This Thursday, September 14, 2023, we organized with the Institut Curie an evening dedicated to the 10 years of our collaboration. Researchers, pediatricians, donors and volunteers came together to celebrate 10 years of innovations for the benefit of children and adolescents with cancer. Among the 42 research programs that we have funded since 2011, 12 involved researchers from SIREDO of the Institut Curie, representing funding of 3.5 million euros. Those present at this evening were able to discover research programs that make it possible to better understand and better treat each tumor of each patient, and thus participate in the acceleration of precision medicine.
“When I started in the early 1980s, we cured less than one in two children. Today, 8 out of 10 recover. This is considerable progress. But this also means that nearly 20% of sick children, that is to say 1 in 5 children, die. Progress is possible and the challenges are major. » Pr. Thierry Philip, Chairman of the Board of Directors of the Institut Curie.
BETTER UNDERSTANDING OUR CHILDREN’S CANCER
Thanks to progress in fundamental and clinical research, of the 2,500 children diagnosed each year, it is possible to cure 2,000. This progress nevertheless remains unsatisfactory since 500 patients present with incurable cancers which escape first-line treatments.
ACCELERATE PRECISION (MOLECULAR) MEDICINE: CURE TUMOR BY TUMOR
To better care for a child, it is necessary to give them the treatment best suited to the specificities of their tumor. This therefore requires identifying the genes and factors involved and understanding their role in tumor progression and resistance to therapies. Imagine for Margo took a large part in this titanic work by heavily funding the MAPPYACTS and MICCHADO projects, which were presented by Dr. Gudrun Schleiermacher of the Institut Curie. This pediatric oncologist, head of the RTOP research team, is firmly convinced of the direct benefit of these projects:
“If these cancers with a poor prognosis are known, and certain treatments are effective at diagnosis, it is not possible to increase the intensity of these standard treatments. This is why we must design targeted approaches to increase the chances of cure and fight against tumor resistance. »
Thanks to the MAPPYACTS and MICCHADO projects, respectively carried out on 757 children with relapsed cancer and 554 others with high-risk cancer, it is now possible to establish the molecular profile of each tumor, and to find and target the “Achilles heels”. More concretely, thanks to the MAPPYACTS project:
- One or more abnormalities were found in 69% of patients
- 30% were able to benefit from targeted therapy, notably as part of the AcS2-ESMART program, which Imagine for Margo also co-finances.
ALSO UNDERSTAND THE EVOLUTION OF EACH TUMOR, ALMOST IN REAL TIME
This work showed the usefulness of complete tumor sequencing, which is now covered by Health Insurance. However, these analyzes only provide a snapshot of the tumor genome at the time of analysis, generally at the time of diagnosis or relapse. In order to better understand the evolution of tumors, Dr. Gudrun Schleiermacher showed the importance of analyzing fragments of tumor DNA, which circulate in the patient’s blood. As part of the MAPPYACTS trial, physician-researchers sequenced the circulating tumor DNA of 56% (128/225) of patients. They found 76% of the abnormalities present in the original tumor and identified 35 new potentially exploitable mutations. This technique, tested for the first time in the pediatric population, has the advantage of being much less invasive, since it requires a simple blood test to be sufficient. This research is continuing and more than 50 additional studies will be carried out using data from the MAPPYACTS 2 study.
SHARE CLINICAL DATA AND BRING TOGETHER THE BEST EXPERTS AT CHILDREN’S BEDSIDE
In addition to MAPPYACTS and MICCHADO, similar programs have emerged in Europe and around the world: INFORM in Germany, ITHER in the Netherlands, SM-PAEDS in Great Britain or PROFYLE in Canada. The ITCC network, a major partner of Imagine for Margo, has created a hub to bring together and share the clinical and molecular data of each patient, obtained in these programs from 13 countries. With its connections to more than 60 clinical trial centers in Europe and thanks to this hub also co-funded by Imagine for Margo, researchers will be better able to accelerate precision medicine.
Across our country, interdisciplinary committees or tumor boards have also been created and use this genetic and immunological data. Thanks to this teamwork, the children treated have a greater chance of receiving the treatment best suited to the specificities of their cancer and see their chance of recovery increased.
