A molecule of hope against pediatric cancers: the Flavien Foundation calls for donations to finance a clinical trial
In medicine, between the first research on the “bench” of a laboratory and the realization of a clinical trial, there is often a chasm.
In Monaco, thanks to a molecule called “hope”, researchers at the Monaco Scientific Center are about to change the lives of children with childhood cancers. Without the moral and financial support of Denis Maccario and his Flavien Foundation, nothing would have been possible. The father of little Flavien, who died in 2014 at only 8 years and 9 months of metastasized brain cancer, was able to unite all the forces around this scientific project. We explain to you.
What is this molecule of hope?
Developed by the pharmaceutical company Pfizer, this molecule responds to the name of axitinib. Already used for the treatment of kidney cancer in adults, it arouses a wave of hope among families of children suffering from medulloblastoma, a tumor located in the cerebellum, or other pediatric cancers.
Because the researchers of the Scientific Center of Monaco have demonstrated that it could possibly lead to their daily life, punctuated by heavy treatments – radiotherapy session and to promote but also excision surgery -, sources of side effects and sometimes irreversible sequelae.
What are the benefits for sick children?
After six years of research and scientific publications, therefore, the benefits have been proven. By cell culture in vitro, first of all. “We have seen that the molecule can eliminate tumor cells while preserving healthy cells. In another case, it allows less proliferation”, summarizes Dr. Vincent Picco who heads the “stem cells and brain tumors” unit at the Department of Medical Biology of the Scientific Center of Monaco.
Less resistance than a conventional treatment, therefore, which proved effective during experimentation live thanks to the implantation of tumors in the brain of mice.
A particularly technical operation led by Marina Pagnuzzi, the cornerstone of the project. “All the lights were green.”continues Dr. Vincent Picco.
Next step: the clinical trial
This is the next step in the process: phase 1 of the clinical trial, says the test of axitinib otherwise directly on young sick patients. “We enter the hard with a therapeutic possibility. Clinicians will check if this is of benefit to children.”summarizes Gilles Pagès, INSERM research director at IRCAN in Nice.
Something made possible thanks to the bridge made by the Flavien Foundation between the CSM and Professor Nicolas André of the Public Assistance – Hospitals of Marseille.
It is this internationally renowned pediatric cancer specialist, and former doctor of Flavien, who will pilot the clinical trial for three years with 36 children, on the basis of their respective hospitals. “These will be patients in relapse, in therapeutic impasse with conventional treatments [30% d’échec, N.D.L.R.], the outcome of which we know is fatal in the months or years to come”, explains Gilles Pages. Could this be the last chance treatment?
“Scientists and doctors are modest and cautious, does he nuance. If we manage to improve the quality of life of these children who, in relapse, have severe effects, it will already be a huge step forward..
Phase 1 will thus make it possible to answer many questions: is the product effective? Does it limit side effects? Is the tumor shrinking in size? Do the metastases regress or disappear?
If it proves conclusive, a phase 2 with a higher contingent of patients would be initiated with, for epilogue, a therapeutic hope.
Fundraising needed
To conduct such a clinical trial, because it takes money. The force of the war. For the planned three years, 620,000 euros are needed. The Flavien Foundation has already pledged 200,000 euros. CFM Indosuez Wealth Monaco has just signed a check for 40,000 euros and an additional 5,000 euros have been distributed by patrons. The call for donations is therefore launched and many generous souls are expected.
To use axitinib other than for its initial function, the authorization of its owner is necessary. “A year and a half battle”according to Gilles Pages.
Because if Pfizer had given the green light in 2020, at the regional level, the refusal was categorical in 2021 on the part of Pfizer USA. It took the intervention of Prince Albert II, a letter from the Minister of State in January 2022, for the pharmaceutical company to change its mind.
“Our scientific publication was also supported by a group of German researchers who published results similar to ours. This validated the performance of the molecule. Pfizer gave up under scientific pressure”, says Gilles Pagès. If Pfizer provides the molecule free of charge, it does not finance the clinical trial.
There are now 375,000 euros left to collect. Nothing insurmountable for the Flavian tsunami, the name given to the momentum around the eponymous Foundation.
To make a donation
https://www.fondationflavien.com/faire-un-don/
Young patients provided axitinib in “compassionate access”
Denis Maccario, President of the Flavien Foundation, relayed the testimonies of young patients in a therapeutic impasse who were able to benefit from the “axitinib” molecule in compassionate access. A legal requirement although outside the framework provided by their marketing authorization.
Among them, Cécilia, a 22-year-old Marseillaise, who, in 2015, learned of the existence of a malignant tumor lodged in her brain. Followed by Professor Nicolas André at the AP-HM de la Timone, in the heart of the Marseille city, Cécilia has since undergone very heavy treatments and surgical interventions.
Oscillating years between hopes of recovery and relapses. And this unpleasant feeling that the disease will never give him respite. Like in 2020.
“Still having these control MRIs. In December, I was told I had a relapse. According to the MRIs, the tumors are described in the brain but they made babies along my spine”she confides.
In response, oral promotion will yield nothing. Failing to regress, the tumors multiply. In 2022, after its last relapse, axitinib is then administered by doctors in treatment.
“I’m still on treatment and my hair is growing back. details Cecilia. Good luck to families and especially to children with cancer, we must not stop believing in it, we are stronger than this fucking disease, we must hang on and have a lot of patience.
Hope is therefore allowed for all the children who will participate, if the funds are met, in the clinical trial led by Pr Nicolas André. Permitted thanks to the expected research at the Scientific Center of Monaco.