A new international Phase I clinical trial in childhood cancer research involving Dr. Lucas Moreno and Dr. Constantino Sábado, from the Translational Research in Childhood and Adolescence group at Vall d'Hebron Research, has concluded that the drug Ceritinib, already effective in adults, could be effective in children. The drug has been tested in patients between 12 months and 18 years with malignant tumors (anaplastic large cell lymphoma, neuroblastoma, rhabdomyosarcoma or inflammatory myofibroblastic tumor) with alterations in the anaplastic lymphoma kinase (ALK). The results of this study are published in the journal Lancet Oncology.

The study was in collaboration with 23 hospitals from 10 European and American countries. Children and adolescents participated in the study, in which the dose of the drug used in adults was adapted according to the characteristics of the child patient (age, weight, height, etc.).

According to the results of the study, which analyzed the side effects and the optimal treatment dose of the drug Ceritinib, it has been shown to be safe if administered at a dose of 500mg/m2. In addition, the first data provided by this study suggest that this drug is also effective in reducing and targeting tumors with ALK gene alterations.

This trial is a paradigm of personalized medicine. In tumors of children with ALK gene alterations, this drug was named as effective: more than 70% of children with anaplastic lymphoma or myofibroblastic tumor responded. In neuroblastoma, the patients who responded maintained the good results for more than a year.

For Dr. Moreno, head of the Pediatric Oncology and Hermatology Department at Vall d'Hebron Hospital and head of the Translational Research Group in Childhood and Adolescent Cancer at Vall d'Hebron Research, "this trial is a great advance in the development of new drugs for childhood cancer and in the incorporation of personalized medicine for these patients. Although ALK gene alterations occur in children, as they do in tumors in adult patients, there is no drug approved in pediatrics for these patients without other therapeutic alternatives. This study paves the way to improve access to new drugs that will be more effective and less toxic in the near future."

"We pediatric oncologists are fighting to shorten the time it takes for new therapies to reach children with cancer. This study is a small victory in this fight. I am very proud of the prominent place that Vall d'Hebron Hospital occupies in the development of clinical trials with new drugs for children with rare diseases", explains Dr. Sábado, associate physician of the Pediatric Oncology Service of Vall d'Hebron Hospital and researcher of the Translational Research Group in Childhood and Adolescent Cancer of Vall d'Hebron Research.

The Translational Research Group in Childhood and Adolescent Cancer has three laboratories dedicated to research on biomarkers and new drugs in minority anemias, sarcomas and neuroblastomas and brain tumors, together with lines of research in hematopoietic progenitor transplantation, a precision medicine program in childhood cancer and a phase 1 clinical trials unit. A total of 52 publications related to cancer in children and young people were made during 2020 alone and they currently have 16 ongoing projects. One of them is the PENCIL-SESHOP project, recently awarded by the Instituto de Salud Carlos III and led by Dr. Moreno, which will develop the national strategy for Personalized Medicine for pediatric cancer. This project will receive a total of one million euros, money that Dr. Moreno and his team will use to improve the lives of patients and their families.