What’s Next for NF1: Webinar Recap

Two approved treatments for plexiform neurofibromas, the first-ever NF1 platform trial launching in Europe, and 50 active CTF-funded research projects across the pipeline: that was the picture of NF1 research shared at the Children's Tumor Foundation's (CTF) recent webinar, "What's Next for NF1: Progress in the Research Pipeline, Clinical Trials and New Treatments."

NF1 is a complex genetic condition that affects people in different ways, and the webinar covered research across that range, from early laboratory discoveries to clinical trials and approved treatments.

The session was hosted by Barbara Gallagher, CTF's Vice President of Individual and Foundation Giving, and featured:

• Annette Bakker, PhD, Chief Executive Officer
• John Puziss, PhD, Chief Scientific and Impact Officer
• Darek Adamczewski, PhD, Managing Director, CTF Europe
• Irene Morgenstern, PhD, Director of Preclinical Initiatives

Watch the Recording

These two resources complement the recording:

• CTF's NF1 portfolio summary, a printable overview of every active CTF-funded NF1 research project as of October 2026.
• A glossary of terms used in the webinar, in the order they come up, written for families.

What We Covered

• The breadth of NF1 research. CTF is funding 50 active NF1 research projects at 32 institutions in six countries. They span tumors and cancer, learning and behavior, pain, vision, bone health, and gene therapy, and half focus on MPNST, the most serious complication of NF1.
• How CTF moves the strongest ideas toward patients. Bringing a new drug to patients can take 10 or more years. CTF shortens that path through its Preclinical Hub, partnerships with drug companies, and clinical trial networks, and by investing directly in companies developing NF1 therapies.
• What is in the clinic now. Koselugo and Gomekli are two globally approved treatments for plexiform neurofibromas. Clinical trials are underway or in development for MPNST, cutaneous neurofibromas, and muscle weakness, alongside clinical research on early cancer detection, pain, vision, and cognition.
• What is coming next. The first-ever NF1 platform trial, launching in Europe, will test multiple therapies in parallel for multiple manifestations of NF1, with new treatments added as they are ready.

Your Questions, Answered

="">What are the most impactful things families affected by NF1 can do to move research forward?

Families affected by NF1 are essential partners in moving research forward. Your support, your lived experience, and your voice all matter. Here are some meaningful ways to get involved:

Families affected by NF1 are essential partners in moving research forward. Your support, your lived experience, and your voice all matter. Here are some meaningful ways to get involved:

• Support the work in whatever way is meaningful to you. Make a direct donation, fundraise through NF Walk, take part in an NF Endurance event, or bring people together for a special event. Every contribution helps CTF advance research and bring treatments to patients.
• Join the NF Registryand keep your information up to date. By sharing information about your symptoms and experiences, you help researchers understand how NF affects people and changes over time. The Registry can also connect you with clinical trials and research studies you may be eligible for.
• Take part in educational programs and share your perspective. Programs like NF Summit let you learn about research advances and connect with experts and other families. Through CTF Engage, patients and caregivers can share their lived experience to help shape research priorities and study design. You don't need to be a scientist to bring valuable expertise: your experience matters.
• Consider joining a clinical trial. Patient participation is essential to testing potential treatments and generating the evidence needed to move them toward approval. Talk with your NF care team about studies that might be right for you or your child. Learning about a study does not commit you to joining it.
• Stay connected and help spread the word. Sign up for CTF's newsletter, follow CTF on social media, and explore ctf.org for research updates and ways to get involved. Sharing CTF's news with your own networks helps build awareness and support.

There is no single right way to contribute, and not every opportunity fits every family. We are grateful for whatever form your partnership takes.

="">What role does the NF Registry play in driving research forward?

The NF Registry connects patients' experiences to scientific progress. Members complete a brief survey about their health and experiences once a year. This information gives researchers a clearer picture of NF, guides new studies and potential treatments, and can connect patients to future clinical trials. Learn more at nfregistry.org.

="">How can families stay connected to opportunities to get involved?

Follow CTF on social media, join the NF Registry, sign up for our newsletter, and visit ctf.org regularly. We share opportunities to take part in research, clinical trials, advocacy, educational programs, and more.

="">How can a family member's tumor and health information be used for research?

Please join the NF Registry. Registry information helps researchers understand the natural history of NF, accelerate drug development, guide clinical trials, and improve patient care. For tissue donation, see our biobank resource page and contact Irene Morgenstern, PhD, CTF's Director of Preclinical Initiatives, at [email protected].

="">Are there advances in treating ADHD behaviors in children with NF1?

The effect of MEK inhibitors on cognition is currently being tested in a clinical trial. There is limited anecdotal evidence that these drugs may improve cognition and behavior. Other studies that may be of interest:

• Guanfacine for ADHD and cognitive challenges in children and adolescents with NF1 - Read more here
• The NF114 study - Read more here
• The Stanford BridgeLab study, now enrolling - Read more here

="">Is research looking at ablation treatments, or only drugs?

Yes. Clinical trials have tested radiofrequency ablation for cutaneous neurofibromas. Read more here.

="">Do large numbers of mast cells disturb sleep cycles in adults with NF1?

There is a biological connection between mast cells and sleep: mast cells release histamine and other inflammatory substances that interact with the pathways that regulate sleep and daily rhythms. Sleep problems are also documented in people with NF1. However, there is not yet direct evidence that increased mast cells contribute to sleep problems in NF1.

Cognitive behavioral therapy for sleep is being studied in people with NF1 by Dr. Natalie Pride and by a team at Massachusetts General Hospital.

="">What research is being done on the link between NF1 and tumor-induced osteomalacia?

Reports of NF1-associated oncogenic hypophosphatemic osteomalacia, also known as tumor-induced osteomalacia, are extremely rare. You can read more about these cases in this case report and this article.

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