RARE Daily

Cancer Researchers in UK Collaborate on Finding Drugs for Rare Cancers

December 21, 2021

Cancer Research UK, The University of Manchester and Roche have entered 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.

Photo: Matthew Krebs, chief investigator for the DETERMINE trial at The University of Manchester

Worldwide, rare cancers make up 22 percent of 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 drugs, meaning those which are already approved for more common types of cancer, could also benefit patients with rare cancer types that the drug isn’t currently licensed for.

The DETERMINE trial will be one of the largest precision medicine platform trials targeting these populations and it will enroll patients who have an identifiable genetic alteration in their cancer that can be targeted by repurposing drugs approved to treat other cancer types.

The partners said the trial’s unique design means that any treatment shown to benefit patients on the trial could be fast-tracked towards approval on the UK’s National Health Service.

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 pediatric 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 seven of its 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 pediatric cancers, as well as more common cancers with rare genetic alterations that could be targeted by the drugs being studied in the trial.

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 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. 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 five years.

“Patients with rare cancer often have few treatment options available and it’s vitally important we increase our research efforts for these patients,” said Matthew Krebs, chief investigator for the DETERMINE trial at The University of Manchester and The Christie NHS Foundation Trust. “With technological advances in genetic testing we’ve learned that some rare tumors 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.”

Author: Rare Daily Staff

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