MUSC researcher, part of team behind new neuroblastoma drug, celebrates FDA approval

When Jacqueline Kraveka, D.O., learned that 14 years of painstaking work had finally resulted in Food and Drug Administration approval of a drug to prevent relapse in high-risk neuroblastoma patients, one of the first things she did was to text the news to a patient’s mom.

The patient wasn’t yet 2 years old when she came under Kraveka’s care for neuroblastoma, a cancer of nerve tissues that’s most common in children under the age of 5. The little girl participated in the clinical trial that led to this month’s FDA approval of eflornithine, or DFMO, and is now, at age 8, four years after finishing the trial treatment, doing well – “fantastic” in fact, Kraveka said.

It’s an outcome she hopes for all patients with neuroblastoma.

“Neuroblastoma is one of the most aggressive pediatric tumors that we see. It’s one of the most common solid tumors we see but also has one of the worst prognoses. About half of children who are diagnosed with this tumor have high-risk neuroblastoma, and survival is only about 50%,” said Kraveka, who treats patients at MUSC Children’s Health and conducts research as part of MUSC Hollings Cancer Center.

DFMO is an antiparasitic approved for use in African sleeping sickness, but it’s also being studied in breast and colon cancer because it blocks a protein involved in cell growth. It came to the attention of neuroblastoma researchers because it blocks an enzyme that is a target of MYCN, a protein that’s often overexpressed in neuroblastoma. The Beat Childhood Cancer Research Consortium, founded by Giselle Sholler, M.D., conducted the research that led to the approval.

Kraveka has been deeply involved in the consortium since its inception. Today, it’s a network of more than 50 hospitals that collaborate on pediatric cancer research and clinical trials, but it was a small group of determined researchers when it began, Kraveka said.

She sits on both the executive committee and the scientific committee and was part of the core team that designed the clinical trials and helped to shepherd the drug through the approval process.

“I'm really humbled and just thrilled to have been part of this group and really be involved from the very beginning,” she said.

According to US WorldMeds, the pharmaceutical company that picked up DFMO, 84% of patients had no relapse by four years after immunotherapy, compared with 73% of patients in a control group. In addition, 96% of the children who received DFMO were still alive, compared with 84% of the children in the control group.

But as good as those results are, Kraveka said there’s more to be done to help children with cancer.

“One of the key things is that we need more research. We need funding. Pediatric cancer research is underfunded, with less than 4% of the budget of the National Cancer Institute dedicated to pediatric cancers,” Kraveka said. “It's through research that we've been able to improve outcomes for children with cancer.”

At the same time, Kraveka feels it’s immensely satisfying to know that with this approval, any child with high-risk neuroblastoma has the option of receiving the DFMO without needing to seek out one of the select centers offering it as part of a clinical trial.

“What’s great is now everyone can get it. Anyone can prescribe it. They don’t have to travel somewhere to get this medication,” she said. “It really is a huge help for our families and kids.”

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