Targeted therapy at the heart of hope for children with rare tumors of the central nervous system
Presented on May 31, 2025 at the prestigious World Congress of Cancer of Asco in Chicago, a study carried out by the Institut Curie offers a glimmer of hope for children confronted with cancers of extreme rarity. This is the Imatrix clinical trial, coordinated by Professor François Doz, an oncologist pediatrician, whose results open the way to a new therapeutic strategy thanks to the use of alectinib, a molecule specifically targeting the alterations of the ALK gene.
Less than ten cases per year in France, but a world hope
Certain pediatric malignant tumors of the central nervous system, such as high -grade gliomas or sarcoma forms, as well as inflammatory myofibroblastic tumors, are characterized by mergers at the Alk gene. These pathologies, which affect less than ten children per year in France, are particularly complex to treat and often dark prognosis.
Alectinib, a new generation oral inhibitor targeting alk oncogen, has already been used in pediatric cancers such as large cell anaplastic lymphomas or certain neuroblastomas. But so far, it has never been tested on these brain tumors or as rare sarcomas.
Very encouraging results for young patients
The Imatrix test, phase I/II, aimed to assess both the tolerance, the optimal dose and the effectiveness of alectinib in patients under the age of 18 for whom conventional treatments had failed. Twenty-two children were included in the study. The results presented are remarkable: an overall tumor response rate greater than 87 % on a sample of 16 patients, and stabilization of the disease in two others.
Above all, the treatment has proven to be well tolerated, an essential criterion in such a vulnerable population.
The Curie Institute, through its Siredo Center dedicated to childhood cancers, adolescents and young adults, is one of the European leaders in this area. It was in this center that in 2008, doctors Olivier Delattre and Isabelle Janoueix had highlighted the role of the Alk gene in Neuroblastoma. Since then, research has continued to progress, notably under the leadership of Dr. Gudrun Schleiermacher.
The center today has an arsenal of skills, from basic research to clinical research, including somatic genetics tools, allowing an integrated and rapid approach to therapeutic development.
For Pr François Doz, this advance is the fruit of a unique ecosystem: “The strength of the Curie Institute, and the Siredo Center, is this constant dialogue between fundamental, translational and clinical research. It is this virtuous synergy that makes it possible to innovate and accelerate research for our young patients. »»
In a medical world often focused on the most frequent pathologies, this initiative illustrates the capital importance of research dedicated to rare diseases, and the capacity of French institutions to play a leading role internationally.
Photo credit: DR (Illustration Photo)
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