The NHS in England will be the first health system in the world to roll-out a ‘trojan horse’ targeted therapy for blood cancer patients which could halt the disease’s progression by nearly three times as long as existing treatments. 

Around 1,500 patients a year with multiple myeloma – an incurable cancer of the bone marrow – could now be offered belantamab mafodotin on the NHS in England, after it was approved by the National Institute for Health and Care Excellence (NICE) yesterday [12 June].

The drug will be offered to eligible patients whose cancer has progressed or not responded to first-line treatment with another drug, lenalidomide.

Belantamab mafadotin – which was research and developed in the UK – is an antibody-drug conjugate, a type of treatment which targets and attaches to cancer cells.

The drugs have been described by researchers as ‘trojan horses’ as they are designed to be taken up by the cancer cell, before releasing a high concentration of a lethal molecule to destroy the cell from within.

Trials showed that in a broader group of patients with relapsed or refractory multiple myeloma, belantamab mafodotin (in combination with bortezomib and dexamethasone) delayed progression of the disease by an average of three years, compared to just over a year for patients taking commonly-used drug daratumumab (with bortezomib and dexamethasone).

Progression-free survival data for the narrower group of patients for whom the drug is being recommended by NICE is not publicly available.

NHS England is fast-tracking access to the treatment for patients from today, through immediate funding via the Cancer Drugs Fund.

Eligible patients will be treated via an infusion every three weeks in combination with other cancer drugs, bortezomib (injection) and dexamethasone (orally).

Paul Silvester, 60, from Sheffield, was diagnosed with myeloma in July 2023 after his cancer caused broken bones in his back and a tumour in his spine. After initial treatment failed to stop his cancer’s progression, he received belantamab mafadotin via an early access programme and was in remission within weeks. Paul was treated at the Royal Hallamshire Hospital.

He said: “I feel like this treatment has brought the party balloons back in the house. It has been amazing – within the first two or three weeks, after the first dose, I was in remission.

“It gives me quite a lot of confidence in the drugs and it makes me more optimistic about the future. I’ve been feeling well and I’m still quite active – that’s what’s important in terms of your quality of life. One of my daughters is graduating from university in October and it’s a goal for me to be there.”

Multiple myeloma is a type of bone marrow cancer than often affects multiple parts of the body, including the spine, skull, pelvis and ribs. It is more common in men than women, adults over 60, those with a family history of the condition and is twice as common in black populations than white and Asian populations.

Myeloma cannot be cured, and patients often experience multiple relapses – treatment is usually about halting the cancer for as long as possible with the least side-effects from treatment. The disease can have a significant impact on quality of life, with the possibility of relapse having a major psychological effect on patients.

Each year, more than 6,000 people are diagnosed with multiple myeloma in the UK, and it is estimated there are around 33,000 people living with the cancer.

Professor Peter Johnson, NHS England’s National Clinical Director for Cancer, said: “Myeloma is an aggressive type of blood cancer, but we have seen a steady improvement in the outlook for patients over recent years as we have introduced new targeted therapies.

“I am delighted that patients in England will be the first to benefit from this new treatment, which has the potential to keep cancer at bay for years longer, giving people the chance of more precious time with friends and family.

“This treatment could be life-changing for many patients and their families,  and that’s why it is so important that the NHS continues to secure quick access to the latest, innovative treatments like this, at affordable prices to the taxpayer.”

Health Minister, Karin Smyth, said: “This groundbreaking therapy puts the NHS at the forefront of cancer innovation. By harnessing cutting-edge ‘trojan horse’ technology, we’re offering new hope to blood cancer patients across the country.

“We’re determined to back scientific breakthroughs that deliver real results for patients – bringing the latest treatments from the lab to those who need them most. It’s another example of how we’re building an NHS fit for the future, one that embraces medical innovation to transform patient care.”

Shelagh McKinlay, Director of Research and Advocacy at blood cancer charity Myeloma UK, said: “It’s fantastic to see the UK at the forefront of myeloma treatment. NHS England has demonstrated that it is possible for myeloma patients to have world-first access to innovative drugs.

“We have been working very hard for the last year to get this treatment approved and we know it will transform the lives of thousands of people with myeloma.”

Antoine Herbaux, Vice President, Head of Oncology UK, GSK, said: “We are delighted to have worked with NHS England and NICE on this significant recommendation in helping support unmet need in multiple myeloma.

“Today’s announcement highlights an example of local innovation – Belantamab mafodotin was partly discovered in Stevenage, the first patient to receive it in clinical trials was in London, and now the UK is the first country to grant patient access. This milestone is a great example of the power of scientific innovation and open collaboration to achieve positive outcomes for patients in the UK.”

Patients must have routine ophthalmological assessments before starting treatment and after each of the first three treatments. Side effects can include ocular toxicity, difficulty seeing clearly, blurred vision, dry eyes and photophobia.

Cutting-edge treatments like belantamab mafodotin have been enabled by the country’s thriving life sciences sector and the largest cluster of cell and gene therapy companies outside the US, centred around Stevenage and extending to Oxford, London, and Cambridge.