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Drug Studied in Neuroblastoma Patients

CancerWise - February 2008

By Darcy De Leon

A new clinical trial studying the experimental drug vandetanib (Zactima™ or ZD6474) has begun in children with neuroblastoma, an aggressive pediatric cancer that develops in immature nerve cells. It is the first time vandetanib has been studied in children.

The Phase I clinical trial, being conducted at the Children's Cancer Hospital at
M. D. Anderson, targets high-risk patients: those whose cancer is refractory (did not responded to therapy) or relapsed (returned after treatment).

Significance of the study

Better treatments with fewer drug toxicities are needed for this high-risk group of patients, who have an average five-year, disease-free survival rate between 25% and 35%, says Peter Zage, M.D., Ph.D., principal investigator on the study and an assistant professor at the Children's Cancer Hospital.

Standard treatment for children with high-risk neuroblastoma includes chemotherapy, stem cell transplantation, surgery, radiation therapy and 13-cis-retinoic acid, a form of vitamin A that has shown anti-cancer effects in neuroblastoma cells.

One of the most significant aspects of the trial is that vandetanib inhibits a protein and growth factors (molecules that fuel cancer development) that feed neuroblastoma tumors, Zage says.

They are:

  • RET ("Rearranged during transfection", a protein)
  • VEGF (Vascular endothelial growth factor)
  • EGF (Epidermal growth factor)

"We're excited because laboratory studies and clinical trials of adults have shown that vandetanib targets this specific type of cancer and has been shown to have minimal side effects," Zage says.

Goal of the study

The primary goal of the study is to determine vandetanib's:

  • Safety
  • Dose-limiting toxicities
  • Maximum tolerated dose (MTD)

The second goal is to measure how tumors respond to treatment and how much time passes without the disease progressing. Researchers also will explore blood-based biomarkers (signs of disease or health) before and after treatment.

Study description

The trial will be conducted in three parts to establish MTD and safety of the pill or liquid form of vandetanib alone and vandetanib with 13-cis-retinoic acid.

In part A, children will receive escalating doses of vandetanib alone. In parts B and C, children will receive different dosing schedules of vandetanib with 13-cis-retinoic acid.

Possible side effects

In previous studies, side effects were measured at an MTD of 300 milligrams per day.

Those side effects have included:

  • Skin rash
  • Diarrhea
  • Irregular heart beat


Pediatric patients entering the trial must:

  • Be older than 2 years old
  • Have a diagnosis of either:
    • Relapsed neuroblastoma
    • Refractory neuroblastoma
  • Not have a history of cardiac disease

Parents should ask about other exclusions for participation, Zage says.


Vandetanib was granted approval from the U.S. Food and Drug Administration (FDA) in October 2005 for the treatment of thyroid cancer under terms of the Orphan Drug Act (ODA). The ODA provides financial incentives to drug manufacturing companies that produce drugs for rare diseases affecting fewer than 200,000 people nationwide.

Annually, thyroid cancer is diagnosed in approximately 33,550 people, and multiple myeloma is diagnosed in approximately 650 people, according to the American Cancer Society.

Vandetanib also is approved by the FDA for use in adults with lung cancer and has been studied in other adult cancers.

For more information about the trial, contact askMDAnderson at 1-877-632-6789.

M. D. Anderson resources:

  • Children's Cancer Hospital

© 2015 The University of Texas MD Anderson Cancer Center