In 62% of the just under 2,500 patients tested, opportunities for tailored treatment or investigation options were found in their tumour DNA. Of these, 31% were possibly suitable for an approved drug, 18% on-label (covered by health insurance) and 13% off-label (not covered). The remaining 31% were patients who might be eligible to take part in experimental research into new drugs.
The study was conducted by the Centre for Personal Cancer Treatment (CPCT), which was co-founded by the Netherlands Cancer Institute and to which 46 Dutch hospitals are currently affiliated, along with the Hartwig Medical Foundation. This privately funded foundation specialises in reading the whole tumour DNA, and it manages the largest databank in the world of metastasised tumours in which biological data is linked to clinical data such as treatment outcomes.
A spin-off from the study in 2016 - with funding from the Dutch Cancer Society and Barcode for Life - was the Drug Rediscovery Protocol (the 'DRUP study'). This involves patients with metastasised cancer whose treatment options have run out and who, based on abnormalities in their tumour DNA, could possibly benefit from drugs already on the market for other types of cancer. The question is: do these patients actually benefit from off-label drugs that are suitable for their tumour DNA?
In another Nature article, published 29 September, the researchers reviewed the first three years of the DRUP DRUP. Of the first 215 patients involved, distributed among 76 cohorts, an average of 34% were found to benefit from their trial drug: the cancer remained at least stable for four months, or the tumour shrank in size.
In a DRUP cohort of 30 patients with a specific DNA abnormality that makes their tumours extremely susceptible to mutation (MSI-h), 67% benefited from treatment with the immune drug nivolumab. Given this hopeful outcome, the DRUP researchers, the Dutch National Health Care Institute, the health insurers and the pharmaceutical company have together developed a tailored insurance system which will enable new patients whom this drug was found to help in the DRUP study to have access to it after the trial phase.
The next step: the Netherlands Cancer Institute, UMC Utrecht and the Hartwig Medical Foundation are now examining whether reading the entire tumour DNA (whole genome sequencing) could also be used outside clinical trials in standard hospital diagnosis for patients with metastasised tumours. The aim is to have tailored treatment available for as many patients as possible, based on their unique tumour profiles. For this work the researchers have received 1.5 million euros this year from the Netherlands Organisation for Health Research and Development.