Breakthrough trial involving experts in West Yorkshire helping people with blood cancer live longer
A major cancer study, led specialists including from the University of Leeds is offering new hope for people with tough-to-treat blood cancers.
Last updated 9 hours ago
A pensioner given two years to live after being diagnosed with aggressive blood cancer is still alive eight years on after signing up for a trial investigating precision treatment for high-risk patients.
Ken Theobold, 76, said he is able to enjoy spending time in his motorhome and being with his grandchildren after being given drugs tailored to his disease in the new trial.
The study, led by experts at The Institute of Cancer Research (ICR), London, involved 107 people with newly diagnosed high-risk multiple myeloma – previously known as ultra-high-risk myeloma.
Patients were given state-of-the-art diagnostics and tailored treatment using a range of drugs.
Long-term follow-up data has now shown that patients live significantly longer when their treatment is adapted to the molecular biology of their disease, the ICR said.
The ICR, along with experts from the University of Leeds, The Royal Marsden NHS Foundation Trust and hospitals across the UK, treated patients with a stem cell transplant and a combination of five medicines already used in the NHS – daratumumab, cyclophosphamide, bortezomib, lenalidomide and dexamethasone.
But patients were given these drugs in a different way, with intense treatment at the start and continuing combination therapy for as long as the disease remained controlled, the ICR said.
Mr Theobold, who was diagnosed with high-risk myeloma in 2018 after a routine blood test, was referred to The Royal Marsden and has been treated on the MUK Nine trial for the last eight years.
He was given five drugs at the start of the trial and now remains on two.
“After my diagnosis, I was told that I might only have two years to live, which was incredibly difficult to hear,” he said.
“Joining the trial was an absolute no-brainer. If there was a chance it could help me and help future patients, I wanted to be part of it.
“When I first joined the trial, I was taking five different drugs, which was quite intense but I have since moved to the maintenance part of the trial which only involves two drugs.
“Today, I’m able to enjoy life with my wife, Brenda. We love travelling in our motorhome, and spending time with our five grandchildren is what matters most.”
Long-term results from the trial have just been published in the journal Lancet Oncology.
The authors of the paper said the personalised therapy “shows sustained, progression-free survival and overall survival improvement supporting implementation of molecular diagnostics and tailored treatment in patients with newly diagnosed high-risk multiple myeloma”.
At around six years of follow-up, 70% of patients who took part in the Optimum trial were still alive, compared with around 40% on standard therapy.
Some 54% of patients on the trial remained progression-free at six years, compared with 18% of patients who received usual care.
First author Martin Kaiser, professor of molecular haematology at the ICR and consultant haematologist at The Royal Marsden NHS Foundation Trust, said: “High-risk myeloma has traditionally been one of the toughest challenges we face, with patients often relapsing early despite the best available treatments.
“These long-term results show that when we adapt treatment to the biology of the disease, we can significantly extend survival for many patients who previously had very limited options.”
Researchers also examined whether the personalised approach benefitted people with subgroups of the disease.
They used a test called MMProfiler SKY92, which identified people as high-risk by their gene expression.
This gene expression profiling is not routinely available in the NHS, meaning patients who may be high-risk may not be identified as such, the ICR said.
The study found that 62% of this subgroup of patients who were given the personalised risk-adapted treatment were still alive and progression-free at six years compared with 20% of patients who had received conventional treatment.
The ICR said that the test is being evaluated by health regulators for potential use across the health service.
Prof Kaiser said: “This study also shows that some patients with aggressive disease are currently being missed because the necessary molecular tests are not routinely available. Identifying these patients earlier could allow us to tailor treatment from the start and change the course of their disease.”
ICR chief executive Professor Kristian Helin said: “These results are a powerful example of how understanding the biology of cancer can transform patient outcomes.
“By matching treatment to the molecular features of each person’s disease, we are beginning to outsmart cancers that were once extremely difficult to treat – and high-risk myeloma has long been one of the hardest to treat effectively.”
Dr Sophie Castell, chief executive at blood cancer charity Myeloma UK, said: “Identifying which patients are more likely to benefit from a personalised approach to treatment has been shown to keep myeloma at bay longer and give people a better chance to live well with their disease.”
Around 5,900 people are diagnosed with multiple myeloma – a cancer of the plasma cells – each year in the UK. Around a quarter of these have aggressive disease which responds poorly to conventional treatment.