Cancer Research UK’s Drug Development Office has re-launched a trial of a promising drug to treat inherited breast and ovarian cancer – but this time taken as a tablet by outpatients.

The Phase II clinical trial is led by The Sir Bobby Robson Cancer Trials Research Centre, Newcastle University and The Newcastle upon Tyne Hospitals NHS Foundation Trust, and also runs across The Beatson West of Scotland Cancer Centre, UCL, Plymouth Oncology Centre, University of Birmingham Hospital, Christie Hospital, Manchester; and St James’ University Hospital, Leeds.

Women with advanced breast or ovarian cancer with faults in the known high-risk BRCA1 or BRCA2 genes will receive the drug called Rucaparib (CO-338; formerly called AG-014699 or PF-01367338) – which belongs to a promising class of drugs called PARP inhibitors. The trial is also open to women with advanced serous ovarian cancer but unknown BRCA status.

When both copies of the BRCA1 or BRCA2 genes are faulty, the cells rely on the alternative PARP pathway to repair damaged DNA – preventing cancer-causing mistakes being passed on to daughter cells. By also blocking PARP with drugs, cancer cells which have lost BRCA1 or BRCA2 can no longer repair DNA damage at all, causing these cancer cells to die. Serous ovarian cancer patients are also included in the study as they may have DNA repair deficits that could make them sensitive to treatment with a PARP inhibitor.

Patients take the drug as a daily tablet at home over a period of 21 days, only coming to hospital for check-ups and tests. This treatment plan replaces an earlier version of the trial of the drug where it was delivered intra-venously to patients on five days over the same period.

The trial will initially establish the dosing schedule for the drug and evaluate whether it is effective for these patients.

Professor Ruth Plummer, the trial’s chief investigator, at the Northern Institute for Cancer Research, at Newcastle University, said: “We’re seeing encouraging results in women with breast or ovarian cancer treated with PARP inhibitors. It’s great news that we’re able to run a trial of this exciting drug as a tablet which will be a much more convenient and comfortable way to receive the treatment.

“Patients will be able to take a tablet at home – which will mean they can go to work or stay at home with their families, instead of spending long periods of time at hospital with the discomfort of receiving the drug through a drip.”

Faults in the BRCA1 or BRCA2 genes account for around five per cent of the 44,000 women in the UK with breast cancer and for more than five percent of the 6,600 women with ovarian cancer.

PARP inhibitors are being used alone in clinical trials to treat patients with specific types of breast, ovarian and prostate cancers. They can also be combined with existing cancer treatments – including chemotherapy and radiotherapy.

This latest trial is being funded and managed by the charity’s Drug Development Office (DDO).
Dr Nigel Blackburn, director of drug development at Cancer Research UK’s Drug Development Office, said: “It’s incredibly encouraging to launch a trial of this promising drug which is personalised to target the different genetic make-up of patients with breast and ovarian cancer. Providing the drug as a tablet will give patients a much improved quality of life.

“This drug was developed through work led by Cancer Research UK scientists and we’re continuing to invest further in targeted drugs like this. We hope that this new treatment approach will help extend the lives of women with breast and ovarian cancer. We look forward to the results with great interest.”


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The drug Rucaparib (CP-338; formerly called AG-014699 or PF-01367338) was recently licenced to Clovis Oncology from Pfizer.

About PARP inhibitors
PARP inhibitors block PARP, a protein which is part of DNA’s ’emergency repair kit’ in cells – it prevents mistakes being passed on when cells grow and divide. An alternative ‘repair kit’ is also controlled by the BRCA1 and BRCA2 genes – these genes are faulty in some cancer cells. When both copies of the BRCA1 or BRCA2 genes are faulty, the cells rely on the PARP pathway to repair damaged DNA. By blocking PARP with drugs, cancer cells which have lost BRCA1 or BRCA2 can no longer repair DNA damage and they die. This is why PARP inhibitors are effective in treating the small percentage of cancers in which BRCA1 or BRCA2 is faulty.
Because cancer treatments such as radiotherapy and chemotherapy kill cells by damaging DNA, PARP inhibitors also have the potential to increase the effectiveness of some of these treatments, even in cancers that do not have mutations in BRCA1 or BRCA2.