Clinical trial investigates pomalidomide in only U.S. study for multiple myeloma

Multiple myeloma, a type of blood cancer in which malignant plasma cells are overproduced in the bone marrow, has led to an estimated 20,000 new diagnoses and 11,000 deaths in 2010. Patients with the disease, who have already failed previous treatments, may be considered for a currently enrolling Phase II study, examining a unique drug combination.

Myeloma begins when a plasma cell becomes abnormal. The abnormal cell divides to make copies of itself, making more myeloma cells, which collect in the bone marrow, and may damage the solid part of the bone. Symptoms of multiple myeloma include bone pain (usually in the back and ribs), broken bones (usually in the spine), feeling weak and very tired, feeling very thirsty, frequent infections and fevers, weight loss, nausea or constipation, and frequent urination.

The clinical trial, available only at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, is investigating the use of an experimental drug called pomalidomide.

"This trial is the only study in the United States evaluating pomalidomide in combination with Biaxin for multiple-myeloma patients who have failed three or more previous therapies," says Dr. Ruben Niesvizky, the study's principal investigator, associate professor of medicine at Weill Cornell Medical College, and director of the Multiple Myeloma Service at the Center of Excellence for Lymphoma and Myeloma at NewYork-Presbyterian Hospital/Weill Cornell Medical Center.

Pomalidomide is an immunomodulatory agent that stimulates the body's own immune system to fight off cancer cells. The drug is taken in pill form and has the unique action of targeting the microenvironment of the cancer cell, not just the malignant cell itself.

The compound is a variation of thalidomide, which has already been shown to be effective in treating multiple myeloma. The new compound is believed to have less toxicity for patients, demonstrating a potentially lower risk of neuropathy, which is damage to nerves, causing chronic pain.

"Patients often become resistant to thalidomide or lenalidomide treatment, so pomalidomide may offer them a new treatment option, if proven to be effective," explains Dr. Niesvizky.

Along with the new experimental drug, patients who are enrolled in the trial are also given an antibiotic called Biaxin and a steroid called Decadron. Both act to kill multiple-myeloma cancer cells, and are also taken in pill form.

To be considered for this trial, patients must be at least 18 years of age and have relapsed or have disease that has progressed after at least three prior therapeutic treatments or regimens for multiple myeloma. Patients must have also been previously treated and failed prior therapy with lenalidomide. For more information about enrollment, please contact Patrice Mignott, R.N., at (212) 746-1480, or visit weill.cornell.edu/cancercare/trials/lymphoma/multiplemyeloma.html.