A new once-daily pill, when added to an existing drug combination used on the NHS, could help patients with an incurable blood cancer live longer without their disease worsening, according to researchers. The trial found that patients on the triplet therapy, which included the novel treatment mezigdomide, lived more than twice as long without their cancer progressing compared to those on a standard two-drug regimen.
Understanding multiple myeloma
Approximately 6,200 people in the UK are diagnosed with multiple myeloma each year. This type of blood cancer develops from abnormal plasma cells in the bone marrow. In patients with relapsed or refractory multiple myeloma, the cancer has either returned after a period of remission or has stopped responding to treatment.
How mezigdomide works
Mezigdomide operates by attaching to a specific protein in the body. It acts like a magnet, attracting disease-causing proteins that are vital for the survival of multiple myeloma cells and degrading them. By eliminating these cancer-promoting proteins, the drug also stimulates the immune system to attack and kill remaining cancer cells.
Trial details and results
The trial, conducted by researchers in the US and presented at the American Society of Clinical Oncology annual meeting in Chicago, included 479 people with relapsed or refractory multiple myeloma. Some 288 patients received the new triplet combination of mezigdomide, carfilzomib, and dexamethasone, while 191 received only carfilzomib and dexamethasone.
After a follow-up period, those in the mezigdomide group lived for a median of 18 months without their disease progressing, compared to 8.3 months in the two-drug group. This improvement was observed across various sub-groups, including patients with more aggressive forms of cancer.
The study also found that approximately eight in ten (80%) of the mezigdomide patients responded to treatment, compared to just over half (53.4%) in the other group. Among those receiving mezigdomide, more than a quarter (26.7%) had no evidence of disease, compared to only 8.9% of patients on carfilzomib and dexamethasone alone.
Expert commentary
Paul Richardson, director of clinical research at the Jerome Lipper Multiple Myeloma Centre at Dana-Farber and the R J Corman Professor of Medicine at Harvard Medical School, said: “There are relatively few accessible therapeutic options for patients whose multiple myeloma has returned or stopped responding to current standard treatments. These results support mezigdomide, a potent and novel oral therapy, as a new standard of care for relapsed or refractory multiple myeloma across multiple settings.”
He added: “There are several pivotal studies with mezigdomide in relapsed or refractory multiple myeloma exploring its use in combination with other standard treatments, such as bortezomib and dexamethasone, to confirm its value in real-world practice, as well as providing a broad platform for regulatory approval. In addition, its ability to reverse immune exhaustion and enhance immune activity makes it an ideal partner to current immunotherapies as well as other novel oral agents, and numerous trials are now under way.”
Current NHS availability and next steps
Currently in England, patients with multiple myeloma can receive carfilzomib plus lenalidomide and dexamethasone on the NHS if they have had only one previous therapy that included the drug bortezomib. The National Institute for Health and Care Excellence (NICE) is currently assessing whether mezigdomide, in combination with dexamethasone and carfilzomib, can be used on the health service for patients with relapsed or refractory multiple myeloma after at least one line of treatment.
