Clinical trial for pediatric cancer drug now has N.J. openings

In In The News by Barbara Jacoby

By: Kathleen O’Brien | NJ Advance Media for


A national clinical trial for an unorthodox drug to treat a cancer that strikes very young children now has openings in New Jersey, thanks to a foundation.

The Phase 2 trial for a medication to treat neuroblastoma recently added Hackensack University Medical Center as a research site – one of only two East Coast research sites between Connecticut and Washington, D.C.

The drug in question is typically given to treat African sleeping sickness, but has already been shown to reduce tumors in mice. An earlier phase of the clinical trial also proved it safe enough for study in humans.

The drug, abbreviated DFMO, is being tested for its ability to prevent relapses in children with neuroblastoma.

Neuroblastoma is a cancer of the nervous system that typically shows up in very young children, with diagnosis made at 18 months on average. If tumors reappear after treatment, the prognosis becomes very grim.

The American Cancer Society calls neuroblastoma the most common cancer in infants under the age of one. There are about 700 new cases each year, accounting for about 6% of all cancers in children.

“The addition of DFMO to neuroblastoma treatment may prove to be a breakthrough therapy that prevents relapse and substantially improves the cure rate for these children,” said Derek Hanson, pediatric neuro-oncologist at Hackensack.

There are two parts of the clinical trial: one for children who have just been diagnosed, and another for children who have been treated but need to guard against relapse, Hanson said.

For the newly diagnosed participants, the study adds two components to the standard chemotherapy a child receives for the cancer, with one of them chosen after a genetic analysis of the child’s tumor.

For those participants who already received standard treatment, the experimental drug will be given for two years as a maintenance therapy.

The drug being tested is an anti-parasitic medication used to treat sleeping sickness. A researcher at the Grand Rapids-based Neuroblastoma and Medulloblastoma Translational Research Consortium realized it had properties that might make it effective against neuroblastomas, and conducted the initial research.

“Children’s cancer research in general is very underfunded, so we don’t have a lot of new drugs,” Hanson said. “So often they look at previously approved drugs.”

When the Sohn Conference Foundation, based in New York City, heard of the clinical trial, it donated funds to Hackensack so a site could be available in the greater New York metropolitan area, Hanson said.

In order to participate in the study, children must be treated at Hackensack. Any extra cost resulting from the study – lab work, etc., – would be covered, while the regular charges of treatment would be billed to the patient’s insurance.

The initial goal of 20 children has almost been met, Hanson said, but he expects the study to be expanded soon. “There are plenty of slots that are going to be open,” he said.

People interested in learning more about joining the trial can contact the hospital at 551-996-5437.