Solid Tumors and Brain Tumors

Although great strides have been made in the cure of many pediatric tumors, a subset of solid tumors remains for which the chance of survival remains uniformly poor. The probability of achieving disease-free survival for children with newly diagnosed metastatic rhabdomyosarcoma, Ewing's/PNET, or high-grade glioma remains less than 25%, despite multi-modal conventional therapy. Likewise, the survival from a variety of pediatric solid tumors and brain tumors such as meduloblastoma, which have proven resistant to or have recurred following conventional multi-modal therapy remains poor with less than 10% of patients achieving long-term survival. Autologous bone marrow stem cell transplants have been utilized as the final stage of therapy for these pediatric patients with some encouraging improvement in survival. Nonetheless, at least half of the patients develop recurrent disease. A principal barrier to survival is the development of tumor resistance to standard chemotherapy drugs. Patients with recurrent lymphoma also benefit from autologous transplant.

We use several different conditioning regimens for patients with solid tumors depending upon the type of tumor and clinical circumstances. They include:

• topotecan, thiotepa, carboplantinum
• VP-16 (etoposide), melphalan, carboplantinum
• cyclophosphamide (cytoxan), BCNU, VP-16

Eligibility for the solid tumors protocol

The following groups of patients are eligible for this protocol:

• Patients with metastatic rhabdomyosarcoma or metastatic peripheral PNET in at least a partial remission following therapy.
• Patients with refractory or recurrent brain tumors, soft tissue sarcomas, Wilms' tumor, or germ cell tumors who have achieved a second or greater complete remission.
• Patients with progressive or recurrent Hodgkins or non-Hodgkins lymphoma

Brain Tumors Protocol

Patients with high grade brain tumors, in particular those less than 3 years of age, have a very poor survival rate when treated with prolonged chemotherapy and radiation. Furthermore, those who survive this intensive therapy tend to have significant developmental delays. Preliminary results from a previous protocol using this protocol are encouraging with fewer long-term side effects.

The goals of this study are the following:

• to evaluate the benefit and side effects of a new combination of chemotherapy agents as the initial treatment for newly diagnosed gliomas followed by high dose chemotherapy and autologous stem cell rescue;
• to evaluate benefit and side effects of a new chemotherapy regimen containing high dose Methotrexate as the initial treatment for newly diagnosed  high grade primitive neuroectodermal tumors (PNETs) followed by high dose chemotherapy and autologous stem cell rescue;
• to determine whether the use of radiation can be avoided or decreased in a subgroup of patients who have a complete response following the initial course of chemotherapy.

Eligibility for the brain tumors protocol

Patients who will eligible for this protocol will be:

• less than ten years of age;
• newly diagnosed with a high grade malignant brain tumor.