Unlocking New Hope: PHASE ONE Funds Phase I/II Trial Investigating a Novel Approach to Pediatric Cancer Treatment for Solid Tumors
PHASE ONE Foundation has awarded Dr. Allison F. O’Neill at Dana-Farber Cancer Institute $370,000 to fund a Phase I/II trial studying a novel therapeutic approach to jump-start the immune system against solid tumors in pediatric patients.
Approximately 1 in 285 children in the U.S. will be diagnosed with cancer before their 20th birthday.
Most of these cancers originate from solid organs or soft tissues, primarily the brain or extracranial sites.
While advancements in therapies have improved survival rates, treating advanced cases, especially those that have spread, remains challenging and treatment is often associated with long-term toxicities.
Immunotherapy, a preferred alternative to standard chemotherapy, has shown promise in adults but has not had much success in children with relapsed or refractory solid tumors.
The development of immune therapies for pediatric patients has been limited due to the lack of evidence to support anti-tumor immunity in young patients despite the fact that pediatric tumors harbor dormant immune cells.
To address this critical knowledge gap, Dr. O’Neill is investigating a potential breakthrough treatment involving a new antibody called 2141-V11. This antibody aims to activate immune cells within pediatric solid tumors and enhance the body's natural defenses against cancer.
The antibody targets a receptor called CD40, found on various immune cells, that when activated can activate the immune cells to better recognize cancer cells directly and stimulate T cell responses— both of which are crucial for fighting cancer.
The primary goal of this study is to evaluate the safety and effectiveness of delivering this antibody directly into the tumor (intratumoral injection) for pediatric patients with solid tumors.
This innovative approach holds promise due to its potential to activate immune cells within the tumor microenvironment, which might overcome the challenges posed by low tumor immunogenicity (ability to provoke an immune response) commonly observed in pediatric cancers — potentially revolutionizing our understanding of treating solid tumors in children.
The direct delivery of this antibody into the tumor compartment will provide an unparalleled platform for tissue collection supporting translational, longitudinal research studies— allowing researchers to investigate and potentially identify biomarkers associated with effective anti-tumor immunity in children.
“We are grateful to the PHASE ONE Foundation for providing us invaluable support to launch this clinical trial and we remain eager to gain precious insights to the pediatric solid tumor immune landscape.” – Allison O’Neill, MD
To achieve these aims, Dr. O’Neill will conduct a two-stage clinical trial involving pediatric patients with different types of solid tumors. The trial will focus on determining the optimal dosage, evaluating safety, and assessing the antibody’s potential to improve tumor control and patient outcomes.
“This research represents a crucial step towards better understanding and harnessing the immune system's potential to fight pediatric solid tumors, potentially leading to improved treatments and outcomes for young patients facing these challenging diseases.” –Amanda Salvado, MD, PHASE ONE Granting Committee Co-Chair
The results from this study are poised to fill a critical gap in what we know about how the immune system fights cancer in children, and if successful, could pave the way for a new era of targeted therapies that harness the power of the immune system, offering renewed hope for young patients and their families.