Children’s Hospital of Philadelphia announced $1.8 million in combined V Foundation grants to two researchers: $1 million to develop a CAR T-cell therapy for a hard-to-treat pediatric leukemia, and $800,000 to pilot a blood-based early detection test for children with a rare inherited cancer syndrome. The announcement is especially timely, coming during September’s observance of both National Childhood Cancer Awareness Month and Leukemia and Lymphoma Awareness Month.
The Two Grants: A New CAR T-Cell Approach to Fighting T-Cell Leukemia in Children
Andrei Thomas-Tikhonenko, PhD, Chief of the Division of Cancer Pathobiology at CHOP, was awarded the $1 million V Foundation Pediatric All-Star Cancer Research Grant to develop a novel chimeric antigen receptor (CAR) T-cell therapy for children with T-cell acute lymphoblastic leukemia (T-ALL), an aggressive form of blood cancer. His team will investigate P2RX5, a promising target found on leukemia cells, using genetic and biological datasets, laboratory studies, and preclinical models to evaluate the safety and efficacy of P2RX5-targeted CAR T cells. The research could pave the way for a first-in-human clinical trial within the next several years, ultimately providing a more precise treatment option for children with relapsed or treatment-resistant T-ALL. The work may also help address disparities in cancer outcomes, as P2RX5-positive leukemias are more prevalent among children of African and Hispanic ancestry.
Suzanne MacFarland, MD, an attending physician in the Cancer Center at CHOP and Director of its Cancer Predisposition Program, received an $800,000 V Foundation Pediatric Translational Cancer Research Grant to study a blood-based “liquid biopsy” approach for children and young adults with Li-Fraumeni syndrome (LFS), a rare inherited condition associated with a significantly increased risk of developing cancer. Researchers will pilot the use of a blood-based assay to detect tumor DNA in the bloodstream, aiming to detect cancer earlier than current screening methods. The study will also assess how liquid biopsy testing affects patients and caregivers, serving as a crucial first step toward understanding the feasibility of this screening method ahead of a larger national trial.
How CHOP and V Foundation Leadership Are Framing the Awards
Susan L. Furth, MD, PhD, Executive Vice President and Chief Scientific Officer at CHOP, said the awards advance two powerful frontiers in pediatric cancer research, from developing more precise, potentially lifesaving immunotherapies for children with difficult-to-treat leukemia to pioneering blood-based approaches that could detect cancer earlier in those at highest genetic risk. She added that together they bring the field closer to a future where cancer care is more targeted, more equitable, and more effective for every child, and expressed gratitude to the V Foundation for its longstanding partnership.
Kara Coleman, Vice President of Grants and Research at the V Foundation, called the awards a testament to the ingenuity, dedication, and vision of Drs. Thomas-Tikhonenko and MacFarland, whose research is pushing the boundaries of what is possible in pediatric cancer care. She said their work exemplifies the power of scientific discovery to address some of the greatest challenges facing children and families, bringing the field closer to more effective treatments, earlier detection, and better outcomes for future generations.
Funding History and Context
The V Foundation has awarded nearly $6 million in funding to CHOP since 2010, and this year’s announcement marks the largest award CHOP has received in a single year. The All-Star Cancer Research grant is designed to advance transformative, high-risk/high-reward childhood cancer projects and is only awarded to previous V Foundation grant recipients. Dr. Thomas-Tikhonenko has received the V Foundation’s Pediatric Translational Award twice before: in 2011 for “Novel Therapeutic Targets in Pediatric Acute B-Lymphoblastic Leukemia,” and again in 2018 for “Alternative Surface Epitopes in Pediatric Acute Lymphoblastic Leukemia.” These two earlier projects allowed his laboratory to join the Pediatric Immunotherapy Discovery and Development Network (PI-DDN), funded by the National Cancer Institute’s Cancer Moonshot Initiative. The Pediatric Translational Cancer Research grant more broadly supports research that moves promising cancer discoveries from the lab toward clinical trials and improved patient care.
