Inhibiting Tumor Angiogenesis in Children

Name of the Trial

Phase I Study of Cediranib in Pediatric Patients with Refractory or Recurrent Extracranial Malignant Solid Tumors or Acute Myeloid Leukemia (NCI-06-C-0152). See the protocol summary.

Principal Investigator

Dr. Elizabeth Fox, NCI Center for Cancer Research

Why This Trial Is Important

Great progress has been made in the treatment of childhood cancers over the past 30 years, thanks primarily to advances in chemotherapy and a high level of participation in clinical trials by pediatric patients. This progress, however, is in danger of stalling without new treatment advances.

One approach that has shown effectiveness in the treatment of some adult cancers is inhibiting the growth of new blood vessels to tumors (i.e., blocking tumor angiogenesis). Without angiogenesis, solid tumors are unable to grow beyond a few millimeters in size. Because drugs that inhibit angiogenesis may cause different side effects in children than they do in adults, it is important to carefully test their use in pediatric cancer patients.

In this trial, researchers are testing an angiogenesis inhibitor called cediranib in pediatric patients who have solid tumors (except brain tumors) or acute myeloid leukemia (AML), a type of blood cancer. Although solid tumors are not formed in AML, there is evidence that a protein known to be important in tumor angiogenesis (i.e., vascular endothelial growth factor, or VEGF) may also be important for the growth of AML cells. Cediranib blocks all three known receptor proteins for VEGF.

“Solid tumors in children tend to be highly vascular, and adult AML patients with elevated levels of VEGF typically don't survive as long as those with lower levels,” said Dr. Fox. “So, there's a strong rationale for using VEGF inhibitors for these cancers. However, it is vitally important to assess the toxicities and establish the appropriate dosage of cediranib for pediatric patients.”

“Furthermore, we plan to study how cediranib affects a number of markers related to cancer progression and, at least preliminarily, determine tumor responses to cediranib in these patients,” she added.

For More Information

See the list of entry criteria and trial contact information or call the NCI Clinical Trials Referral Office at 1-888-NCI-1937. The call is toll free and confidential.