Advancing Biological Understanding of Adamantinomatous Craniopharyngioma for the Design of Clinical Trials
Understanding and Treating ACP: A New Hope for Children with a Rare and Devastating Brain Tumor
Adamantinomatous Craniopharyngioma (ACP) is a rare but serious brain tumor that primarily affects children, though it can also occur in adults. Despite being non-cancerous (or benign) in terms of spreading to other parts of the body, ACP causes significant and often permanent damage to the brain. It accounts for 6–8% of all brain tumors in children, and due to the delicate brain structures it affects, it leads to some of the most severe long-term health problems seen in any pediatric brain tumor.
ACP tends to form both solid tumor masses and large fluid-filled cysts. These cysts often grow over time, pushing on vital areas of the brain that control vision, hormone balance, appetite, and memory. As a result, children with ACP may face blindness, severe hormone imbalances requiring lifelong medication, obesity due to hypothalamic injury, and cognitive impairments. Even after surgery and radiation therapy, many children continue to suffer from lifelong disabilities. For this reason, finding new ways to treat ACP, especially treatments that can stop or reverse cyst growth, could dramatically improve the lives of these young patients.
Project Goals
Breaking the Cycle of Damage in Childhood Brain Tumors:
Our project is guided by one central goal: to understand what drives ACP to grow and cause harm—especially the formation of fluid-filled cysts—and to develop safer, more effective treatments that preserve brain function and improve the quality of life for children living with this disease.
This leads us to the central therapeutic goal of our project: to test whether combining IL-6 and VEGF blockade can shut down the harmful signals that sustain ACP—and reprogram the tumor’s immune environment from one that helps the tumor to one that helps the patient.
We’ve designed two main research goals to help us achieve this vision:
Goal 1: Understand Why IL-6 Therapy Sometimes Fails
Goal 2: Test Combination Therapies in the Lab
A Vision for the Future
We believe that ACP doesn’t have to be a life-altering diagnosis. If we can stop the tumor from manipulating the immune system—if we can cut off the signals that lead to inflammation, cyst growth, and brain damage—we can preserve what matters most: children’s vision, memory, hormones, and quality of life.
Our project combines cutting-edge science with a heartfelt mission to improve the lives of children and families affected by this terrible disease. By deeply understanding how ACP tumors operate—and by attacking the disease from multiple angles—we hope to deliver treatments that not only stop the tumor but also give children their futures back.
