The ESMART trial, supported by the INCa, will enable 260 children and adolescents experiencing treatment failure to access the latest generation treatments.
Childhood and young adult cancers remain rare diseases, which in 80% of cases can be treated with standard treatments. But for young patients experiencing therapeutic failure, access to innovative molecules is essential. As part of the Cancer Plan, the National Cancer Institute (INCa) is committed to facilitating the inclusion of children and adolescents in early clinical trials. The AcSé-ESMART trial, launched on Tuesday, should make it possible in two years to double the number of drugs for these children.
Ten molecules available
ESMART is a unique trial in the world in pediatric cancers. It will allow 260 young patients experiencing therapeutic failure to have access to around ten innovative molecules (targeted therapies and immunotherapies), developed for adults, but which may prove effective in pediatric cancers.
Patients will be cared for in one of the 6 INCa-certified early trial centers (CLIP), now open to children. “The CLIPs were reserved for adults until 2016, the labeling of these 6 centers for pediatric cancers is a strong message from INCa”, underlines Prof. Gilles Vassal, director of clinical research at Gustave Roussy.
The children included in the study will benefit from molecular diagnostics to identify possible therapeutic targets on their tumour. Depending on this “genetic portrait”, an innovative treatment will be offered to them.
Find the program L’invité santé, with Dr Dominique Valteau Couanet
child and adolescent oncology department, Gustave Roussy, Villejuif (June 9, 2016)
Too late in pediatric trials
These drugs are molecules developed for adults and for which pediatric use is not yet possible routinely, due to insufficient data. “There is a lot of delay in pediatric trials, deplores Gilles Vassal. The law provides that at the end of phase 1 trials in adults, a pediatric study plan is filed, but this is not the case. »
Delays which sometimes lead to absurd situations, an anti-PD1 antibody, whose effectiveness against Hodgkin’s lymphoma has been demonstrated, has just been authorized in the United States. But for lack of pediatric data, the drug can only be prescribed to adults, but “this lymphoma is a disease typical of adolescents and young adults”, underlines Gilles Vassal.
Strengthen legislation
Long singled out for their lack of innovation in pediatric drugs, pharmaceutical companies – for some – are beginning to review their position on the subject. The ESMART trial is also the result of a collaboration between INCa, the parents’ association Imagine for Margo, Gustave Roussy and three laboratories. Astrazeneca, BMS and Novartis have thus committed to supplying several of their latest molecules. “There is an ongoing reflection among manufacturers, who want to do better and differently,” notes Gilles Vassal.
Gilles Vassal, director of clinical research at Gustave Roussy: “ We started to take off with the regulations now we have to completely change gear! (…) We must also change the state of mind…»
But for the specialist, who is also president of the European Society of Pediatric Oncology, accelerating access to innovation necessarily involves mobilizing decision-makers. “The law on pediatric drugs has really changed the landscape but it does not go far enough, and it does not correspond to the needs of children,” said Professor Vassal.
Promulgated in 2007, this text provides that any molecule developed in adults be the subject of pediatric trials, “if the need exists”. A clarification that in many cases allows manufacturers to request an exemption. This is particularly the case for crizotinib, a molecule used in the treatment of lung cancer. Cancer which of course does not exist in children. Gold A try, conducted at Gustave Roussy, and whose results were presented by Gilles Vassal at the last Congress of the American Society for Clinical Oncology, shows that the molecule is effective in different pediatric cancers.
A reassessment of the law was planned after 10 years. Europe will therefore have to take a position next year. Oncologists and parents hope that it will strengthen the legislation to limit the number of derogations, and allow faster access to innovation for all children in the European Union. Today, only one in ten has access to an innovative molecule when treatment fails.
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