The current study was a phase II clinical trial of 50 patients (ages 33 to 78 years) with newly diagnosed, active multiple myeloma. Of the 50 patients, 32 (64 percent) achieved a 50 percent or greater reduction in tumor volume with the combination oral treatment.

"The goal of both the standard chemotherapy approach and our research on the use of thalidomide plus dexamethasone is to reduce the amount of the cancer so patients can undergo stem cell retrieval and transplantation," said Vincent Rajkumar, M.D., the lead researcher.

"Our study with thalidomide plus dexamethasone represents a significant advancement because physicians now have an alternative to the more toxic and cumbersome chemotherapy regimens used to treat patients with newly diagnosed myeloma," said Dr. Rajkumar. "For patients who are newly diagnosed with multiple myeloma, the study means they may not need to receive the series of intravenous chemotherapy treatments, and they won't experience the side effects often seen with such chemotherapy, including nausea, vomiting and hair loss."

"The toxicity of thalidomide plus dexamethasone appears lower and the response rate is as good or better than that obtained using complex combinations of chemotherapy regimens," he added. "The most serious side effect seen in six patients in the study involved blood clots in the legs. Other side effects included constipation, skin rash, numbness in the hands and feet, and sleepiness."

He noted that patients who are not candidates for stem cell transplantation may have the option to continue the thalidomide plus dexamethasone treatment at reduced doses.

Despite the encouraging and promising results, Dr. Rajkumar cautioned that further studies are needed before the combination treatment can be recommended for routine clinical use; he is currently leading an Eastern Cooperative Oncology Group phase III clinical trial to investigate the effectiveness of thalidomide plus dexamethasone versus dexamethasone alone for treatment of the same (newly diagnosed) patient population. The results of the prospective randomized trial will help to establish the role of thalidomide plus dexamethasone in the initial treatment of multiple myeloma.

Although multiple myeloma accounts for only one percent of all cancers, it is among the most difficult cancers to treat and cure. This year, about 14,000 new cases will be diagnosed in the United States, and more than 11,000 patients will die from it. The average survival time for a patient diagnosed with multiple myeloma is about three to four years. However, there is significant hope that survival can be significantly extended based on promising results seen with thalidomide and other novel agents.

Thalidomide was first introduced in the mid-1950s as a sleeping pill. The drug was subsequently found to effectively control morning sickness during pregnancy. By 1962, however, the drug was taken off the market worldwide because of reports of severe malformations of the arms, legs and organs in newborns exposed to the drug in utero.

In the last 10 years, researchers have begun studying thalidomide as an anti-cancer agent. Although the exact mechanism of action in multiple myeloma is unknown, researchers have found that thalidomide effectively decreases the blood supply to malignant tumors and boosts the activity of the immune system.