Granting Hope for NF Patients: Children’s Tumor Foundation

From Belgium to France to the United States—and ultimately to the Children’s Tumor Foundation (CTF)—CEO Annette Bakker, PhD, has traveled the world studying how cells function and communicate. Her background and research in biochemistry would later prove invaluable in the fight against rare diseases.

A personal experience fueled her mission: a close friend’s battle with cancer led Annette to shift her focus to drug discovery, where she spent two decades in the pharmaceutical and biotech industries. But as she advanced in her career, she grew frustrated with the disconnect between scientific research and the speed at which treatments reached patients.

Bridging the Gap Between Academia and Industry

“Industry partners and academic partners have such opposite agendas,” Annette explains. In academia, scientists need to publish their discoveries to gain recognition and funding. But in the pharmaceutical industry, data is often kept confidential until later stages of clinical trials. This gap slows everything down.”

When Annette discovered the Children’s Tumor Foundation, she saw the potential for nonprofits to serve as the missing link—bridging the worlds of research and drug development to accelerate treatments for patients.

“This is a neutral space,” she says. “At CTF, we aren’t competing for patents or publications. Our only client is the patient, and patients don’t have time to wait. As a philanthropically funded organization, we invest strategically to speed up NF research and bring new treatments to those who need them—together.”

Understanding NF

The Children’s Tumor Foundation serves individuals affected by NF, which includes all types of neurofibromatosis and schwannomatosis—genetic conditions that cause tumors to grow on nerves throughout the body. Depending on where they develop, these tumors can lead to severe challenges, including learning disabilities, chronic pain, hearing and vision loss, disfigurement, or cancer. 

“One of the biggest challenges with NF, like many rare diseases, is that it affects multiple systems in the body, and how symptoms evolve over time is unknown. They can get worse.” Annette explains. “To drive drug development, we need to clear a path for treatments targeting the full range of NF symptoms.”

And that’s exactly what CTF is doing. “We don’t wait. We don’t stand on the sidelines. We’ve built a model that accelerates research, removes roadblocks, and gets treatments to patients faster. By identifying existing drugs with untapped potential, we’re reshaping what’s possible for NF—and for rare diseases as a whole.”

NF isn’t just one disease—it’s a roadmap to solving many. “Because NF affects multiple biological pathways and different organ systems, NF research has implications far beyond itself,” Annette explains. “From cancer to neurology, immunology to orthopedics, ENT to dermatology—the potential impact of NF research is enormous.”

Getting Drugs to Patients—Faster

CTF works tirelessly to accelerate treatment development through a range of initiatives:

• Discovery Fund – This program provides seed funding for early-stage, high-potential ideas, helping researchers build the evidence needed to compete for larger grants from organizations like the National Institutes of Health.
• Partnerships – CTF works closely with the other NF funders, pharmaceutical companies, academic labs, regulators and patients to make drug development more efficient. “How fast can we move a promising treatment forward? Just as importantly, how fast can we rule out what isn’t working and redirect resources?”
• Clinical Trials & Drug Repurposing – CTF collaborates with the Clinical Trials Consortium funded by the Department of Defense to present promising NF treatments to potential industry partners and investors. CTF has also funded platform-basket trials to accelerate clinical evaluation of promising treatments.

“We tell pharmaceutical companies, ‘Bring us your drug. We will move it forward—faster than you ever thought possible,’” Annette says.

A Personal Connection to the Cause

CTF’s mission is personal to Annette. Her own son has a rare disease, giving her deep empathy for NF families.

“When you meet parents in the NF community and see what they go through, I so recognize their urgency because I have felt it myself,” she says. “This is a close-knit group of people facing extraordinary challenges, and I’m honored to be part of their lives, and I pledge to do what I can to help this amazing community.”

She’s also continually inspired by NF patients themselves. “They are some of the most resilient and courageous people I’ve ever met.”

Recent Progress and Future Hope

Annette and the team at CTF are dedicated to amplifying the voices of the NF community and bringing more people into the fight.

After years of investment in research, she believes real breakthroughs are on the horizon.

“We are at a turning point,” she says. “The science is being translated into treatments that will help patients.”

CTF recently celebrated a major victory: in February 2025, a drug that had once been shelved by a pharmaceutical company was successfully approved—proof that persistence and strategic investment can change lives. And there’s more in the pipeline.

“We have over sixty clinical trials running in the U.S. right now and more than twenty pharmaceutical and biotech companies working on potential NF treatments,” Annette says.

Through its newly launched fund with the Community Foundation, CTF is poised to accelerate this momentum. “It raises awareness. It raises funds. It puts NF on the map.”

Annette’s message to potential supporters is simple: this fight needs you.

“Everything we do at CTF comes down to one goal: getting treatments to patients faster. And none of it happens without the generosity of people like you. Together, we can make a difference.”

To support Children’s Tumor Foundation and the incredible work they do, please visit https://cfneg.fcsuite.com/erp/donate/create/fund?funit_id=5160