By Raymond Thertulian, MD.
March is Multiple Myeloma (MM) Month! It is the month chosen to celebrate the courage of MM patients and their caregivers and to increase awareness about the disease; thereby increasing the prospect of funding for more research. Indeed it has been a success story to build on. In the past few months, five different drugs and drug regimens have been approved by the FDA to treat the disease. This certainly increases the prospect of increasing the survival of patients with this disease, which was until now considered incurable. Following the lead of Bart Barlogie, MD, PhD, a prominent researcher and MM expert, it is time to start thinking about and working toward the cure.
First, what is MM? MM is a blood disorder that involves abnormal proliferation of plasma cells, the cells responsible to make antibodies to protect us against infections. For people who suffer from this cancer, their immune system is very low; that puts them at risk for infection. This disease also causes anemia, renal failure, muscle, heart problems, and bone disease leading to fractures and high calcium in the blood causing symptoms such as drowsiness.
The American Cancer Society estimates that 30,330 people will be diagnosed with MM and 12,650 will die of the disease in 2016. The number of people diagnosed with MM has shown an increasing trend over the past decade. Fortunately, the death rate from MM has not shown the same increasing trend due to the introduction of more effective drugs and treatment regimens. Patients with MM are living longer and longer. Indeed, over the past few months, the Federal Drug Agency (FDA) has approved five different drugs and drug regimens to treat MM.
For one of my patients, the approval of Daratumumab (Darzalex), a human antibody aimed at a surface marker on the myeloma plasma cells named CD38, could not have come at a better time. He had exhausted most standard treatments including experimental regimens on a clinical trial. His bone marrow could not withstand any further assault from chemotherapy. After much discussion, we agree to start Daratumumab. He has had an excellent response, one of the 30 percent of patients who usually respond to this drug, including bone marrow recovery, that may allow him to explore other options later on, a true success story.
Another patient of mine in a similar predicament was started on another recently approved new regimen of Elotuzumab (Empliciti), another antibody aimed at another surface marker on myeloma plasma cells named SLAMF7 in combination with lenalidomide (another very effective drug in MM) and dexamethasone (a steroid). He is also experiencing a very good response.
These drugs are clearly very effective and have the potential of changing the way we treat MM. Other drugs and drug regimens recently approved include Ixazomib (Ninlaro), an oral proteasome inhibitor approved in combination with lenalidomide and dexamethasone; Carfilzomib (Kyprolis) with lenalidomide and dexamethasone; and panobinostat (Farydak), a new drug approved in combination with bortezomib and dexamethasone.
The basic theme of MM treatment is the combination of drugs is better than single agents and the combination of drugs with different mechanisms of action is effective even in cases where the single agents by themselves may not be completely active and thus can overcome resistance. As always, when living in abundance, we need to learn not to waste. In the case of MM, we need to use our bounty when it benefits patients best. That is a learning process since we do not have yet clinical trial data to guide us currently. Until then, it is best to seek the experience of an MM doctor.
Raymond Thertulien, MD, PhD is a former member of the Myeloma Institute of Research and Therapy in Little Rock, Arkansas and has performed extensive research and published several papers on MM. He is a member of Asheville Hematology and Oncology and a board member of the Western Carolina Medical Society Association.