Dr. Jaume Mora receives the IV FERO Dr. Baselga Grant to promote new therapies against pediatric cancer

Dr. Jaume Mora, scientific director of the Pediatric Cancer Center Barcelona at SJD Barcelona Children’s Hospital and head of the "Sarcomas and Neuroblastoma" research group at the Institut de Recerca Sant Joan de Déu, has been awarded the IV FERO Dr. Baselga Grant, endowed with €300,000, for the project "Exploiting Cell State Vulnerabilities to Target Pediatric Cancers." Dr. Mora had already received the FERO Dr. Baselga Grant in 2009 to investigate Ewing sarcoma. This grant, awarded by the Fundación FERO, recognizes high-impact translational research projects in oncology.

The project, lasting three years, focuses on identifying and exploiting the molecular vulnerabilities of different cellular states in pediatric tumors, with the goal of designing new therapeutic strategies that are more specific and less aggressive for children with cancer.

"Pediatric cancer is the leading cause of disease-related mortality in children in developed countries. Unlike adult tumors, cancers in children stem from failures in embryonic development pathways and show few mutations, which makes it difficult to apply targeted therapies," explains Dr. Mora.

The project will initially focus on rhabdomyosarcoma (RMS), the most common soft tissue sarcoma in children and adolescents, which originates from a block in muscle differentiation. Dr. Mora's team will investigate the role of the PRKG1 protein as a possible biomarker capable of determining the tumor's 'cell state,' and will explore the use of an AKT inhibitor drug to restore cellular differentiation and slow tumor growth.
In fact, at this moment Dr. Estela Prada, researcher in the Sarcomas and Neuroblastoma group at IRSJD led by Dr. Jaume Mora, has published as first author the study "PRKG1 hinders myogenic differentiation and predicts response to AKT inhibitor Ipatasertib in Rhabdomyosarcoma" in the scientific journal Nature Communications, which demonstrates for the first time the efficacy of the AKT inhibitor drug in preclinical models developed from patient samples with rhabdomyosarcoma at Hospital Sant Joan de Déu (HSJD). However, many challenges remain ahead, including evaluating this same drug in a clinical trial to determine its real effectiveness in pediatric patients. Now, thanks to the funding received from the IV FERO Dr. Baselga Grant, as well as the collaboration of the pharmaceutical company responsible for the AKT inhibitor drug and the international network Beat Childhood Cancer, this second phase of the project will move forward with the aim of laying the foundations for the first PRKG1-guided clinical trial in rhabdomyosarcoma.

"By integrating molecular profiling, functional pharmacology, and patient platforms, we aim to redefine RMS therapy and establish a replicable model for other pediatric cancers," concludes Dr. Mora.

About the FERO Dr. Baselga Grant

The FERO Dr. Baselga Grant, created in tribute to Dr. Josep Baselga, recognizes projects that drive translational cancer research and promote the link between basic and clinical research. In this fourth edition, the grant continues Dr. Baselga's legacy by supporting projects that help improve prognosis and quality of life for cancer patients.