Immune-based drug approved in Europe for pediatric cancer patients

March 10, 2009

HOUSTON ? The European Commission, which oversees legislation and regulation for the European Union, has approved a therapy for pediatric patients with non-metastatic, resectable osteosarcoma, a type of bone cancer. The approval is based on clinical studies led by researchers at The University of Texas M. D. Anderson Cancer Center and a national co-operative group.

MEPACT (mifamurtide, L-MTP-PE) is an immune-based therapy, that when combined with chemotherapy, resulted in approximately a 30 percent decrease in the risk of death with 78 percent of patients surviving more than six years following treatment. This therapy is the first in more than 20 years to improve the long-term survival of osteosarcoma patients.

Eugenie Kleinerman, M.D., head of the Children's Cancer Hospital at M. D. Anderson Cancer Center, was the first investigator to translate the drug from preclinical testing to a Phase I clinical trial in humans. She also led the Phase II clinical trial for pediatric patients with relapsed osteosarcoma, which was followed by a Children's Oncology Group Phase III trial for newly diagnosed patients.

Kleinerman originally proposed the use of this immune therapy for osteosarcoma after Isaiah J. Fidler, D.V.M., Ph.D., professor in M. D. Anderson's Department of Cancer Biology and director of the Center for Metastasis Research, demonstrated that MEPACT induced the regression of melanoma lung metastases in mice.

"When he showed that MEPACT caused the macrophages in the lung to kill tumor cells, I decided that the drug may have therapeutic potential in patients with osteosarcoma, which most often metastasizes to the lungs," says Kleinerman. "From my own preclinical research, we were able to show how MEPACT stimulated human immune cells to react against osteosarcoma cells."

MEPACT works by stimulating certain white blood cells, called macrophages, to kill tumor cells. The drug is shaped in a sphere, also known as a vesical, made up of lipids. Inside the vesical is muramyl tripeptide (MTP). The lipids trigger the macrophages to consume MEPACT. Once consumed, the MTP stimulates macrophages, particularly in the liver, spleen and lungs, to find tumor cells and kill them.

Patients undergo pre-operative chemotherapy followed by surgery to resect the bone tumor and then receive post-operative chemotherapy. While receiving post-operative chemotherapy, patients also are given the immune therapy intravenously twice a week for three months and then once a week for six months. The chemotherapy acts like a bomb sent in to destroy the tumor, while MEPACT acts as a special forces unit sent in to clean out any remaining pockets of microscopic disease.

"Relapsed osteosarcoma is often resistant to chemotherapy," says Kleinerman. "By giving MEPACT to newly diagnosed patients, we hope to prevent relapse by taking care of any remaining tumor cells after chemotherapy."

Currently, only relapsed pediatric patients with osteosarcoma are able to receive treatment with MEPACT through compassionate use in the United States. MEPACT was granted orphan drug status in the United States in 2001 but has not been approved by the Food and Drug Administration for use in newly diagnosed patients. Orphan drug status is given to therapeutic agents that target rare diseases as an incentive for pharmaceutical companies to manufacture these agents.

"We have been working with this therapy for more than two decades, so getting approval in Europe is a huge milestone for those of us fighting pediatric cancer," says Kleinerman. "This drug has made significant strides for long-term survival of children with osteosarcoma."
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About M. D. Anderson

The University of Texas M. D. Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. M. D. Anderson is one of only 40 comprehensive cancer centers designated by the National Cancer Institute. For four of the past six years, including 2008, M. D. Anderson has ranked No. 1 in cancer care in "America's Best Hospitals," a survey published annually in U.S. News & World Report.

About the Children's Cancer Hospital at M. D. Anderson

The Children's Cancer Hospital at The University of Texas M. D. Anderson Cancer Center has been serving children, adolescents and young adults for more than 50 years. In addition to the groundbreaking research and quality of treatment available to pediatric patients, the Children's Cancer Hospital provides its patients with comprehensive programs that help children lead more normal lives during and after treatment. For further information, visit the Children's Cancer Hospital Web site at www.mdanderson.org/children.

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University of Texas M. D. Anderson Cancer Center

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