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Family Matters - Spring 2009

Translating Laboratory Research into New Treatments for Childhood Cancer

Mechanism Identified That Increases Effectiveness of Therapy Against Leukemia

In a recent issue of the medical journal BLOOD, researchers from the Children’s Cancer Hospital at M. D. Anderson reported that combining two specific drugs appears to be five times more effective against leukemia than either cancer agent alone.

For the first time, associate professor of pediatrics Joya Chandra, Ph.D., and graduate student Claudia Miller showed that the novel proteasome inhibitor, NPI-0052, shares similar functions as the histone deacetylase (HDAC) inhibitor, vorinostat.

“Combining the two anti-cancer agents should allow clinicians to use a lower dosage of each drug, which hopefully will result in fewer side effects for the patient,” says Chandra. “For pediatric patients in particular, the fewer side effects they have from treatment could mean a better quality of life as survivors in the long run.”

NPI-0052 is currently being tested with solid tumor malignancies and recurrent lymphoma in Phase I human clinical trials at M. D. Anderson. A combination trial to test NPI-0052 with vorinostat is in the planning.

Device Aims to Decrease Wait Period for Patients Needing Immunotherapy

Paul (Yoonsu) Choi, Ph.D., from the Children’s Cancer Hospital at M. D. Anderson has created a device, called HitMeD (high throughput medical electroporation device), that significantly decreases the time needed to produce genetically manipulated T cells in preclinical tests for leukemia.

Multiple relapsed acute lymphocytic leukemia (ALL) in pediatric patients is a rapidly progressive cancer that is often resistant to chemotherapy, leading to poor survival prognosis. Since chemotherapy typically fails these patients, new approaches, such as cell-based therapy, are needed to combat the quickly spreading leukemia.

Choi, along with senior researcher Laurence Cooper, M.D., Ph.D., from the Children’s Cancer Hospital, are studying ways to genetically manipulate T cells, an important component of a person’s immune system, to specifically attack tumor cells while keeping risk to the patient at a minimum.

Cooper and researchers are planning a Phase I trial that could open this year. This trial would allow multiple-relapsed ALL patients to receive manipulated T cells that have been processed by HitMeD. These special T cells will act like an army of antibodies rushing in to attack tumor cells, but quickly retreating after their ammunition is used. With HitMeD, doctors hope to infuse additional doses of T cells more rapidly to sustain the fight until the patient can receive additional treatment, such as a stem cell transplant.

Immune-Based Drug Approved in Europe for Pediatric Bone Cancer Patients

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 Children's Cancer Hospital at M. D. Anderson 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% decrease in the risk of death with 78% 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.

Patients receiving MEPACT in Europe will undergo pre-operative chemotherapy followed by surgery to resect the bone tumor and then receive post-operative chemotherapy. While receiving post-operative chemotherapy, patients will also be 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 Eugenie Kleinerman, M.D., head of the Children's Cancer Hospital and lead researcher for MEPACT. “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.

“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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