A Hope for a Cure for a Paediatric Brain Tumour from a Derivative of Vitamin A

Fenretinide, a derivative of vitamin A, could form the basis of a new treatment capable of curing a paediatric brain tumour, known as diffuse midline glioma (DMG). This statement is supported by an international research team, which includes two scholars from the University of Bologna – Isabella Orienti and Giovanna Farruggia from the Department of Pharmacy and Biotechnology – in a study published in the journal Neuro-Oncology.

(More) notably, the University of Bologna research group has developed a new fenretinide formulation called NanoFenretinide, which has shown significative antitumour efficacy without side effects when administered to mice bearing human DMG tumours.

“NanoFenretinide, consisting of fenretinide nanoaggregates and phospholipids, makes the drug suitable for intravenous administration”, says Isabella Orienti. “This increases its ability of reaching and penetrating body tissues, including brain tissue, and its antitumour efficacy”.

Diffuse midline glioma is a brain tumour which primarily affects children, mainly between the ages of 5 and 10. At present, radiotherapy is the only treatment capable of slowing the development of the disease. Nevertheless, the average survival time remains extremely low, ranging between 9 and 12 months.

Scholars have screened 3570 drugs already being used for different pathologies to identify new therapeutic solutions. The goal was to find existing and approved active substances capable of tackling diffuse midline glioma, thus reducing the development times required for a new therapy.

The analysis has highlighted six molecules which have shown promising results in in vitro cell cultures. Only one of these has proven to be effective even in vivo on animal models: fenretinide.

“When administered to mice with diffuse midline glioma, NanoFenretinide has proven to be effective in fighting the disease, without significant side effects”, says Giovanna Farruggia. “These results raise expectations that NanoFenretinide may enter clinical trial as soon as possible, to give hope of recovery to the young patients affected by DMG and other brain tumours”.

Furthermore, scholars highlight that, considering the gathered data, NanoFenretinide might prove to be effective against other types of tumours as well, such as neuroblastoma, some forms of leukaemia, melanoma and some types of colon, lung and breast cancer.