First-ever study in the personalised treatment of rare cancers brings promising results

The first-ever trial to treat patients for rare cancers in a personalised manner based on the characteristics of their individual disease has wrought promising results, it has emerged.

The approach employed by the study differs from the traditional methodology which is generally determined by a cancer’s origin point, size and the stage of the disease, irrespective of the individual characteristics of the particular case. This has historically led to a huge discrepancy in success rates from patient to patient.

The study examined 40 rare cancer patients at the Rare Tumour Clinic at the University of California San Diego Moores Cancer Center, taking blood and tissue samples to analyse the molecular and genetic features of each tumour to identify potential drug targets.

The team managed to find appropriate therapies for just over half of patients, including chemotherapy, and 52.4% of those treated had their tumours halt progression, shrink or disappear, with those who had received previous treatment seeing longer-term results.

"Rare cancers make up around a quarter of all cancers, but these patients are often not eligible for traditional clinical trials because of difficulties recruiting sufficient numbers," explained Dr Nischalan Pillay, a sarcoma expert from University College London funded by Cancer Research UK. "This small study provides a way to pursue personalised treatment for these patients.

"There is an urgent need to develop new ways to design clinical trials for patients with sarcomas and other rare cancer types," he continued. "The design of this particular trial, where recruitment is based on the molecular characteristics in a patient’s tumour rather than on the tumour type, is one way of addressing this. In order to fulfil the promise of personalised medicine more of these kinds of trials should be undertaken."