brought their eight year total to over $10 million for NF research. Everyone’s favorite Valentine’s-themed party has shown us all once again what an amazing group of people can do for the NF cause.