Each year, 2,500 cancer cases are diagnosed in those under 19. Considered rare diseases, they are different from those that affect adults. Researchers are carrying out specific work and the care is adapted.
In laboratories, scientists are trying to decipher the mechanisms of pathologies affecting children and adolescents. Objective: to develop treatments adapted to the youngest for these rare diseases.
Ewing tumor, retinoblastoma, osteosarcoma… 85% of childhood cancers are diseases that are not found in adults and therefore require appropriate treatments. Problem: there is no drug development today that would target the abnormalities characteristic of tumors in the youngest.
As for standard treatments (chemotherapy and radiotherapy), they are effective but toxic in the short and long term and can generate serious sequelae. This low availability of pediatric drugs is explained by the rarity of these diseases: with 2,500 cases diagnosed each year, it is difficult to conduct a clinical trial, but especially to interest manufacturers.
Analysis of the tumor genome as breadcrumbs
Scientists do not disarm. They focus on identifying the origins of these cancers, an essential step if we want to be able, in the long term, to develop suitable treatments. Unlike adult cancers, which appear under the pressure of external aggressions such as tobacco or asbestos, pediatric cancers occur without really knowing why. The researchers’ main mission then consists of identifying the genetic modifications of tumors, in order to better understand the biology of cancers, what triggers them, maintains them, etc.
The example of neuroblastoma
Within the Genetics and Biology of Cancers unit at Inserm and the Institut Curie, researchers are interested in neuroblastoma, a cancer affecting embryonic cells of the sympathetic nervous system mainly in the abdomen or along the spine.
The cause of this disease is a receptor which, when mutated, instructs cells to multiply constantly. But if we block its activity, cancer cells no longer proliferate. There is a molecule, crizotinib, capable of blocking this receptor.
Today, this anticancer medicine is only authorized for adult patients with lung cancer. But a clinical trial, launched in July 2013 as part of INCa’s AcSé program, is studying whether, as researchers suspect, it could also be effective on neuroblastoma that affects children: after genetic analysis of their tumor, we can know if it can be attacked by crizotinib. Such work allows children of the boom to benefit from targeted therapies, adapted to each case, and immunotherapy, both already offered to adults but very late for children.
Alice bomboy
File produced in partnership
with Science & Health, the Inserm magazine
.