Cancer researchers across the UK join forces to find drugs for rare cancers

Published date:
December 20 2021

Cancer Research UK, The University of Manchester and Roche, today announce a partnership to run a multi-drug, precision medicine platform trial for adults and children with rare cancers who have run out of other treatment options.

The DETERMINE* trial will be one of the largest precision medicine platform trials targeting these populations and it will enrol patients who have an identifiable genetic alteration in their cancer that can be targeted by treatments that are already approved for use in other cancer types. Its unique design means that any treatment shown to benefit patients on the trial could potentially be fast-tracked towards approval on the NHS**.

The University of Manchester will lead the trial which will be run in collaboration with the Royal Marsden NHS Foundation Trust, The University of Birmingham, and the Christie NHS Foundation Trust***, with contribution from the adult and paediatric Experimental Cancer Medicine Centres (ECMC) network****. Under the terms of the partnership, Cancer Research UK’s Centre for Drug Development will sponsor and manage the trial, with Roche providing 7***** of their targeted therapies to be evaluated in the first instance. More pharmaceutical partners are expected to join and contribute their drugs as the trial progresses.

The trial is aiming to recruit patients with rare adult and paediatric cancers, as well as more common cancers with rare genetic alterations that could be targeted by the drugs being studied in the trial.

Worldwide, rare cancers make up 22 out of every 100 (22%) cancers that are diagnosed each year****** which is more than any single type of cancer. If we were to define all rare cancers as a single type, they would top the list of the most prevalent cancers worldwide, above lung, breast and colorectal cancer. But despite their prevalence, fewer treatment options exist for patients with rare cancers.

The DETERMINE trial aims to find out whether existing ‘licensed’ drugs, meaning those which are already prescribed by doctors for more common types of cancer, could also benefit patients with rare cancer types that the drug isn’t currently licensed for. The medicines being used in the trial are targeted to specific genetic faults that occur in cancer. Genetic testing is increasingly being undertaken for cancer patients in the UK either on the NHS or as part of other research trials. The genetic testing will help assess whether a patient is eligible for the study and which drug is most likely to benefit the patient.

Any drug in DETERMINE that is shown to benefit patients, even if only in a small group of patients with rare cancers, could be submitted for review by the Cancer Drug Fund (CDF)*******. They would then decide whether to collect more data to assess if the drug could be used more routinely for patients with this cancer type as a treatment option potentially available in an NHS setting. Ultimately the study will create a ‘roadmap’ to help establish new treatment options for patients with some types of rare cancer. This precision medicine trial is set to open to recruitment nationwide in early summer 2022, with the entire length of the trial spanning 5 years.

Dr Matthew Krebs, Chief Investigator for the DETERMINE trial at The University of Manchester and The Christie NHS Foundation Trust, said: “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. We will undertake in-depth research to understand which patients with rare cancers could benefit from these treatments. With the potential to change outcomes for adults, teenagers and children with rare cancers, this trial will be ground-breaking for a patient population who often feel neglected by current cancer research."

Iain Foulkes, Executive Director of Research and Innovation at Cancer Research UK, said: “With numerous researchers involved nationwide and potentially around 850 people with rare cancers taking part, this trial represents a significant undertaking by Cancer Research UK and our partners. But what makes this even more exciting is that we will be able to fast track the approval of any promising drugs, opening the ‘door’ to treatments for patients who have historically been left with limited options.”

Richard Erwin, General Manager, Roche Products Ltd, said: “We are committed to helping find ways to identify the right treatment for the right person at the right time. This study will help in identifying novel treatments for adults and children living with rare cancers.”

Notes to editors


For media enquiries contact Ellie Bennett in the Cancer Research UK press office on 020 3469 5370 or, out of hours, on 020 3469 8301.


*DETERMINE: Determining Extended Therapeutic indications for Existing drugs in Rare Molecularly-defined Indications using a National Evaluation platform trial:

A Cancer Research UK adaptive umbrella basket platform trial to evaluate the efficacy of targeted therapies in rare adult, paediatric and teenage and young adult (TYA) tumours with actionable genomic alterations, including common cancers with rare actionable alterations. 

**Only applicable to England but the teams are in discussions with the devolved nations around treatment access.

***Dr Matt Krebs, from the University of Manchester, is the Chief Investigator for the trial, Dr Lynley Marshall is the paediatric lead at the Royal Marsden Hospital with Professor Gary Middleton and Professor Cindy Billingham from the University of Birmingham the Translational Lead and Statistics Lead, respectively.

****The Experimental Cancer Medicine Centres (ECMC) network is an initiative funded by Cancer Research UK in partnership with the four health departments of England, Scotland, Northern Ireland and Wales. Launched in 2007 with a total investment by the funders of over £100million, this infrastructure award supports a network of 18 adult centres (of excellence) and 11 paediatric locations throughout the UK.

*****Combination treatments Cotellic + Zelboraf and Herceptin + Perjeta. Plus, single agents Alecensa, Rozlytrek and Tecentriq.

******Cancer Research UK:



About Cancer Research UK’s Centre for Drug Development 

Cancer Research UK has an impressive record of developing novel treatments for cancer. The Cancer Research UK Centre for Drug Development has been pioneering the development of new cancer treatments for 25 years, taking over 140 potential new anti-cancer agents into clinical trials in patients. It currently has a portfolio of 21 new anti-cancer agents in preclinical development, Phase I or early Phase II clinical trials. Six of these new agents have made it to market including temozolomide for brain cancer, abiraterone for prostate cancer and rucaparib for ovarian cancer. Two other drugs are in late development Phase III trials.

About Cancer Research UK

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The University of Manchester, a member of the prestigious Russell Group, is one of the UK’s largest single-site university with more than 40,000 students – including more than 10,000 from overseas. It is consistently ranked among the world’s elite for graduate employability. The University is also one of the country’s major research institutions, rated fifth in the UK in terms of ‘research power’ (REF 2014). World-class research is carried out across a diverse range of fields including cancer, advanced materials, global inequalities, energy and industrial biotechnology.