Adding a new once-daily pill to a combination of drugs already used on the NHS could help patients with a with an incurable blood cancer live longer without the disease getting worse, according to researchers.
A trial found that patients on the triplet therapy – which included the new treatment mezigdomide – lived more than twice as long without their cancer progressing.
About 6,200 people every year in the UK are diagnosed with multiple myeloma, a type of blood cancer that develops from abnormal plasma cells in bone marrow.
In patients with relapsed or refractory multiple myeloma, the cancer has either returned after remission or stopped responding to treatment.
Mezigdomide works by attaching to a specific protein in the body.
It then acts like a magnet, attracting disease-causing proteins that are vital for survival of multiple myeloma cells and degrading them.
By wiping out these cancer-promoting proteins, the drug also stimulates the immune system to attack and kill remaining cancer cells.
The trial by researchers in the US, which is being presented at the American Society of Clinical Oncology annual meeting in Chicago, included 479 people with relapsed or refractory multiple myeloma.
Some 288 patients were given the new triplet drug of mezigdomide, carfilzomib, and dexamethasone, while 191 had 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 group given the two-drug combination.
The improvement was also seen across different sub-groups, including patients with more aggressive cancer.
The study also found that around eight in 10 of the mezigdomide patients responded to treatment compared to a little over half (53.4 per cent) in the other group.
Among patients in the mezigdomide group, more than a quarter (26.7%) had no evidence of disease, compared to only 8.9 per cent of patients on just carfilzomib and dexamethasone.
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.
“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.”
Currently in England, patients with multiple myeloma can have carfilzomib plus lenalidomide and dexamethasone on the NHS if they have had only one previous therapy which included the drug bortezomib.
The National Institute for Health and Care Excellence is currently assessing if mezigdomide with dexamethasone and carfilzomib can be used on the the health service for patients with relapsed or refractory multiple myeloma after at least one line of treatment.
