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Precision medicine trial opens for rare adult and paedatric cancers

Its unique design means that any treatment which appears to be working for patients on the trial will be submitted for review by the Cancer Drug Fund (CDF)**** who will then decide whether to collect more data and assess if the drug could be used as a routine treatment option on the NHS for patients with this cancer type.

Cancer Research UK’s Centre for Drug Development is sponsoring and managing the trial, with the University of Manchester leading it. Roche is providing 7***** of their targeted drugs to be evaluated in the first instance. Further pharmaceutical partners are encouraged to join and contribute their drugs as the trial progresses.

The first trial site has opened at the Christie NHS Foundation Trust with other sites to follow, including the University of Glasgow, the Royal Marsden NHS Foundation Trust and the University of Birmingham, as well as sites across the entire adult and paediatric Experimental Cancer Medicine Centres (ECMC) network******.

Iain Foulkes, Executive Director of Research and Innovation at Cancer Research UK, said:

“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 which 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.

“Cancer Research UK’s Centre for Drug Development is unique in the world and the partnerships they have built with pharmaceutical companies will enable this critical trial.”

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.”

Pete Burchill and Jacqui Gath, Patient Representatives on the Trial Steering Committee agree that “This project is an exemplar for co-creation. A large number of people affected by cancer have been involved from the very beginning of the trial. The result is a clinical trial that supports patients throughout all stages of their clinical trial experience and a protocol that is designed to answer the questions most important to patients, in a patient friendly way.”

*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. 

**Cancer Research UK:

***DETERMINE will set a new paradigm in precision medicine trials by incorporating a focused, real-time multi-omic translational research programme. The translational data will not only inform the real-time optimisation of patient selection into the study, but also be used to discover potential new targeted therapy options in rare and paediatric cancers.


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

******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.

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