by Ashley WennersHerron, Pennsylvania State University

Credit: Unsplash/CC0 Public Domain

In 2003, the first year of her fellowship in pediatric oncology, two of Giselle Saulnier Sholler's first three patients had died from neuroblastoma, closely matching the 30% survival rate expected at the time for high-risk neuroblastoma. She knew research was the only way to change these outcomes.

Now, 20 years later, the U.S. Food and Drug Administration (FDA) has approved the first drug—based on Sholler's research and clinical trials she led through the Beat Childhood Cancer Research Consortium—to reduce the risk of relapse for pediatric high-risk neuroblastoma patients.

"Neuroblastoma is rare, with only 800 cases diagnosed in the United States each year," said Sholler, who is now the division chief of pediatric hematology and oncology at Penn State Health Children's Hospital and the director of pediatric oncology research at Penn State College of Medicine.

"I am so thankful that our research studying this drug, both in our laboratory and through clinical trials, over the past 14 years has brought so much hope to our pediatric oncology teams and the families of children with high-risk neuroblastoma."

Neuroblastoma is a cancer that develops primarily in children less than six years old. It originates in immature nerve cells found in several areas of the body, most commonly in the adrenal gland and next to the spine. About 50% of the patients already have advanced metastatic disease by the time they are diagnosed, with spread to lymph nodes, bones, bone marrow and liver most commonly. Of these high-risk patients, Sholler emphasized that about 40% of those who enter remission will relapse within four years, and of those, fewer than 10% will survive five more years.

The drug, called eflornithine (DFMO) and newly branded as IWILFIN by US WorldMeds, reduces the risk of relapse for pediatric high-risk neuroblastoma patients achieving at least a partial response after multiagent, multimodality therapy including immunotherapy by more than 50%.

This finding was the result of a trial analysis led by Sholler and the Beat Childhood Cancer Research Consortium in collaboration with US WorldMeds. The study has been published in the Journal of Clinical Oncology . According to Sholler, the evidence provided by this study led directly to FDA approval.

"Dr. Sholler and her team have an unwavering singular focus on doing what is best for the children in the clinic today," said Patrick Lacey, who founded the Beat Childhood Cancer Foundation and has a child with neuroblastoma.

"That belief drives everything they do and mirrors the indefensible hope that we have as parents of children with cancer that something can be done. This drug approval has the potential to dramatically change the landscape for kids with neuroblastoma, and it all started with the belief that it was possible—followed by the sheer determination to take every step necessary to achieve that goal. We are fortunate to be able to help to fund these efforts to help improve survival for children who need our help."

Kristen Gullo, vice president of development and regulatory affairs at US WorldMeds, spearheaded the company's efforts.

"We are thankful for our collaboration with the Beat Childhood Cancer teams to be able to make such a difference in the lives of children with cancer," said Gullo, whose family was also touched by neuroblastoma.

Yatin M. Vyas, professor and chair of the Department of Pediatrics in the Penn State College of Medicine and pediatrician-in-chief of the Penn State Health Children's Hospital, echoed the sentiment.

"Collaborations among clinicians, researchers, industry and the people most directly affected by pediatric cancer—our patients' families—can produce incredible results," said Vyas, who is also the vice president of Penn State Health Children's services and the Children's Miracle Network and Four Diamonds Endowed Chair.

"It is this type of dedicated work that can change lives, and we are proud of Dr. Sholler and grateful for her commitment to improve outcomes for what has been such a devastating disease."

More information: Javier Oesterheld et al, Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score–Matched Survival Outcome Comparisons, Journal of Clinical Oncology (2023). DOI: 10.1200/JCO.22.02875

Journal information: Journal of Clinical Oncology  , Scientific Reports 

Provided by Pennsylvania State University