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Adapted from the NCI Cancer Bulletin, vol. 5/no. 4, February 19, 2008 (see the current issue ³).

Patients with osteosarcoma who received the experimental drug mifamurtide (L-MTP-PE) along with chemotherapy fared better than patients who received chemotherapy alone, according to the Feb. 1, 2008, Journal of Clinical Oncology (see the journal abstract ⁴). Osteosarcoma is a rare but often fatal cancer of the bone. The disease typically affects children and young adults, and no new therapies have been introduced in two decades.

The study - conducted by the Children's Oncology Group ⁵ - was the largest final-stage randomized trial in this disease and included 662 patients with newly diagnosed nonmetastatic osteosarcoma.

After six years of follow-up, overall survival was 78 percent in the group receiving mifamurtide plus chemotherapy compared with 70 percent in the group receiving chemotherapy alone. "This is an almost one-third reduction in the risk of death," write Dr. Paul A. Meyers of the Memorial Sloan-Kettering Cancer Center and his colleagues.

A second goal of the National Cancer Institute-sponsored study was to evaluate the addition of ifosfamide to the three chemotherapy drugs used in the study (cisplatin ⁶, doxorubicin ⁷, and methotrexate). Adding this agent did not enhance event-free survival or overall survival for patients in the trial.

As an experimental agent, mifamurtide is available only through clinical trials. In 2006, its manufacturer, IDM Pharma, sought approval for its use in treating osteosarcoma from the U.S. Food and Drug Administration, but the agency requested more information. The company plans to submit new data showing an overall survival benefit in the disease this year.

Glossary Terms

An anticancer drug that belongs to the family of drugs called alkylating agents.

A drug used to treat some types of cancer, severe skin conditions such as psoriasis, and rheumatoid arthritis. Methotrexate stops cells from making DNA. It is a type of antimetabolite. Also called amethopterin and Rheumatrex.

Cancer that has not spread from the primary (original) site to other sites in the body.

A cancer of the bone that usually affects the large bones of the arm or leg. It occurs most commonly in young people and affects more males than females. Also called osteogenic sarcoma.

A study in which the participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively. At the time of the trial, it is not known which treatment is best. It is the patient's choice to be in a randomized trial.