A previously shelved drug could prove effective for treating a group of rare childhood cancers, and Cancer Research UK today announced it has been granted exclusive marketing rights for the drug in these indications by the European Commission and the Food and Drug Administration (FDA).
The drug, called fenretinide, was first made in the 1970s by pharmaceutical giant Johnson & Johnson as a possible treatment for breast cancer, but the company didn't bring it to market. Now out of patent, fenretinide has been studied by research groups in relation to various types of cancer, including the Ewing's sarcoma family of tumours (ESFT), a group of rare cancers that affect around one in every million young people (0-24 years old), usually diagnosed in adolescence, in the EU each year.
A number of laboratory studies of fenretinide in ESFT cells were carried out by Dr Sue Burchill, based at Cancer Research UK's Clinical Centre in Leeds and supported by the local children's cancer charity, Candlelighter's. Her promising results convinced Cancer Research UK to apply for orphan designation for the use of fenretinide in the treatment of soft tissue sarcomas and malignant bone tumours – two categories of disease in the ESFT group.
Drugs for rare diseases are called 'orphans' because the small number of patients they would treat means they are often not developed by the pharmaceutical industry. Today's decision will make it easier for Cancer Research UK's development company, Cancer Research Technology Ltd (CRT), to attract biotech companies to partner with the charity to help develop fenretinide for clinical use. Fenretinide will be accelerated through the European Medicines Agency's (EMEA's) and FDA's approval processes, and CRT and its partners will have ten and seven years to exclusively market the drug in the EU and US respectively.
The next step in the development of fenretinide is a phase II trial, which has been approved for funding by Cancer Research UK.