Patients with rare cancers often face limited treatment options due to the rarity of their condition, which makes it difficult to conduct large-scale trials and secure regulatory approval for new treatments. Although each rare cancer type is uncommon, collectively they account for approximately 20% of all cancers diagnosed worldwide each year — a larger share than any single type of cancer, including lung, breast, or colorectal cancer.
To address this, Dr. Matthew Krebs from The University of Manchester (UoM) is leading the DETERMINE trial, the UK's first precision medicine study focused on rare cancers. Sponsored and managed by Cancer Research UK's Centre for Drug Development, the trial is being conducted in partnership with the University of Birmingham, the Royal Marsden NHS Foundation Trust, and the Christie NHS Foundation Trust — bringing together leading expertise in oncology and precision medicine. This trial tests existing cancer drugs on tumors with specific genetic mutations, even if the drugs were originally designed for other cancer types. The goal is to identify whether these treatments can benefit patients with rare cancers based on the genetic profile of their tumors rather than the cancer's location.
If successful, the trial could provide new treatment options for rare cancer patients and speed up NHS approval through the Cancer Drug Fund (CDF). This innovative approach represents a shift toward personalized medicine, offering renewed hope to patients with limited options.