The Children's Tumor Foundation
11/04/2026 | Press release | Distributed by Public on 11/04/2026 10:15
CTF Invests in Research to Advance Clinical Trials for NF1-Associated Scoliosis
A Clinical Research Award will support a multicenter study led by Jonathan J. Rios, PhD, to determine which children are most at risk for scoliosis progression and how future treatments should be evaluated.
The Children's Tumor Foundation (CTF) has awarded a $200,000 Clinical Research Award to Jonathan J. Rios, PhD, director of Molecular Genetics at Scottish Rite for Children and associate professor at UT Southwestern Medical Center's Eugene McDermott Center for Human Growth and Development, to advance research into scoliosis associated with neurofibromatosis type 1 (NF1).
Scoliosis is among the skeletal complications that can affect children with NF1. Compared with scoliosis in children without NF1, it may develop at a younger age, progress more rapidly and can be more difficult to treat. Bracing may not prevent progression, and children with NF1-associated scoliosis are more likely to require surgery.
Right now, clinicians lack the evidence needed to predict which children are most likely to experience rapid progression or determine how potential treatments should be evaluated in clinical trials.
"CTF is supporting this research because NF1-associated scoliosis should be a treatable problem, not a complication we simply learn to manage," said Annette Bakker, PhD, CEO of the Children's Tumor Foundation. "To get there, we need the evidence to understand who is most at risk, when to intervene, and how to measure whether a treatment is working. We are investing in this study so researchers can move faster toward clinical trials and bring better treatment options to children with NF1."
Building the Evidence Needed for Future Trials
With medications now approved for NF1-related plexiform neurofibromas, researchers can begin exploring whether medical treatments could also affect skeletal complications of NF1. Before that possibility can be tested, they need a stronger understanding of how NF1-associated scoliosis develops and progresses over time.
A major barrier is the lack of natural-history data needed to identify who should be included in future trials, define meaningful measures of success and design trials capable of producing reliable results.
"Before we can test promising new therapies, we need to know which children are most likely to benefit and how we should define treatment success," Rios said. "This could mean slowing the progression of scoliosis, delaying surgery or preventing the need for surgery altogether. This study is an important step toward answering those questions."
Rios will lead a multi-institutional research team that includes investigators from Children's Medical Center Dallas, Cincinnati Children's Hospital Medical Center, Mayo Clinic and UT Southwestern Medical Center. During the two-year award period, the team will conduct a retrospective longitudinal study using patient data from two high-volume NF orthopedic centers to:
Identify which children with NF1-associated scoliosis are most in need of intervention
Determine the most appropriate endpoints for future clinical trials
Conduct analyses needed to assess the feasibility and design of future interventional trials
"At Scottish Rite, our physicians and scientists have spent decades working to better understand complex pediatric orthopedic conditions and improve treatment for children," said Robert L. Walker, president and CEO of Scottish Rite for Children. "This research brings together Scottish Rite's clinical expertise, scientific capabilities and collaborative partnerships to address important unanswered questions in the care of children with NF1-associated scoliosis. The knowledge gained through this study will move the field closer to clinical trials and, ultimately, better treatment options for children and families."
Natural history data have previously been used to support clinical trials of mirdametinib and selumetinib in children with NF1-related plexiform neurofibromas. This study aims to generate the evidence needed to support future trials for NF1-associated scoliosis.
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