Potential new drug shows promise in multiple myeloma

A potential new drug for patients with multiple myeloma who have failed to respond to several rounds of existing treatments has shown promise in an early phase trial at UCLH.

The trial at the NIHR UCLH Clinical Research Facility (CRF) looked at the combination of the study drug mezigdomide when given with the existing drug dexamethasone.

The trial was the first time mezigdomide had been administered to patients with multiple myeloma.

Overall, 41% of patients responded to the drug, for an average of four and a half months, and the trial indicated that the drug had a good overall safety profile at the doses given to patients.

While the results of this early stage trial are promising, the research team will continue to examine the safety of the drug, and how well it works, in upcoming research.

Dr Rakesh Popat, Consultant Haematologist and Lead for Cancer trials at the UCLH CRF, was one of the investigators supporting the international trial which has been published in the New England Journal of Medicine.

Despite recent advances in therapy, multiple myeloma remains incurable and new treatments are required. The study recruited patients whose disease had returned following at least 3 previous lines of treatment that included the main classes of drugs used to treat myeloma and did not respond to their most recent treatment.

The study was in two phases.  Phase I assessed the safety and identified the effective dose of the study drug, while the aims of phase 2 included assessing the effectiveness and the safety of mezigdomide plus dexamethasone at the dose determined in phase 1. 77 patients were enrolled into the phase 1 part of the study and 101 to phase 2. Dr Popat recruited 11 patients onto the trial, the largest contribution from the UK.

Mezigdomide belongs to a group of drugs known as cereblon modifying agents (CELMoD agents). The drug binds to a protein called cereblon, a type of protein in multiple myeloma cells which helps them function and multiply. In laboratory studies, mezigdomide has been shown to stimulate normal immune cells in the body causing them to attack and kill myeloma cells.

Dexamethasone is a steroid already used in the treatment of multiple myeloma as it triggers the destruction of myeloma cells. The study investigated the safety and dosage of the investigational drug alone and in combination with dexamethasone

Dr Popat said: “It is exciting to be part of the development of this drug. Further Phase 3 trials combining it with standard treatments are underway as well as investigating it with other novel agents in phase 1 trials. I hope that mezigdomide will soon become a treatment option that we can start offering to patients.”

The study was funded and sponsored by Celgene Corporation.

For information on how to participate in trials at UCLH visit Find a Study or the early phase cancer trial web pages on the UCL website.