The experimentation on precision medicine for rare adult and pediatric tumors opens

Its unique design means that any treatment that appears to work for patients in the study will be reviewed by the Cancer Drug Fund (CDF)**** who will then decide whether to collect more data and assess whether the drug could be used as an option routine care on the NHS for patients with this type of cancer.

Cancer Research UK’s Center for Drug Development is sponsoring and managing the study, led by the University of Manchester. Roche is providing 7***** of its targeted medicines for initial evaluation. Other pharmaceutical partners are encouraged to join and contribute their medicines as the trial progresses.

First trial site opened at Christie NHS Foundation Trust with more sites to follow including the University of Glasgow, Royal Marsden NHS Foundation Trust and the University of Birmingham as well as sites across all experimental medicine centers Adult and Pediatric Cancer Network (ECMC)******.

Iain Foulkes, executive director of research and innovation at Cancer Research UK, said:

“This platform study is a significant milestone in Cancer Research UK’s drive to find better treatments for cancer patients who desperately need them. By examining already available drugs and working with the Cancer Drugs Fund, our study has a direct path to providing patients with long-term access to potentially life-saving drugs that were not previously available to patients with rare cancers.

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

Dr Matthew Krebs, lead investigator for the DETERMINE study at the University of Manchester and the Christie NHS Foundation Trust, said: “Patients with rare cancers often have few treatment options available and it is vitally important to scale up our research efforts for these patients. With technological advances in genetic testing we have learned that some rare cancers contain genetic abnormalities that may benefit from targeted treatment currently available only for the most common types of cancer. We will undertake extensive research to understand which patients with rare cancers might benefit from these treatments. With the potential to change outcomes for adults, adolescents and children with rare cancers, this study will be game-changing for a patient population that often feels overlooked by current cancer research.”

Pete Burchill and Jacqui Gath, patient representatives on the trial steering committee, agree “This project is an example of co-creation. A large number of people with cancer have been involved since the beginning of the trial. The result is a clinical trial that supports patients in all phases of their clinical trial experience and a protocol designed to answer the questions that matter most to patients, in a patient-friendly way.

*DETERMINE: Determining Extended Therapeutic Indications for Existing Drugs in Rare Indications Defined at the Molecular Level Using a National Evaluation Platform Study

A Study on Cancer Research UK’s Umbrella Basket Adaptive Platform to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Pediatric, and Adolescent and Young Adult (TYA) Cancers with Actionable Genomic Alterations, Including Common Cancers with rare feasible alterations.

**Cancer Research UK:

***DETERMINE will establish a new paradigm in precision medicine studies by incorporating a focused, real-time, multi-omics translational research agenda. Translational data will not only inform real-time optimization of patient selection in the study, but will also be used to uncover potential new targeted therapeutic options in rare and pediatric cancers.


*****Combined treatments Cotellic + Zelboraf and Herceptin + Perjeta. In addition, the individual agents Alecensa, Rozyltrek and Tecentriq.

******The Experimental Cancer Medicine Centers Network (ECMC) 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 from funders of over £100m, this infrastructure award supports a network of 18 adult centers (of excellence) and 11 pediatric sites across the UK.

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