Conducting trials in rare cancers is challenging, not least because it is difficult to find enough patients to take part and give a reliable result. Some cancers are so rare that there may only be one or two patients diagnosed in a year in any one country, and even studying existing drugs for use in such a small group is not worthwhile for the pharmaceutical industry. Now, the EORTC CREATE trial is looking at the use of the drug crizotinib in very rare tumours. Crizotinib works by targeting the growth of cancer cells that have specific abnormalities called ALK and MET, and it is already known to be effective in the treatment of advanced lung cancer.
Knowing that ALK and MET abnormalities were involved in some very uncommon cancers, the researchers decided to investigate the effect of crizotinib on six tumour types. The rarity of these cancers can be seen in the fact that fewer than 200 patients in total took part in the trial. Results so far are promising. For example, in inflammatory myofibroblastic tumours, which occur in smooth muscle and connective tissue cells, researchers have found that the tumours respond well to crizotinib treatment. The latest results show that up to around 70% of patients have a positive response to treatment, and these data have been used to support an application to the US Food and Drug Administration for the authorisation of the use of crizotinib in children with inflammatory myofibroblastic tumours.
CREATE has been valuable in encouraging the creation of working groups on the ultra-rare tumours being studied and stimulating radiologists as well as medical oncologists to work on them. Patients
in the trial have also benefited from their treatment; many are still on crizotinib even though it is not yet on the market in some countries or in those where it is, it is not approved for the tumour type being studied. Additionally, the long-term follow-up of the trial will give researchers the chance to better understand the natural course of these rare diseases, and this will aid in the planning of future research.
Study coordinator: Prof. Patrick Schoeffski