CPRIT awards $6 million to find new biomarkers and therapies for childhood liver cancers

Texas Children’s Hospital and Baylor College of Medicine researchers received a $6 million research grant from the Cancer Prevention and Research Institute of Texas to find new predictive biomarkers and therapies for high-risk pediatric liver cancers. This grant addresses an urgent need to find safe, targeted and effective treatments that will improve outcomes for children with high-risk liver cancers.

Dr. Dolores Lopez-Terrada, director of Molecular Oncology and Cancer Cytogenetics at Texas Children’s Hospital, is leading this multi-investigator grant, which consists of three complementary projects.

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The first project proposes to validate previously identified prognostic and therapeutic biomarkers for high-risk liver cancers. It will be headed by Dr. Pavel Sumazin, director of the Bioinformatics Core at Texas Children’s Cancer and Hematology Center  and Dr. Donald W. Parsons, director of the Pediatric Center for Personal Cancer Genomics and Therapeutics, co-director of the Brain Tumor Program and Cancer Genetics & Genomics Program at Texas Children’s Cancer and Hematology Center.

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The second project, led by Drs. Sanjeev Vasudevan, director of the Surgical Oncology Program at Texas Children’s Hospital and Dimiter Bissig, associate professor at Center for Cell and Gene Therapy in Texas Children’s Cancer and Hematology Center, aims to reactivate p53 tumor suppressor signaling pathway in mouse models of pediatric liver cancers with the ultimate goal of developing targeted therapies to counter chemoresistance and tumor cell dissemination in children with high-risk liver tumors.

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The aim of the third project led by Dr. Andras Heczey, director of the Liver Tumor program at Texas Children’s Cancer and Hematology Center, is to develop targeted immunotherapy for high-risk liver tumors by examining the safety and efficacy of genetically engineered Glypican-3-specific chimeric antigen receptors (CAR)-T-cells.

Hepatoblastoma (HB) and hepatocellular carcinoma (HCC) are the most frequently diagnosed liver tumors in children. The HBs are most commonly present in young children younger than 5 years of age, whereas HCCs are more commonly seen in adolescents. Children with high-risk liver cancers, defined as those tumors that are surgically-intractable or malignant, do not respond to even high doses of chemotherapy.

Current treatments for HBs and HCCs are often associated with low rates of survival and significant toxic short- and long-term effects on heart, kidney and brain function, as well as other secondary malignancies. As of now, complete surgical removal of the tumor is the only treatment option available and offers little benefit to patients with metastatic forms of the disease.

By bringing together investigators with diverse expertise in cancer biology, this multi-investigator CPRIT grant employs a comprehensive and systematic approach to uncover novel biomarkers and therapies that could have direct clinical benefits.

The Cancer Prevention & Research Institute of Texas (CPRIT) launched in 2009 following a constitutional amendment to commit $3 billion over 10 years to fight against cancer. It is charged by the Texas Legislature to create and expedite innovative therapies in the area of cancer research; attract, create or expand research capabilities by helping to recruit outstanding cancer scientists to academic institutions across Texas; and to coordinate and implement policies and programs related to cancer prevention, detection and treatment.

The generous support from CPRIT provides an outstanding opportunity for the researchers at the Liver Tumor Program at Texas Children’s Cancer Center to identify new treatments that will improve outcomes for children with high-risk liver tumors.