New hope for some advanced bowel cancer patients, after NICE approves drug on the NHS

A new bowel cancer drug, which can double progression-free survival time for some people with advanced bowel cancer, has been approved by the National Institute for Health and Care Excellence (NICE) for patients in England and Wales.

Pembrolizumab will now be available on the NHS as a first line treatment for people with specific genetic changes, known as 'high microsatellite instability or mismatch repair deficiency'. People with advanced bowel cancer – where the disease has spread to other parts of the body – are normally treated with chemotherapy, sometimes in combination with other treatments. But around 5% of advanced bowel cancer patients have changes to their mismatch repair genes, which can result in cancers with high microsatellite instability (MSI-H) or mismatch repair deficiency (dMMR). This also includes some people with Lynch syndrome. This makes them less likely to respond well to current treatments, often leading to poorer outcomes.

International clinical trials showed the new treatment can increase the amount of time patients live with the disease without it getting worse from an average of 8.2 months for those who were given chemotherapy, to 16.5 months on average for people treated with pembrolizumab. Scientists also found that 11% of patients treated with pembrolizumab had a 'complete response' where their disease became completely undetectable on scans.

Genevieve Edwards, Chief Executive of Bowel Cancer UK, says: "This is heartening news for this small, but sadly often overlooked group of advanced bowel cancer patients in England and Wales. Pembrolizumab may stop the disease getting worse for a number of valuable months, and will bring hope to those with high microsatellite instability or mismatch repair deficiency."

While there continues to be very few treatment options for people with advanced bowel cancer, NICE has also recently approved a combination of two drugs – encorafenib (BRAFTOVI®) and cetuximab (Erbitux®) – for those with a BRAF V600E mutation. This combination can be used as a life-extending treatment with patients who have had previous treatment.

Around 10,000 people in the UK are diagnosed at the latest stage of the disease each year. Those with advanced bowel cancer typically have poorer survival rates, but access to surgery and drugs can help to extend life.

Pembrolizumab (also known as Keytruda®), is an immunotherapy, which works by helping the immune system to recognise and attack cancer cells.

Hayley Crawley was diagnosed with stage 4 bowel cancer in 2014, aged 46: "My family's lives were completely turned upside down after the end-of-life palliative conversation I had with my medical team. For me it became a grieving process for the life I had lived and already treasured. I mourned for my past. I was alive but not really living. Unable to create happy memories with my husband and 8-year-old son. Sports days, school plays and parents' evenings became significant risks, as I was immune-suppressed and very weak. I became the 'absent parent'. Slowly deteriorating too, during the relentless chemotherapy cycles.

"I promised my husband and son that I would never give up while treatments showed promise, but survival was a hugely daunting daily burden. We clung tightly together as a family, cast adrift and waiting for the next storm to hit, knowing it might be my end.

"By 2017, because of our relentless personal research, I had it confirmed that I was MSI high and knew that pembrolizumab had proven effective for this rare sub-type of bowel cancer. Cruelly, however, this potential lifesaver was only available on the NHS for a very limited range of cancers. Alone we could not afford the significant cost of a full course of treatment. The inequality in access hit me with full force, as I was aware of patients with the 'right' cancers receiving the drug for free in the NHS unit I was getting my chemotherapy in.

"I calculated that the cost of the immunotherapy worked out at about £240 per day. Was my life not worth this? I was feeling so desperate, yet precious hope emerged when we decided to take on the monumental challenge of crowdfunding. The response was beyond our wildest expectations. Family, friends, former NHS colleagues, parents and pupils at my son's school and strangers together contributed over £110,000.

"A local consultant agreed to provide me with the immunotherapy, privately funded by these generous crowdfunders. I was lucky to experience no side effects from the immunotherapy and wonderfully I have had 'no evidence of disease' since 2018. I am living a full life, including giving back to other patients where I can, personally and with a number of organisations. I long for others who could benefit to have access to this life saving treatment.

"I owe my life to immunotherapy but only got access because of the generosity of crowdfunding. I feel privileged to support this game-changing treatment for people enduring a similar diagnosis to me through my campaigning work as a patient representative with Bowel Cancer UK. Potentially debilitating treatments will be avoided, with far fewer side effects and with the possibility for years of an improved quality of life."

Mo Haque was diagnosed with advanced bowel cancer in 2014, aged 31. "Living with stage 4 bowel cancer is devastating and instantaneously life changing. I couldn't go into work, I lost my independence and leant on others for the very basics. My everyday routine was completely overhauled. My life was all about hospitals, appointments, procedures, whilst feeling sick, fatigued, and in lot of physical, psychological and emotional pain. My family were left helpless.

"After two lines of chemo failed over the course of a year, there was nothing else the NHS could offer. This was an unbearable to hear, especially so because pembrolizumab shows an incredible response rate for people with this condition.

"My only option was raising £200,000 to have immunotherapy. We raised £100,000 in two months – this meant I had enough money to start the treatment, which I did in June 2016. Five years later I have no evidence of the disease. This extra life has allowed me to spend quality and meaningful time with my friends and family. I have been able to publish my book Choosing To Stay, been invited to speak at events and medical conferences, have helped other patients with their care, and I'm on a number of cancer related advisory groups.

"This is a lifesaving decision, which will literally mean the world. It will give some stage 4 bowel cancer patients the chance to spend quality time with loved ones which they otherwise wouldn't get."

Hayley and Mo both kindly shared their stories as patient representatives at the NICE committee. By sharing their experiences, and answering questions from the committee, it helped the members to understand the patient perspective of accessing pembrolizumab. At this stage, Hayley and Mo have no evidence of disease, and are no longer on this treatment.

• Read more about pembrolizumab and how we were involved in our blog
• Find out more about our Get Personal campaign, aiming to improve access to treatment and quality of life for those with advanced bowel cancer
• Learn more about advanced bowel cancer
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