A new drug for multiple myeloma patients

The Lancet Oncologypublished findings that, in resistant or recurrent multiple myeloma, Pomalidomide (Pomalyst®) plus low-dose dexamethasone substantially prolongs progression-free and overall survival compared with high-dose dexamethasone alone.

Multiple myeloma develops from plasma cells, a type of white blood cell in the immune system. Patients with multiple myeloma have large numbers of abnormal plasma cells that increase the number of dysfunctional antibodies detectable in the urine and/or blood. To date, patients with resistant or recurrent multiple myeloma have had limited treatment options, including lenalidomide (Revlimid®) and bortezomib (Velcade®). There is no standard treatment for these patients, the course of the disease is unfavorable, and average survival is less than 1 year.

Pomalidomide; is an immunomodulatory drug that regulates the immune system and has shown effectiveness in patients with multiple myeloma resistant to bortezomib (Velcade®), thalidomide (Thalomid ®), and lenalidomide (Revlimid®). Pomalidomide directly inhibits angiogenesis (the formation of blood vessels) and myeloma cell growth. Given orally as a tablet, pomalidomide supports the body's immune system in destroying cancer cells and preventing their growth. This treatment is intended for patients with multiple myeloma who have received at least two previous treatments, failed to respond to them, and experienced disease progression within 60 days after the last treatment (patients with resistant and recurrent cancer).

Pomalidomide alone showed limited activity in patients with recurrent multiple myeloma, but its synergistic effect when combined with dexamethasone was notable. Australia, Canada, Europe, Russia and Americaincluded a total of 455 patients with resistant/recurrent multiple myeloma who had received at least two previous treatments with bortezomib and lenalidomide. Randomly, 302 patients received pomalidomide (orally on days 1 and 21, at 4mg daily) plus low-dose dexamethasone (orally on days 1, 8, 15, and 22, at 40mg daily), while the other 153 received high-dose dexamethasone (orally on days 1-4,9-12, and 17-20, at 40mg daily). After 10 months of observation, average progression-free survival was determined to be 4 months in patients using pomalidomide and low-dose dexamethasone, compared with 1.9 months in the control group. Subgroup analysis showed that average progression-free survival was longer in patients using pomalidomide than with their previous treatment. Average overall survival was 12.7 months in patients using pomalidomide and 8.1 months in the other group. Treatment response was %31 in patients receiving pomalidomide/low-dose dexamethasone, whereas it remained around %10 in patients using high-dose dexamethasone alone.

Effects were more frequent with pomalidomide but manageable in both groups.

These promising research findings showed that the combination of pomalidomide and additional low-dose dexamethasone should be considered a new treatment option for patients with recurrent/resistant multiple myeloma.