Mayo Clinic update on multiple myeloma

Dear Mayo Clinic: Is thalidomide now approved for use in patients with multiple myeloma? My father took it "off label" several years ago and had a very good result. Are there other treatment options for this disease?

Answer (from Dr. S. Vincent Rajkumar in hematology at the Mayo Clinic in Rochester, Minn.): Despite thalidomide's notorious past - having caused numerous cases of severe birth defects when used by pregnant women - the drug is making a comeback as treatment for several conditions.

One of them is multiple myeloma, a cancer of the bone marrow's plasma cells. Abnormal versions of these cells proliferate, leading to anemia, immune deficiency, weakened bones, kidney problems and other symptoms.

At present, there is no cure for multiple myeloma, but we are steadily adding treatments that lengthen patient survival time and improve quality of life.

Until recently, conventional chemotherapy was our only option, and drugs were limited to two types: alkylating agents (such as melphalan and cyclophosphamide) and corticosteroids (prednisone and dexamethasone). These drugs kill abnormal plasma cells, called myeloma cells.

But now our arsenal is being supplemented by other medications such as thalidomide, bortezomib and lenalidomide, which target the myeloma cells as well as the microenvironment that nurtures them. The new drugs may suppress the blood supply or growth factors on which the abnormal cells depend. As a result, we are achieving greater success rates, especially when new and conventional drugs are used in combination.

A recent large French study showed that adding thalidomide to standard chemotherapy prolonged survival in multiple myeloma. In another study, my colleagues and I found that average time to progression was more than twice as long in patients on thalidomide and dexamethasone compared to those on dexamethasone alone. Thus they enjoyed longer periods of being relatively symptom-free.

The FDA recently approved thalidomide in combination with dexamethasone for treatment of newly diagnosed multiple myeloma.

New options don't necessarily displace old treatments but can be additive and even synergistic.

We are now planning three-drug trials and look forward to four-drug trials. The individual agents, by themselves, are not panaceas, but in combination they lengthen survival. Our hope is to find the ultimate combination - from currently available drugs as well as newer ones now being tested - that will constitute a cure for multiple myeloma.