For Young Children with Brain Cancer, Innovative Therapy is Promising

Press Release
(Embargoed for release Dec. 30, 1997)

Contact: Peter Ferrara, (212) 263-5800

For Young Children with Brain Cancer, Innovative Therapy is Promising

Malignant brain cancers in young children can be eradicated with high-dose chemotherapy and stem-cell transplants, eliminating the need for conventional radiation therapy, which causes irreparable physical and psychological damage in young children, according to two studies by New York University School of Medicine researchers.

The new studies, led by Jonathan Finlay, M.B., Ch.B., Director of the Stephen D. Hassenfeld Children's Center for Cancer and Blood Diseases at New York University Medical Center, are published in the January issue of the Journal of Clinical Oncology.

"Our work is very encouraging," said Dr. Finlay, who conducted the study while at Memorial Sloan-Kettering Cancer Institute in New York. "It suggests that we can cure more children with malignant brain cancer and improve their quality of life by treating them with a short course of high-dose chemotherapy and stem-cell rescue. Using this protocol, most children do not have to undergo radiation treatment."

Despite advances in treating many childhood cancers, the prognosis for children with brain cancer is grim. Brain cancer is the second leading cause of cancer-related death in children under the age of 15. In the U.S., about 1,700 brain tumors are diagnosed each year in children.

Malignant brain cancer usually is treated by surgery to remove as much of the tumor as possible without harming vital brain regions and is followed by radiation therapy to the brain and sometimes the spinal cord. However, in young children, especially infants and pre-schoolers, radiation therapy causes memory loss, learning delays, shortened attention spans, psychological problems and growth retardation.

Fortunately, there may now be an alternative. The most common type of malignant brain tumor in children, called medulloblastoma, which originates in the back of the brain, can respond to chemotherapy, according to recent studies. It is now hoped radiation treatment in young children can be eliminated, or at least delayed, by using chemotherapy to eradicate this cancer.

One approach has been to administer standard chemotherapy over a period of two years, but results from these studies have been disappointing-less than one-third of young children with medulloblastoma survive free of disease five years after treatment.

More children with this type of tumor survive free of cancer three years after treatment using another approach developed by Dr. Finlay.

Led by Dr. Finlay and co-investigators at five medical centers, the treatment approach combines high-dose chemotherapy with autologous stem-cell transplants. Stem cells are the progenitors of all the body's blood-forming cells. Autologous (meaning from the patient) transplants allow much higher doses of chemotherapy to be administered than otherwise possible.

Very high doses of chemotherapy kill more cancer cells, but the doses also kill stem cells, impairing the body's ability to fight infection. In Dr. Finlay's protocol, a portion of the stem cells is removed prior to high-dose chemotherapy and is frozen. After the chemotherapy, the stem cells are reinfused into the patient, which reconstitutes the blood-forming cells of the body.

The new study involved 62 children under the age of six with newly diagnosed medulloblastoma and other malignant brain tumors. Most of these children received five rounds of standard, but intensive chemotherapy over a five-month period. After the first round, the stem cells were removed from their blood. Following the fifth round, extremely high doses of the drugs carboplatin, thiotepa and etoposide were administered in more than half of these children. The stem cells were then restored to their blood.

Three years after the treatment, more than half of the 37 children who underwent the combined treatment are cancer free and they are normal physically and psychologically. "I've never seen such normally functioning survivors amongst pre-schoolers with brain tumors in my more than 20 years of treating childhood brain cancers," said Dr. Finlay.

Based on these results, a larger study is underway with cancer centers in Australia, Argentina and the United States, said Dr. Finlay, who will be coordinating the study.

A second study in the same issue of the Journal of Clinical Oncology suggests that the combined treatment protocol can benefit children with recurrent medulloblastoma, a virtually fatal disease in which the cancer recurs after initial treatment. In this study, seven of 23 patients are cancer-free survivors two years to more than 6 years after the combination treatment. "We aren't used to seeing children with recurrent medulloblastoma survive without disease for this long. We find it very hopeful," said Dr. Finlay.