INNOVATIONS FOR BETTER HEALING
As Professor Thierry Philip underlined in his introduction, the research, care and transmission of knowledge continuum is absolutely fundamental to progress in our fight against childhood cancer. This base of values is strongly supported by Imagine for Margo, which thanks to its interactions with the SFCE and the ITCC and the creation of Fight Kids Cancer, has enabled clinical and translational research projects to see the light of day, particularly at the Curie Institute. These projects, TAG-N-TRAK, ASI-DNA, EPIRT, RETINOBLASTOMA, COBIORB, COMBALK or BioESMART, have explored new treatments, opened up promising avenues, and provided more effective or less toxic therapeutic solutions. In addition, these projects target high-risk cancers for which research has not yet made it possible to improve the survival rate (e.g. Ewing sarcoma, high-grade gliomas, rhabdoid tumors, neuroblastoma, etc.).
TAG-N-TRAK: FROM RESEARCH FOR A MUTATION TO A SYRUP TO TAKE TWICE A DAY
This project, co-funded by Imagine for Margo, started in 2019 and is led by Dr. Daniel Orbach, pediatric oncologist and director of the clinical department of pediatrics – adolescents – young adults at the Institut Curie. It has been shown that an abnormal protein, NTRK, is involved in childhood and adult cancers, particularly fibrosarcomas and brain tumors. However, and as he clarified in his presentation during this evening, a new drug had shown its effectiveness, in adults, to target this alteration. With the agreement of the families, Dr. Orbach and his team were able to identify, thanks to the genetic data previously collected, the children carrying this mutation. In collaboration with the other pediatric oncology centers of the SFCE, the clinical, therapeutic and evolutionary data of these children could be retrieved and analyzed. This step made it possible to launch the VITRAKVI clinical trial in 2022 and to test this drug in children, generally infants, affected by fibrosarcoma including the NTRK anomaly. The results are then spectacular. “The tumors melt away visibly,” Dr. Daniel Orbach tells us. Thus, this treatment, which is a syrup to take morning and evening and associated with surgery, makes it possible to definitively defeat this form of cancer. But the story does not end there. An international collaboration has been set up to detect NTRK in brain tumors, with the hope that this drug, too beautiful and yet true, could be just as effective there.
To discover all of the Curie Institute’s research programs funded by Imagine for Margo: Consult the evening booklet
A COMMITMENT TO CARRYING THE VOICE OF EVERY CHILD LIVING WITH CANCER
As Dr. Olivier Delattre and Professor François Doz indicated in their intervention during this evening, Imagine for Margo does not only finance research programs. Thanks to our numerous collaborations, meetings, symposia and participation in national and international committees, we carry the voice of all patients to advance the cause of childhood cancer at the medical, scientific and political level.
“Imagine for margo is not just a financial partner. Its actions go beyond financial support for research. They also go into the structuring of pediatric oncology and its research, into lobbying at parliamentary level and also into its relations with drug manufacturers during the ACCELERATE forums for example. But also, Imagine for Margo is a partner of the projects themselves. Its president participates in meetings of the ITCC brain tumor group to support research projects and express her point of view on progress in pediatric oncology. » François Doz, pediatric oncologist, Deputy Director of clinical research, innovation and teaching at SIREDO.
During his speech, Dr. Olivier Delattre insisted on the fact that in France, Imagine for Margo is the association which has carried out the most patient advocacy (or advocacy for patients) for childhood cancers. This citizen activity, strongly developed internationally, contributes to the redefinition of health policies, to make them more effective. By carrying the voice of patients and families, by emphasizing the difficulties of doctors and researchers in accelerating research and medicine, we have participated in this movement which has caused the modification of European regulations on medicines for pediatric and orphans.
GO, FIGHT, WIN…THREE UNITERATING WORDS THAT ARE THE DRIVERS OF PROGRESS
The missions carried out by Imagine for Margo are all the more essential as we see the fruits of 40 years of fundamental research which today lead to new medicine, more targeted and bringing great hope. As Dr. Delattre indicated: “Research today has a certain degree of maturity which makes it possible to propose solutions. “. Precision molecular medicine, immunotherapy, or acceleration of bioinformatics are all examples presented during this evening which show that we are on the path to healing all children.
“Let’s keep running, fighting and winning. Our ability to come together, in France and in Europe, to explore, advance and invent is a great strength that will make tomorrow a world with cancer-free children. » Patricia Blanc, president of Imagine for Margo.
Imagine for Margo warmly thanks Pr. Thierry Philip, Pr. François Doz, Dr. Olivier Delattre, Dr. Gudrun Schleiermacher, Dr. Daniel Orbach, Mrs. Tatiana Malherbe and Mrs. Tatiana Romero as well as all the researchers and doctors at the Institut Curie who are mobilizing for our children and who welcomed us for this beautiful evening, placed under the sign of progress and hope.
