A new treatment offers hope for UK myeloma patients. The drug, mezigdomide, is an oral therapy designed specifically for those with relapsed or refractory multiple myeloma — a form of blood cancer that has either returned after a period of remission or stopped responding to existing treatments. The National Institute for Health and Care Excellence (NICE) is currently assessing mezigdomide in combination with dexamethasone and carfilzomib for use on the NHS in England, after at least one prior line of therapy.
How the new treatment works
Mezigdomide works through a novel mechanism that targets the disease at a molecular level. It attaches to a specific protein inside the body, acting like a molecular “magnet” to draw in and degrade other disease-causing proteins that are essential for myeloma cell survival. At the same time, the drug stimulates the immune system to attack and destroy any remaining cancer cells. This dual action makes it particularly suited to patients whose disease has become resistant to earlier treatments.
Recent clinical trial results have shown a significant benefit for patients receiving mezigdomide as part of a three-drug combination. Those given mezigdomide together with carfilzomib and dexamethasone lived for a median of 18 months without their disease progressing, compared to 8.3 months for patients who received only carfilzomib and dexamethasone — more than double the progression‑free survival time. Around eight in ten patients in the mezigdomide group responded to treatment, compared to just over half (53.4%) in the control group. More than a quarter (26.7%) of patients in the mezigdomide group achieved no evidence of disease, versus 8.9% in the other group.
Understanding multiple myeloma
Multiple myeloma is an incurable blood cancer that originates from abnormal plasma cells in the bone marrow. It affects approximately 6,200 people in the UK each year, according to the original source, while Cancer Research UK estimates around 6,500 new cases annually, making it the 19th most common cancer in the country. Incidence rates are highest in people aged 75 and over, with a significant proportion diagnosed in the 85–89 age group. Rates have been increasing in the UK since the early 1990s. The disease is slightly more common in men than women and is two to three times more common in Black people, who are also more likely to be diagnosed at a younger age.
Patients often experience periods of remission followed by relapse. Common symptoms include bone pain — particularly in the back, hips and ribs — fatigue, recurring infections, bone fractures, anaemia, elevated blood calcium levels and kidney problems. Diagnosis can sometimes be delayed because the symptoms are non-specific, and some patients report that their early complaints were trivialised by GPs who may lack expertise in diagnosing rare cancers. While there is no cure, treatments aim to manage symptoms and achieve remission; 37.8% of patients survive for ten or more years in the UK.
Patient group and other advances
The new mezigdomide combination is aimed specifically at patients with relapsed or refractory multiple myeloma — those whose cancer has returned after remission or has stopped responding to previous therapies. This group faces limited options and often a poorer prognosis, making new treatments particularly valuable.
Mezigdomide is not the only novel therapy making headway. The antibody‑drug conjugate belantamab mafodotin — described as a ‘trojan horse’ therapy — targets myeloma cells expressing the BCMA protein, delivering chemotherapy directly to the cancer cells and flagging them for the immune system to destroy. The NHS in England was the first health system globally to roll out this drug as a targeted therapy for blood cancer patients. It is now available on the NHS in England and Wales as a second‑line treatment, often in combination with bortezomib and dexamethasone (BorDex). Clinical trials showed that in a broader group of relapsed or refractory patients, belantamab mafodotin (with bortezomib and dexamethasone) delayed disease progression by an average of three years, compared to just over a year for patients taking daratumumab with bortezomib and dexamethasone. In Scotland, this combination (BVD) has been approved on the NHS and shown to stop myeloma in its tracks for over 36 months on average. Around 1,500 patients a year in England could be offered belantamab mafodotin, and NICE is considering widening access to patients who have not had lenalidomide before. Paul Silvester, from Sheffield, who received the drug via an early access programme, reported being in remission within weeks.
Another innovative treatment is talquetamab, a bispecific antibody that links T cells — the immune system’s killer cells — to myeloma cells, helping the immune system find and destroy cancer cells. It targets the GPRC5D protein on myeloma cells and the CD3 protein on T cells. Talquetamab is available on the NHS in England and Wales for patients who have received at least three previous treatments, including an immunomodulatory agent, a proteasome inhibitor and an anti‑CD38 antibody. It has been shown to keep myeloma at bay for over 11 months on average, offering flexibility and respite between doses.
Daratumumab, which targets a molecule on the surface of myeloma cells, is also widely used. Its subcutaneous formulation Darzalex Faspro was the first CD38‑directed antibody approved for multiple myeloma. In combination with bortezomib and dexamethasone, daratumumab is available on the NHS in England for patients whose myeloma has worsened or returned after their first round of treatment. In trials, 61% of patients on the combination were alive for one year without progression, compared to 27% on bortezomib and dexamethasone alone. Darzalex Faspro has also been approved in the US for adults with high‑risk smoldering multiple myeloma.
For many patients, the psychological impact of a myeloma diagnosis is profound. Support groups such as the West Lancs & Merseyside Myeloma Support Group and Myeloma UK offer valuable resources for sharing experiences and information. Shared decision‑making between patients and clinicians is essential at each stage of the disease, and managing symptoms like pain and fatigue is a learned process that prioritises maintaining quality of life, even with ongoing treatment.
