Promising new treatment for childhood liver cancer

- Research

Hepatoblastoma is the most common type of liver cancer in children, primarily affecting those under the age of 3. Its incidence has increased substantially in recent years, though it is still considered a rare disease, which poses challenges for research. Patients are currently treated with chemotherapy and surgery, both of which have significant side effects and cause a severe impact on their quality of life, as well as frequent relapses. For these reasons, there is a pressing need for new treatment strategies.

A preclinical study with support from the Spanish Association Against Cancer (AECC) has identified a potential treatment approach for patients with hepatoblastoma, which involves combining standard chemotherapy with a drug currently undergoing clinical trials. The research was conducted using patient samples and animal and laboratory models, and has been led by Dr Josep M. Llovet, Professor of Medicine at the University of Barcelona and group leader at IDIBAPS-Hospital Clinic in Barcelona, and Dr Carolina Armengol, leader of the Childhood liver oncology group (c-LOG) at the Germans Trias i Pujol Research Institute (IGTP) and member of the CIBER of Hepatic and Digestive Diseases (CIBEREHD).

The researchers confirmed that over 70% of hepatoblastoma patients had an alteration that caused them to produce higher amounts of a protein called IGF2, which is associated with fetal growth and development. This makes the IGF2 protein a promising target for treatments that can inhibit it, such as the drug xentuzumab, which is currently in clinical trials for various types of cancer, including several liver cancers.

In this study, researchers observed that combining standard chemotherapy with the drug xentuzumab significantly increases the treatment's effectiveness and allows for a reduction in the administered chemotherapy dosage in animal and laboratory models. Although more research is needed to translate these findings to patients, this study represents a major advancement towards developing improved treatments for hepatoblastoma.

The Spanish Association Against Cancer continues to give support to Dr Llovet and Dr Armengol in their efforts to increase survival rates and improve the quality of life for children affected by this form of liver cancer.


Abril-Fornaguera J, Torrens L, Andreu-Oller C, Carrillo-Reixach J, Rialdi A, Balaseviciute U, Pinyol R, Montironi C, Haber PK, Del Río-Álvarez Á, Domingo-Sàbat M, Royo L, Akers NK, Willoughby CE, Peix J, Torres-Martin M, Puigvehi M, Cairo S, Childs M, Maibach R, Alaggio R, Czauderna P, Morland B, Losic B, Mazzaferro V, Guccione E, Sia D, Armengol C, Llovet JM. Identification of IGF2 as Genomic Driver and Actionable Therapeutic Target in Hepatoblastoma. Mol Cancer Ther. 2023 Apr 3;22(4):485-498. DOI: 10.1158/1535-7163.MCT-22-0335. PMID: 36780225; PMCID: PMC10073300.