Patients on the trial will have one of the particular genetic mutations among rare cancers.
Cancer Research UK, The University of Manchester, and Roche have announced the start of a multi-drug, precision medicine trial for people with rare cancers who require further treatment options.
The DETERMINE trial is recruiting both adult and paediatric patients with any rare cancer type and is one of the only precision medicine platform studies in the world targeting these wide-ranging populations.
The study aims to establish whether existing therapies – including those which are licensed for more common types of cancer – could benefit patients with rare cancer types that current treatment is not licensed for. Patients who qualify will have undergone genetic screening and discovered that they have one of the particular genetic mutations in their cancer that can be targeted by the specific drug being analysed.
The trial’s design means that any treatment which appears to be working for patients will be submitted for review by the Cancer Drug Fund (CDF). The CDF will then decide whether to collect more data and determine if the drug could be used as a routine treatment option across the NHS.
Cancer Research UK’s Centre for Drug Development is managing the trial, with the University of Manchester leading it. Meanwhile, Roche is providing seven of its targeted drugs to be evaluated in the first instance. Further pharmaceutical partners are encouraged to join and contribute drugs as the trial progresses.
The first trial site has opened at the Christie NHS Foundation Trust with other sites to follow, including the Royal Marsden NHS Foundation Trust, the University of Glasgow, and the University of Birmingham.
Iain Foulkes, executive director of research and innovation at Cancer Research UK, explained: “This platform trial is a significant milestone in Cancer Research UK’s commitment to finding better treatments for cancer patients who desperately need them.
“By looking at drugs that are already available and working with the Cancer Drugs Fund, our trial has a direct route to provide long-term patient access to potentially life-saving drugs which weren’t previously available to patients with rare cancers,” he added.
Dr Matthew Krebs, a chief investigator for the DETERMINE trial at The University of Manchester, commented: “Patients with rare cancer often have few treatment options available, and it’s vitally important we increase our research efforts for these patients. With technological advances in genetic testing, we’ve learned that some rare tumours contain genetic abnormalities, which may benefit from targeted treatment currently only available for more common cancer types.”
Worldwide, rare cancers make up 22 out of every 100 cancers that are diagnosed every year. Despite their prevalence, however, fewer treatment options exist for patients with rare cancers.