A milestone 3,000 participants have been recruited to a clinical trial led by UCL and UCLH, which is aimed at reducing the number of patients with breast cancer who receive unnecessary chemotherapy.
The OPTIMA trial (Optimal Personalised Treatment of early breast cancer using Multi-parameter Analysis) is looking at use of a molecular test to determine which patients need chemotherapy.
The recruitment milestone - reached during Breast Cancer Awareness month in October - represents two-thirds of the target recruitment for OPTIMA, whose Chief Investigator is Professor Rob Stein (UCL Cancer Institute).
Breast cancer treatment has become increasingly successful in recent years, thanks to the routine use of hormone treatment and chemotherapy after surgery.
However, clinicians are concerned that many patients with the common hormone-driven type of cancer don’t benefit from chemotherapy and are being exposed to needless treatment with unpleasant side effects. The clinician’s dilemma is knowing who could benefit from chemotherapy and who can be safely spared.
The OPTIMA trial looks at using a gene test performed on surgically-removed tumour tissue to identify patients who have tumours which will respond to chemotherapy. These so-called multi-parameter tests predict the risk of cancer recurrence by measuring the activity of up to 100 genes known to be important in breast cancer.
Three multi-parameter tests - including one called Prosigna - are currently recommended for some NHS patients. The National Institute for Health and Care Excellence (NICE) considers use of the tests may be effective for patients whose cancer has spread to under-arm (axillary) lymph nodes, and has recommended more research in this area, which is what the OPTIMA study is doing.
The test used in the OPTIMA study is Prosigna and the trial is for patients with hormone sensitive breast cancer who have completed their surgery. The participants mostly have cancer spread to under-arm (axillary) lymph nodes which places them at risk of future recurrence. Because of this they are routinely offered chemotherapy in addition to five or more years of hormone therapy.
Although NICE has allowed testing for patients without lymph node involvement, there is little evidence to support their use for large tumours.
Patients who consent to the trial are randomised to this current standard management (Group 1) or to have a Prosigna test performed on their tumour tissue (Group 2). Those with a high tumour Prosigna test score are treated with chemotherapy and hormone therapy as in Group 1 whilst those with a low score tumour are treated with hormone therapy alone. Patients who receive chemotherapy will not know if they are in Group 1 or Group 2. All other treatments such as radiotherapy are given as usual.
OPTIMA will compare the number of patients whose cancer returns within five years according to how their treatment was decided. The trial hypothesis is that there will be no more cancer recurrence with test-directed treatment (Group 2) compared with standard management. If this can be demonstrated, OPTIMA will have shown that it is safe to use multi-parameter tests to make decisions about chemotherapy in a higher-risk "node-positive" population.
So far about two thirds of patients tested in the trial have avoided chemotherapy.
Professor Stein said: "If we find that we can use tests like Prosigna to determine who can be spared chemotherapy, this will be of huge benefit to patients and will mean treatment is more targeted and personalised.
"We expect the trial results to be applicable to about 10% of women diagnosed with breast cancer annually in the UK - around 5,600 to 5,800 people annually.
"We are very pleased to reach the milestone of 3,000 participants and we thank all patients who have taken part in the trial so far."
OPTIMA is funded through the National Institute for Health Research (NIHR) Health Technology Assessment Programme.
The study is expected to complete recruitment at the end of 2024.
Harriet Quilty was diagnosed with breast cancer in 2014, at which point her specialist thought the cancer was very aggressive. She had a tumour in her left breast as well as swelling under-arm.
She was one of the first patients to get involved in the trial after her oncologist told her about it.
Harriet, who is an illustrator, said: "When you have cancer it is all consuming and it is hard to think about anything else. I was told my cancer was very aggressive and likely to come back again. The future looked grim. Oncologists were worried.
"The worst part for me was that I was going to have chemo. I really didn’t want to go through chemotherapy as I was worried about possible side effects.
"My oncologist told me about the trial, and explained that by taking part there was a small chance I may not have chemotherapy.
"I decided to take part, and a few days before I was expecting to go in for chemotherapy, I found out I wouldn’t be having it. I was so relieved!"
Harriet had radiotherapy and other medication, and is now in remission from breast cancer.
She originally started her treatment at Addenbrooke’s Hospital in Cambridge after a move to Yorkshire to live in the countryside, and her follow up care is now with The Christie NHS Foundation Trust in Manchester.