C H O R O ID P L E X U S C A R C I N O M A ( C P C ) 2 0 2 5
PNOC033: First-Ever Trial for Newly Diagnosed Choroid Plexus Carcinoma (CPC)
The Pediatric Neuro-Oncology Consortium (PNOC): Advancing Childhood Brain Cancer Research
Founded in 2013, PNOC was built on a simple but urgent mission: to accelerate the development of better treatments for children with brain tumors by translating the latest findings in brain tumor biology into clinical care. Over the past 12 years, PNOC has grown into a leading force in childhood brain cancer research, designing and running innovative, patient-centered clinical trials that push the boundaries of what’s possible.
Unlike traditional clinical trial groups, PNOC is a global research network that unites top scientists, clinicians, and institutions to deliver cutting-edge therapies faster and more efficiently. Our trials adapt in real time, allowing us to continuously improve treatment strategies without waiting years for results. We believe that children with brain tumors deserve better, now
From Vision to Action: How PNOC Develops Clinical Trials
Achieving meaningful progress for children with brain cancer demands more than good ideas. It takes a rigorous, collaborative, and fast-moving approach to clinical trial development.
PNOC trials begin with cross-disciplinary working groups of scientists, physicians, and advocates who evaluate emerging research for clinical potential. Promising concepts move through protocol development, peer review, and scientific refinement. Each trial is meticulously structured, incorporating feedback from top experts across our global network. Final protocols can run hundreds of pages and undergo multiple layers of regulatory, ethical, and logistical review before a single patient is enrolled.
PNOC FOUNDATION
PNOC033: First-Ever Trial for Newly Diagnosed Choroid Plexus Carcinoma (CPC)
Choroid Plexus Carcinoma (CPC) is a rare and aggressive pediatric brain tumor, making up less than 1% of all childhood brain cancers. Often associated with somatic and germline P53 mutations, CPC carries a high risk of relapse and secondary cancers and currently lacks any standard treatment protocol leading to inconsistent care across institutions.
PNOC033 is the first clinical trial ever developed specifically for newly diagnosed CPC. Patients will be treated specifically with one of two strategies based upon TP53 mutations, using high-dose chemotherapy followed by autologous stem cell transplants. The chemotherapy regimens were developed through extensive discussion and global collaboration, with careful consideration for radiation exposure particularly in patients with TP53 mutations, where radiation avoidance is the goal. The trial also adds a maintenance therapy phase, using an oral medication following the intensive initial treatment, marking a novel approach to CPC treatment. Important correlative studies include the use of blood and CSF cell-free DNA, comprehensive tumor DNA and RNA sequencing, and the use of patient derived tumors to develop cell lines and explants for preclinical research, supporting future trials and those in the relapsed setting. As with all PNOC clinical trials, data sharing will be emphasized as we recognize that results from this trial will be critical to inform other investigators across the globe of our results.
This trial represents a critical step toward providing families with clarity, consistency, and hope in the face of this devastating diagnosis.
PNOC033: First-Ever Trial for Newly Diagnosed Choroid Plexus Carcinoma (CPC)
Principal Investigator: Wafik Zaky. Study team:
Canada: Lucie Lafay-Cousin (Alberta, Canada)
United Kingdom: Jenny Adamski (Birmingham, UK)
Europe: Denise Obrecht, (Hamburg, Germany)
Global Coordinating Center: Pediatric Neuro-Oncology Consortium
United States: Wafik Zaky (MD Anderson), Karen Wright (Dana Farber), Diana Soraya Osorio, (MD Anderson)
Australia: Jordan Hansford (Adelaide, Australia)
Trial Design and Timeline
Type: Global phase 2 trial
Focus: Newly diagnosed CPC, stratified by TP53 alteration
Enrollment Goal: 44 patients over 5 years
Estimated Cost: ~$30,000 USD per patient
Projected Launch: Q1 2026
Our funding goal for this clinical trial is $1.32 million.
Given the rarity of CPC, international enrollment will be essential. This trial is designed to serve as the foundation for CPC treatment protocols around the world.
hope Patient derived tumor models from the up-front PNOC033 trial will support this research Global investigators from Europe, UK, Australia, Canada and the United States will be involved with this translational effort.
Estimated costs for preclinical studies: $50,000 to $150,000/year
Supporters
This important work is made possible through the generosity of SOCCER FOR HOPE and from the family of a CPC patient in Singapore, whose dedication is driving progress for children with CPC around the world.
Join Us
PNOC033 marks a turning point for CPC research. It’s the first-time children with this diagnosis will have access to a trial built just for them, based on global expertise and focused on long-term survival and quality of life.
Your support helps us open the trial on time, enroll every eligible child, and accelerate progress both in newly diagnosed and relapsed disease. Together, we can rewrite the future for children with CPC.
