Like every year for the past 11 years, Imagine for Margo – Children without Cancer has organized its annual FAST (Further Accelerating Specific Treatments) conference in order to bring together all the players in pediatric oncology to accelerate the development of new drugs. The program of conferences and round tables was designed to explore, over the course of a day, all the possibilities for healing children and adolescents more and better. To achieve this objective, we invited, to the « Châteauform George V » (of which we thank the directors, Mrs. and Mr. Baignières), all the stakeholders concerned, namely pediatric oncologists, researchers, managers of pharmaceutical and biotechnology companies, leaders of bioclusters, families, patients and associations. The complementarity of their expertise in pediatric oncology made it possible to take an exhaustive inventory of recent advances bringing hope, promising new therapies and the challenges of tomorrow. All these topics and discussions were orchestrated by Thierry Guerrier, journalist and health expert, who does us the honor of being by our side every year.
OUR OBJECTIVE TO CURE HIGH-RISK CHILDhood CANCERS MORE AND BETTER IS MORE ACHIEVEABLE THAN EVER
At the start of this conference, we highlighted the major research successes in 2023, which were supported by Imagine for Margo. We were delighted to welcome Dr. Daniel Orbach (Institut Curie), who presented his TAG-N-TRAK project for spectacularly curing childhood fibrosarcomas. An innovative treatment, given in the form of a syrup to be taken morning and evening, which specifically targets a molecular abnormality found in these cancers, destroys them at a spectacular speed, has no side effects and is increasingly recognized as a treatment reference in a large number of countries. This project is undoubtedly a victory for precision molecular medicine, just like the BIOMEDE program, which was presented by Dr. Jacques Grill (Gustave Roussy). This project is dedicated to better healing children suffering from an infiltrating brainstem glioma, whose life expectancy is a few months after diagnosis. Thanks to significant financial and scientific resources, Dr. Grill enabled eight children to be the first long-term survivors of this cancer. In addition, the molecular sequencing of their tumor made it possible to understand the reasons for this success and thus open new avenues to cure ever more children suffering from this cancer.
However, innovation is not only on the treatment side. We had the honor of welcoming Dr. Pablo Berlanga (Gustave Roussy) who presented his SACHA project to us, aimed at securing access to innovative medicines in pediatric oncology. SACHA is above all a method of collecting data on the effectiveness and toxicity of these treatments given to children and adolescents who have relapsed or high-risk cancer and who cannot be included in a clinical trial. For 4 years, this project has accumulated successes: it was published for the first time in July 2023, was presented to ASCO, is recognized by the French HAS (High Authority of Health) and supported by the French ANSM (National Agency Safety of Medicines and health products) and is now extended internationally. This project, unique in the world of health, makes it possible to establish therapeutic recommendations for these new drugs for all pediatric oncologists and thus maximize the chances of cure for children, adolescents and young adults suffering from a cancer.
THESE SCIENTIFIC VICTORIES WILL LEAD TO OTHERS
The scientific progress made in recent years gives us hope of quickly making spectacular advances in the cure of high-risk or relapsed pediatric cancers. In order to discuss the issues that must be addressed to achieve this objective, we brought together, for a round table, Professors Gilles Vassal (Gustave Roussy), Professor André Baruchel (Robert Debré hospital), Dr. Marie Castets (Lyon Bérard Cancer Research Center) and Prof. Franck Bourdeaut (Institut Curie). Major topics were raised such as the lack of targeted therapies revealed by the systematic sequencing of high-risk and relapsed pediatric cancers and the growing need for CAR-T cells, which are showing increasingly strong signals of effectiveness against leukemias and solid tumors such as neuroblastoma. However, these advances will only be possible with effective sharing of biological data and optimal structuring between fundamental, translational and clinical research. In an era where technology allows the analysis of a large mass of data, it appears necessary to advance regulations in France and Europe so that the exchange of information and data between all players in pediatric oncology are fluid. Initiatives carried out by many of our speakers have made it possible to demonstrate the particularly fruitful nature of these exchanges, without harming the property of their authors and respect for the privacy of patients.
