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Neuroblastoma Researcher Investigates Promising New Treatment

Andrea Bachmann

The St. Baldrick’s Foundation, the largest private funder of childhood cancer research grants, has awarded a nearly $100,000 grant to support a Michigan State University scientist’s study of a promising new treatment for neuroblastoma, an often-fatal pediatric cancer.

For 17 years, André Bachmann, professor of pediatrics and the College of Human Medicine’s associate chair for research, has spearheaded preclinical research on a drug called DFMO, or difluoromethylornithine, as a potential weapon against neuroblastoma. DFMO was developed in 1978 and is extremely effective as the front-line therapy against West African sleeping sickness.

This oral drug is in several neuroblastoma clinical trials at more than 40 hospitals, including Spectrum Health’s Helen DeVos Children’s Hospital in Grand Rapids.

DFMO targets a protein called ornithine decarboxylase that, when elevated in the body, promotes the growth of neuroblastoma tumor cells.

“One problem with DFMO, as effective as it is, it doesn’t stay in the body for long,” but is rapidly excreted through the urine, Bachmann said.

The St. Baldrick’s grant will allow him to study an additive he believes will improve the effectiveness of DFMO by keeping the drug in a patient’s body for a longer period.

“Our hypothesis is that this additive” – already approved by the U.S. Food and Drug Administration (FDA) – “will keep DFMO in the blood stream much longer,” Bachmann said. “The benefit is patients won’t have to take as much of the drug, and, hopefully, the potency will increase. Less frequent drug intake at lower quantities will further reduce the already mild side effects and, importantly, lower the therapy costs, two additional benefits.”

Every year, about 700 children in the United States, most of them age 5 or younger, are diagnosed with the highly aggressive tumor, which forms on the nerve cells in several areas of the body. It accounts for about 12 percent of all childhood cancer deaths.

“I’m very excited to work with St. Baldrick’s,” Bachmann said. “I do appreciate that they saw the potential of his project and decided to fund it.”

If the FDA-approved additive improves the retention and effectiveness of DFMO, “it will have an immediate impact on how DFMO is used in the clinic, against neuroblastoma and potentially other forms of cancer,” he said. “Many of us are convinced that DFMO has a bright future. Being able to improve its retention in the blood and extending the life of the drug in the patient is going to make a huge difference in how the patients are treated.”

  • Geri Kelley and Sarina Gleason via MSU Today

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