A bowel cancer treatment that offers hope to people suffering from the same aggressive form that affected Dame Deborah James has been approved for use on the NHS.
The drug combination – two drugs called trifluridine-tipiracil hydrochloride and bevacizumab – is being introduced following a US trial that found it gave patients who had run out of treatment options almost a year more to live.
And in some rare cases, it gave patients up to seven more years.
Experts say this is an important step in the fight against the BRAF V6004E mutation, an incurable genetic form of bowel cancer that often affects young people.
Dame Deborah James had the aggressive BRAF mutation. Under the name Bowelbabe, the deputy headmistress managed to build up a huge following on social media.
Blood sample taken as part of testing for the mutation. Most patients do not live more than ten months after diagnosis.
Of the 42,000 Britons diagnosed with bowel cancer each year, around one in ten is a carrier of the BRAF mutation. Chemotherapy and other drugs quickly lose their effectiveness and most patients do not live more than ten months after diagnosis.
Dame Deborah was just 35 in 2016 when she was diagnosed with BRAF bowel cancer. Under the name Bowelbabe, the deputy headmistress built up a huge following on social media as she documented her progress and raised awareness of the disease and its symptoms until her death in 2022.
The drug combination has been available privately in the UK for eight years, at a cost of around £2,000 a month.
However, last month the NHS spending watchdog, the National Institute for Health and Care Excellence (NICE), gave the green light to the move for patients who have already undergone two failed treatments. It is expected to benefit around 1,000 people a year.
“Many patients have been on this regime for years privately, slowing the development of their cancer,” says Anna Bennett, from the patient group Breaking BRAF, which campaigns for new treatments.
“Some patients have achieved stability that lasted more than one or two years, and we know of one patient who lived almost seven more years. We are delighted with this decision.”
Professor Marco Gerlinger, head of gastrointestinal cancer medicine at Barts Cancer Institute in London, said the approval of the drug combination was a “fantastic step forward”.
“For the last ten years there hasn’t been a very effective drug for this type of cancer,” he added. “But now we have a really great option for patients.”
The BRAF mutation causes cancers to grow faster than they would otherwise. Because of this, people who carry it often develop symptoms at an early age.
The mutation is also linked to skin and brain cancer, and appears to be more common in women.
BRAF cancers quickly become resistant to treatment, meaning patients require different interventions to keep them alive.
During her six-year battle with bowel cancer, Dame Deborah underwent more than a dozen surgeries, 100 rounds of chemotherapy and more than ten targeted drug therapies.
Trifluridine-tipiracil hydrochloride, also known as Lonsurf, is a daily tablet that stops the growth of new cancer cells. Bevacizumab, also known as Avastin, works by blocking the formation of new blood vessels that feed tumors and allow them to grow. It is given intravenously every two weeks in the hospital.
Both medications are taken as long as they remain effective.
The trial, published in the New England Journal of Medicine last year, found that the combination provided an average of 10 months more survival, but many patients lived up to a year and a half longer. The drugs also caused few serious side effects, with nausea and vomiting being the most common.
While patient groups welcomed NICE’s decision to fund the treatment, they criticised the delay which led to many desperate patients turning to private services.
“It’s unforgivable that we’ve reached a situation where so many patients have died without access to a drug that could have given them months, if not years, more to live,” says Ms Bennett, whose sister Rebecca died of bowel cancer.
Mother-of-two Emily Georghiou, 47, from London, was diagnosed with bowel cancer in 2021
“People now have hope when before they were faced with the horrible choice of living or going into debt.”
One patient who could benefit from this combination is Emily Georghiou, a 47-year-old mother of two from London, who was diagnosed with the disease in 2021.
“Ever since chemotherapy failed and I discovered I have the aggressive BRAF mutation, I have felt like a ticking time bomb,” says Emily, a former health policy adviser. “We have no money or private insurance to pay for the drugs, and although I started a fundraising campaign, I knew that sooner or later I would run out of money to stay alive.
‘When you have this condition, you live from one scan to another; at any moment your condition can change.
‘This announcement gives me hope, because if that moment comes, I now know it will be there.
“It’s incredibly important to be able to be there for my beautiful children as much as possible.”
The Breaking BRAF patient group hopes that another promising drug combination, botensilimab and balstilimab, will be approved in the near future. It also argues that it is vital to remind GPs of the signs of bowel cancer in young people, such as blood in the stool, unexpected weight loss, fatigue or a feeling that the bowel is not emptying completely.
“Many patients, including my sister, were dismissed by GPs until it became an emergency,” says Ms Bennett. “As bowel cancer is becoming more common among young people, it is vital that doctors are aware of the early symptoms.”