Who can enter
• Children and teenagers with a type of pediatric cancer in whom the disease has returned (relapse) or responded insufficiently to treatment (refractory).
• Age: 1 up to 18 years.
Goal
The goal of this study is to determine how safe the drug ponatinib is for the treatment of children with cancer, and to find out what effects this drug might have on children and how the disease may respond.
Background
Tyrosine kinases are enzymes that play a role in many important processes in the cell, such as growth and cell division. Certain aberrations in the DNA of tumor cells enhance the activity of these tyrosine kinases. This may help tumor cells to grow and survive. Several drugs specifically block certain tyrosine kinases, and have been shown to be effective in the treatment of cancer. These drugs are called tyrosine kinase inhibitors (TKIs).
The drug ponatinib is such a TKI. It inhibits the activity of BCR-ABL, an abnormal tyrosine kinase that is often found in chronic myeloid leukemia (CML) and certain types of acute lymphoblastic leukemia (Ph+ ALL). Ponatinib also inhibits other tyrosine kinases, among which KIT, RET, FLT3, VEGFR and FGFR. Aberrations in these tyrosine kinases have been found in different types of cancer.
Ponatinib has been approved for the treatment of adults with CML or Ph+ ALL who are refractory or have become insensitive (resistant) to other treatments. However, this drug has not yet been approved for the treatment of relapsed or refractory types of cancer in children. In this study we want to determine if ponatinib is effective in children as well.