Lilly has always dreamed big. Growing up in New York, she wanted to be everything—a journalist, a lawyer, even an American Gladiator. But her life has been shaped by an unexpected reality: neurofibromatosis type 1 (NF1) and cutaneous neurofibromas (cNFs) that came with it. These tumors, which appear on the skin, aren’t just physically painful; they can carry a stigma that can be hard to ignore. For many years, Lilly put parts of her life on hold because of her tumors, from education to career to the simple joy of walking through life with confidence.
Despite these challenges, Lilly has found ways to share her story, as she shares in the video below. When a stranger posted a video about her on TikTok, she fired back with a response about her life with NF1 that struck a chord, going viral and giving her a platform to raise awareness. But Lilly wanted to do more than just share her struggles—she wanted to make real change. That’s what led her to an extraordinary opportunity: speaking at an FDA Listening Session to advocate for better treatments for cNFs.
The FDA Listening Session: Speaking Truth to Power
This past year, Lilly joined five other patients and caregivers at a FDA Listening Session organized by the Children’s Tumor Foundation (CTF), an event designed to give patients a direct voice in the development of treatments. Together, they represented the diversity of the NF1 community—different ages, backgrounds, and degrees of disease severity—but their message was unified: cNFs are more than just a cosmetic issue.
For Lilly, this wasn’t just another opportunity to share her story—it was a chance to advocate for change. She spoke about how managing her cNFs has shaped her life in profound ways, from time-consuming clinical trials to grueling surgical procedures.
“I chose a life as close to what I wanted as possible,” she told the group. “I wanted to be a journalist, I wanted to be a lawyer, I wanted to travel, I wanted to be an American Gladiator, I wanted to be a mom. One way or another, NF has kept me from doing it.”
But it wasn’t just the physical challenges of NF1 that Lilly described—it was the emotional weight. “Even the times I was lucky enough to do what I wanted to do, people seeing my tumors and being disgusted made it nearly impossible to exist in certain spaces.” Her words painted a vivid picture of the isolation and stigma so many in the NF1 community feel.
Hope on the Horizon: The Promise of NFX-179
Lilly’s advocacy comes at a pivotal moment for the NF1 community. For decades, people with cNFs have faced limited treatment options—largely surgical and often inadequate. But that’s starting to change, thanks to new developments like NFX-179 Gel, a topical MEK inhibitor developed by NFlection Therapeutics.
This innovative treatment recently completed a successful Phase 2b clinical trial, supported in part by the Children’s Tumor Foundation. The results were groundbreaking: the gel significantly reduced the size of cNFs in many participants, with the highest dose achieving a 44.2% responder rate. Designed to deliver treatment directly to the tumors while minimizing systemic side effects, NFX-179 Gel could soon be the first FDA-approved therapy for cNFs.
Dr. Miriam Bornhorst, one of the study’s investigators, captured the significance of this development: “Cutaneous neurofibromas, which present in over 95% of people with NF1, are often the most burdensome symptom of the condition. The data shows that NFX-179 Gel significantly reduced the size of cNFs across multiple efficacy endpoints and was generally well tolerated. We are excited to see its potential as the first topical therapy for cNFs.”
A Brighter Future
For Lilly, treatments like NFX-179 represent something far greater than just tumor reduction—they’re a chance to reclaim the life NF has tried to take from her. She closed her testimony at the FDA Listening Session with a heartfelt hope: “I want more options for people with NF. I want us to live the lives we want, not the ones NF makes us settle for.”
Lilly’s journey—from sharing her story on TikTok to standing before the FDA—reminds us of the power of advocacy. It’s a testament to the resilience of the NF1 community and the incredible work being done to bring real, life-changing treatments to those who need them most.
The Children’s Tumor Foundation is working hard to make that hope a reality. Through strategic investments in innovative research and partnerships with companies like NFlection, we’re building a pipeline of treatments that could transform the future for Lilly and the millions of individuals around the world living with NF.
Your ongoing support for the Children’s Tumor Foundation amplies this advocacy, and enables real change. Join us today, your gift today takes us one step closer to treatment options for tomorrow.
Give Now at ctf.org/donate
