2024 Clinical Research Award Collaboration

2024 Clinical Research Award Collaboration: Evaluation of a Novel Physiologic Biomarker of Pain in Neurofibromatosis Type 1

The Children's Tumor Foundation is proud to share the continued funding of a Clinical Research Award collaboration between the National Cancer Institute and Children's National Medical Center. This project has the potential to greatly improve how pain is measured and managed for patients with NF1, leading to more effective treatments and better quality of life.

Principal Investigators:
Pamela Wolters, PhD, National Cancer Institute, Center for Cancer Research
Julia Finkel, MD, Children's National Medical Center and Children's Research Institute

Project Summary:
Neurofibromatosis 1 (NF1) is a genetic condition that causes tumors and other problems, often leading to pain and difficulty with daily activities. One common type of tumor, called plexiform neurofibromas (PNs), affects about half of people with NF1 and can cause significant pain. The first FDA-approved drug for treating PNs in children who can't have surgery is selumetinib, but additional trials are exploring its effects on other NF-related tumors, including cancerous ones.

While some treatments help reduce tumor size, they don't make the tumors disappear completely, and they must be taken for long periods. This makes it crucial to measure how much pain a patient is experiencing to see if a treatment is truly helping. Traditional pain questionnaires can be complicated to use with younger children, people with learning challenges, and those experiencing different types of pain, which can be influenced by mood or outside factors like family support.

To address this need, this collaborative grant will support research into a new tool developed at Children's National Hospital called the AlgometRx Nociometer. This device measures pain by assessing three types of nerve fibers and providing a score that helps identify the type of pain a person feels. It also tracks how well treatments work over time. Our study will focus on using this device with patients who have NF1, especially young children, to see if it can measure different types of pain and give consistent results. We also hope to expand its use to people with schwannomatosis, including NF2-related schwannomatosis, who also experience pain.

By funding this innovative research, the Children's Tumor Foundation aims to fill a gap in NF care by offering a simple, accurate way to measure and monitor pain, helping doctors improve pain management and future treatments.

The Clinical Research Award program supports early-stage pilot clinical trials of candidate therapeutics or interventions for the treatment of physical or psychosocial manifestations of NF1, SWN, or NF2-SWN. This grant mechanism also includes adjunct studies, such as developing biomarkers, imaging protocols, and other clinical trial tools.

Learn more at ctf.org/funding-opportunities

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