FDA Update FDA Approves Lenalidomide for MDS The Food and Drug Administration (FDA) has approved lenalidomide (Revlimid) for the treatment of patients with myelodysplastic syndrome (MDS). The approval is only for patients with a specific chromosomal abnormality, a deletion of DNA on chromosome 5. This is the case in 20 to 30 percent of MDS cases, according to the drug's manufacturer, Celgene Corporation. MDS is a collection of disorders, the hallmark of which is abnormal bone marrow function that leads to inadequate blood cell production. Some forms of MDS can progress to acute myeloid leukemia. MDS patients may need blood and platelet transfusions and antibiotic therapy for infections. Results from a 148-patient, phase II clinical trial indicated that treatment with the drug in patients with MDS and the chromosome 5 deletion eliminated the need for blood transfusions. The transfusion-free period lasted for an average of 44 weeks, according to the FDA. Preliminary trial results were presented at the 2005 American Society of Clinical Oncology annual meeting. Researchers reported that 70 percent of those who responded had fewer bone marrow cells with the chromosomal deletion, and the deletion was undetectable in 44 percent of responders. Lenalidomide is structurally similar to thalidomide, which can cause severe birth defects; it will be made available via a risk-management program designed to prevent fetal exposure.

Name of the Trial
Phase II Randomized Study of Single-Agent Bortezomib Followed by Induction Therapy Comprising Bortezomib in Combination with Dose-Adjusted Etoposide, Prednisone, Vincristine, Cyclophosphamide, Doxorubicin, and Rituximab (DA-EPOCH-R) and Maintenance Therapy Comprising Bortezomib Versus Observation in Patients with Previously Untreated Mantle Cell Lymphoma (NCI-05-C-0170). See the protocol summary at http://cancer.gov/clinicaltrials/NCI-05-C-0170.

Principal Investigator
Dr. Wyndham Wilson, NCI's CCR

Why Is This Trial Important?
Mantle cell lymphoma (MCL) is an aggressive cancer of the immune system. MCL usually responds well to induction chemotherapy (treatment to induce disease remission) but then progresses in most patients. The average survival of patients diagnosed with MCL is 3 to 4 years.

In this trial, researchers will treat MCL patients with the drug bortezomib, followed by induction therapy with bortezomib and a chemotherapy combination known as DA-EPOCH-R, along with the drug filgrastim to stimulate white blood cell production. Half of the patients will then receive maintenance therapy with bortezomib, while the other half will not receive bortezomib unless their disease progresses.

Bortezomib is a new type of drug that blocks the activity of proteins important for cell proliferation, tumor survival, and the formation of new blood vessels to tumors. The NCI-developed DA-EPOCH-R combination has been effective against other difficult-to-treat, aggressive lymphomas. Researchers hope that combining bortezomib with the DA-EPOCH-R regimen will lead to lasting remission of MCL.

"We are exploring the use of targeted therapy combined with very active chemotherapy in hopes of improving the survival of patients with MCL," said Dr. Wilson. "We hope to prolong the period of remission by administering long-term bortezomib maintenance therapy."

Who Can Join This Trial?
Researchers seek to enroll 80 patients aged 18 or over with previously untreated mantle cell lymphoma. See the list of eligibility criteria at http://cancer.gov/clinicaltrials/NCI-05-C-0170.

Where Is This Trial Taking Place?
This study is taking place at the NIH Clinical Center in Bethesda, Md., and the Dana Farber Cancer Institute in Boston, Mass.

Contact Information
Call NCI's Clinical Studies Support Center at 1-888-NCI-1937 or the Dana Farber Cancer Institute at 1-800-790-4500. The toll-free calls are confidential.

An archive of "Featured Clinical Trial" columns is available at http://cancer.gov/clinicaltrials/ft-all-featured-trials.