BETTER STRUCTURING RESEARCH IN FRANCE
We were happy to present, during a round table, the creation of three integrated research centers in France, certified by the National Cancer Institute (represented by Professor Bruno Quesnel): EN-HOPE SMART4CBT (East and North, presented by Dr. Natacha Entz-Werlé), South-ROCK (Lyon & Marseille, presented by Dr. Marie Castets) and Paris Kids Cancer (represented by Prof. André Baruchel). These three integrated research centers have the mission of bringing together fundamental, translational and clinical research in order to perfect our understanding of childhood cancers (origin, tumor microenvironment, anti-tumor immune response) and to open up promising therapeutic avenues. . Interconnectivity within and between these centers will make it possible to implement and pool heavy equipment as well as IT and human resources (bioinformaticians for example). In addition, Imagine for Margo, alongside other associations, is participating in the construction and deployment of the Paris Kids Cancer center in the Paris region.
EXPAND THIS CONNECTION TO the industry
The valley of death, which separates fundamental research from clinicians, is a major obstacle in access to innovative, effective and less toxic therapies for children. Furthermore, all the players involved in these developments make the same observation: we must shorten the times between the establishment of pre-clinical proofs of concept and clinical development. There is a solution to these two issues: break down all barriers and collaborate systemically. These questions were addressed by a panel of experts from the industrial sector (Dr. Iris VALTINGOJER (Sanofi), Dr. Hélène NEGRE (CELLforCURE by Seqens), Dr. Alexis GÉNIN (Brain&Mind), Jean-Claude ROCHE (Recordati Rare Diseases France) and Paul RINAUDO (ADLIN Science).
At the end of 2023, a group dedicated to Pediatric Cancers was founded within the PSCC (Paris Saclay Cancer Cluster). This group, of which our president, Patricia Blanc, is a member, is made up of actors with varied and complementary profiles (industrial, academic, philanthropic associations, biotech, etc.). The objective of this group is to encourage biotech companies, which carry a large part of the development of new therapies, to innovate in pediatric oncology. These bioclusters, like the PSCC or the Brain&Mind, are a strong marker of this move forward, where the way to arrive at new drugs is collectively rethought. Finally, all the manufacturers invited to the FAST conference testified to the urgent need to break down barriers and silos, to accelerate clinical development methods (7-8 years of research are necessary on average), to advance regulations on data sharing and accelerate early access.
MEET THE NEEDS OF FAMILIES
Beyond access to the most appropriate care and treatments for each child’s cancer, Imagine for Margo is also committed to meeting the needs of families and addressing well-being issues throughout the care journey. Many pediatricians and families have noted that chemotherapy treatments, in addition to nausea, modify the perception of taste and no longer make meals a moment of pleasure. In order to respond to this observation, Imagine for Margo has published and distributed free of charge the nutrition guide “Eat? Not even afraid ! “. During this FAST conference, we invited all the people who are at the origin of this work, which brings together useful advice and numerous recipes from great chefs, adapted for sick children. After a video introduction by chef Ryan Callahan, author of the book Cooking for Kids with Cancer, we were delighted to welcome Manal ANAYÉ (Imagine for Margo), Loryane LE CHEVALIER (young cured patient aged 19), Dr Dominique VALTEAU-COUANET (Gustave Roussy) and Annabelle MAEGHT (Oscar Lambret Center in Lille). Finally, this moment dedicated to the needs of families ended with a presentation of the project led by Anne-Laure Delaunay (Mines-Télécom Business School). This project supported by the INCa, and in which Imagine for Margo is participating, will make it possible to identify the information needs of parents during the different phases of their child’s illness (diagnosis, treatment, remission) and their use of technologies (internet, applications, social networks). The aim of this study is to develop a digital solution that answers families’ questions and is adapted to their uses.
A COLLECTIVE FORCE ALONGSIDE CHILDREN
This 11th FAST conference was a highlight of our fight, a few days before the International Day dedicated to childhood cancer. We would like to thank everyone who mobilized on February 10, 2024, namely the 180 participants, our hosts at Chateauform George V, the 21 speakers and all the members and volunteers of Imagine for Margo. As indicated by Mr. Deputy Michel Lauzzana (Vice-President of the Cancer Study Group of the National Assembly) in his closing speech of the conference, the dynamic driven by the collaboration of all stakeholders allows us to be optimistic on the future and to stay the course that we have set for ourselves: to make tomorrow a world with cancer-free children.
