A group of patient advocates from the Children’s Tumor Foundation and the broader NF1 community are attending an NF1 Day event at the Gaithersburg campus of AstraZeneca on July 18, 2019. The goal of the meeting is for NF1 pediatric patients and caregivers to share their stories about living with NF: What was it like to be diagnosed? Challenges? Are you involved in the NF1 community? How?
Patients and caregivers will have the opportunity to meet with AstraZeneca leaders from the medical, marketing, and corporate affairs teams. During the visit patients and caregivers will actively participate on a NF1 Patient Advisory Board, tour the lab lab, and engage in other patient-centered activities. In addition to our ongoing collaboration with AZ, Children’s Tumor Foundation helped facilitate the NF1 day by recruiting patients to attend the meeting.
AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US was granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1) by the FDA. The potential benefit of selumetinib in NF1 is being explored in the US National Cancer Institute-sponsored Phase I/II SPRINT trial in paediatric patients with symptomatic NF1-related PNs. Phase II trial results are expected later in 2018. The NF1 Day will help AstraZeneca further understand NF and how selumetinib can benefit patients with NF1.
Live on the scene, Renie Moss, one of the patient advocates attending from CTF, posted from a social gathering the night before the meeting:
