The Future of NF Research


NF, Children's Tumor Foundation,

Photo © Courtesy of Children’s Tumor Foundation

The Children’s Tumor Foundation’s strategic plan will help patients and save lives.

In April 2020, the FDA approved the first-ever drug therapy for NF, a MEK inhibitor that targets inoperable plexiform neurofibromas. Over 70 percent of those patients have seen a 20–60 percent reduction in the size of their plexiforms. In addition to both visible and actual tumor reduction, patients report higher-quality physical function, reduced pain, improved mobility, and enhanced emotional and psychological status. Koselugo (selumetinib) is now an approved treatment for NF patients in dozens of countries worldwide.

That first approval has heightened interest in NF. The Children’s Tumor Foundation (CTF) is at the forefront, tripling the number of clinical trials for NF over the past decade, establishing collaborative dream teams of the best scientists working on everything from basic discovery to gene therapy, and attracting nearly a dozen pharma/biotech companies to the NF space. All these treatment options and collaborations are the reward of more than 45 years of CTF-funded research. One of the major focuses now is the establishment of platform trials, which will accelerate the testing of treatments and ultimately get us to effective treatments more quickly and at a lower cost. We’re also funding biomarker studies to improve the predictability of whether a tumor will turn malignant.

Historically, CTF has laid a firm foundation for the critical work ahead by building NF awareness, education, and community. CTF envisions a world without NF, and our strategic plan for the next five years aims to make this vision a reality by driving research, expanding knowledge, and advancing care for the NF community. This has complemented a robust emphasis on NF research funding and the building of essential networks. As we move forward, our unwavering focus is to lead and accelerate the innovation that ends NF.

Partner with the Children’s Tumor Foundation to bring this strategic plan to life and help the patients waiting for a cure. If you are a pharma or biotech company, or an investor in bold, innovative ideas that will change the world, contact us at

Making NF Visible

Life with NF (neurofibromatosis and all types of schwannomatosis) can mean contending with things like brain tumors, café au lait spots, neurofibromas, learning difficulties, loss of hearing, excruciating pain, successful surgeries, failed clinical trials, physical scars, emotional wounds, and tremendous anxiety, never knowing what’s around the corner. We are committed to making NF visible to bring to light the visible and invisible ways NF makes itself palpable in a person’s life and why it’s critical to invest in research that will make a difference.

Since NF affects each patient differently, it can be hard to succinctly understand the disease. Sarah Rodbell and CJ Hines both live with neurofibromatosis type 1 (NF1) but are impacted in profoundly different ways. Sarah was diagnosed when she was 10 years old after her mom found a bump while brushing her hair. Later, after a biopsy, they discovered it was a cancerous tumor. Sarah has been cancer-free following two surgeries in 2009, though she still suffers from headaches, itchiness, and a general discomfort in her skin.

What is NF? NF is a group of genetic conditions that cause tumors to grow on nerves throughout the body. One in every 2,000 people is born with some type of neurofibromatosis or schwannomatosis, which may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, or cancer. Roughly half of all cases arise in families with no history of the condition. NF affects all populations regardless of race, ethnicity, or gender. There are many types of NF, including neurofibromatosis type 1 (NF1) and all forms of schwannomatosis (SWN), including NF2-related schwannomatosis (NF2), formerly called neurofibromatosis type 2.

the future of nf research
CJ Hines and Sarah Rodbell © Courtesy of Children's Tumor Foundation
Nissa Novas © Courtesy of Children's Tumor Foundation

But Sarah wants to be seen as more than her NF, “I’m an artist. I’m a teacher. I’m a daughter. I am a college graduate. I have done so much more than what this disease is.”

CJ was six months old when he was diagnosed with NF1. He’s had more consults, surgeries, and MRIs than he can count. Three years ago, CJ began using a wheelchair, and one of the hardest transitions was accepting the loss of freedom he once had.

Unlike Sarah, CJ is visibly affected, though when asked, he thinks he might have it easier, “If you don’t look like you have a disability, but you really do, people might make assumptions that you’re just wanting stuff just to want it or you don’t really need it. Honestly, I feel like it might be harder for people whose tumors aren’t visible to get the help they need, because for me, people can see my wheelchair, they can see the tumors. So people might say, ‘Oh, do you need help?’ more, or I might get more accommodations. I think it might be easier compared to other people where you can’t see their tumors.”

Nissa Novas has had a very different—and difficult—journey. She was diagnosed with NF2-related schwannomatosis (NF2) when she was 22 years old; two weeks later, she was accepted into medical school. Surgery to remove the meningioma pushing up against her brain was successful, and six weeks later, she started med school to become an emergency room physician. Nissa completed her residency and practiced medicine for 10 years before she exhausted all possible accommodations and adjustments available.

In addition to complete deafness, Nissa developed bilateral vocal cord paralysis, which prevents her from speaking or breathing properly, necessitating a tracheostomy tube in 2013. She experiences chronic pain and exhaustion, vertigo, tinnitus, severe headaches, complete loss of balance, inability to swallow, complete right-sided facial nerve paralysis, muscle weakness, cataracts, and severe dry eye, among other symptoms. She had a large amount of tumor removed from her abdomen and pelvis along with a radical hysterectomy and bowel resection in 2015, destroying gut function and making daily life even more difficult. In 2019 Nissa had another brain surgery for a meningioma removal and the placement of a VP shunt to help with the pressure buildup and severe headaches. She also had placement of a feeding tube shortly after her weight plummeted.

But Nissa is a fighter, and the notion of making NF visible, spreading awareness, and expanding education drives her in a way she hasn’t felt since she was a physician, “It rekindles my sense of purpose, gives me a voice, and a way to still save lives.”

The Children’s Tumor Foundation is committed to investing in research, education, and awareness for Sarah, CJ, Nissa, and the millions of people living with NF.

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Isabelle Moulsdale and Shaelyn Coverdale © Courtesy of Children's Tumor Foundation

Children’s Tumor Foundation Research Innovation Pillars

  • Develop More Drugs Expand the research field, develop panels of drug selection tools, double the number of R&D grants, and launch revolutionary projects.
  • Accelerate Treatments Expedite the discovery of life-changing therapies with an innovative preclinical hub-and-spoke model, the ultimate all-in-one solution for pharma and biotech pioneers.
  • Strengthen Clinical Trials Accelerate the development and approval of drugs being evaluated in clinical trial consortia and/or platform trials.
  • Empower Stakeholders Enhance the understanding of NF and the NF Registry, increase patient participation, and train ambassadors. Expand the NF Clinic Network and the number of physicians and specialists who treat NF, including adult care.

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The Children’s Tumor Foundation is revolutionizing the field of NF research and we’re proud of our recent achievements.


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