Study Finds DFMO Significantly Increases Survival for Children with Neuroblastoma

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Trial funded by Beat Nb is the first study shown to decrease the high rate of relapse in kids with high risk neuroblastoma and improve survival.

These results are incredibly exciting and have changed the outlook for our patients with HRNB.

A study funded primarily by the Beat Nb Cancer Foundation found that oral therapy with DFMO reduces the rate of relapse and significantly increases survival for children with high-risk neuroblastoma (HRNB). The trial was performed through the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC), led by Giselle Sholler, MD MSC and NMTRC chair. Sholler presented the results of the study today at the ANR 2016 conference in Queensland, Australia.

HRNB remains a challenge in pediatric oncology, accounting for 15% of all pediatric cancer deaths. Current treatment protocols include harsh therapies which have significant long term side effects for patients. While most patients are able to attain remission, the natural history of HRNB is well documented with about 50% of patients relapsing after completion of therapy. The study, which ran from June 2012 to February 2016, used targeted oral therapy of an ODC inhibitor, difluoromethylornithine (DFMO), as a maintenance therapy to prevent relapse in HRNB patients after standard therapy. DFMO works by targeting specific cancer stem cell pathways and “turning off” the cells, thereby preventing the cancer from growing back.

101 children took part in the study, receiving oral DFMO over a two-year period, with minimal side effects reported. At the completion of the study, the event-free survival (EFS)* rate for the children who participated was 88%. That’s nearly a 20% increase from the typical EFS rate for children two years into remission. In addition, the study demonstrated a 99% overall survival rate at 2 years.

“These results are incredibly exciting and have changed the outlook for our patients with HRNB,” Sholler said. “In this first study, by using DFMO for two years after finishing conventional therapy, we’re seeing the overall survival rate for these kids at 99%. More work needs to be done to further study this. We would not be able to fund studies like this without the support of groups like Beat Nb, who are committed to changing the treatment paradigm for kids with neuroblastoma.”

Established in 2010, Beat Nb funds the discovery of new, effective therapies to increase survival and decrease the mortality associated with neuroblastoma.

“Results like these are why Beat Nb exists,” said Kyle Matthews, executive director of Beat Nb. “Thanks to the work of Dr. Sholler and her colleagues at the NMTRC, we’re changing the story for kids with neuroblastoma. This study confirms how well DFMO works in reducing the typically high relapse rate for kids with HRNB and shows a vast improvement in overall survival rates.

"Not only that, it’s an oral treatment that’s gentle on kids – it’s like night and day from the difficult therapies so many of them have to undergo. It’s crucial that we keep raising funds to get this groundbreaking research out of the lab and into our local hospitals, so we can help the families battling this disease today and keep working towards a cure.”

For more information or to find out how you can help save kids with cancer, visit

*Event-free survival (EFS): No progression of illness or other complications

About Beat Nb
Beat Nb is a 501(c)3 tax-exempt public charity dedicated to funding research and clinical trials focused on helping save the lives of children with neuroblastoma – an extraordinarily deadly cancer that most often strikes children under 5. Neuroblastoma is a nervous system tumor, and the most common cancer in infants and toddlers. Beat Nb funds the groundbreaking research and trials of the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC) – a national consortium of hospitals focused on saving kids fighting to stay alive. Beat Nb is led by Kyle Matthews (Ezra's dad) as Executive Director; founded by Patrick Lacey (Will's dad). Both fathers of sons with neuroblastoma, they directed two separate nonprofits with a shared mission before merging in June of 2016. Their shared passion has driven nearly $5m in research dollars to help fund the discovery of new, effective therapies to increase survival and decrease the mortality associated with neuroblastoma. To learn more, visit

The Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC) is a group of 25 universities and children’s hospitals, headquartered at the Helen DeVos Children’s Hospital in Grand Rapids, MI, that offer a nationwide network of childhood cancer clinical trials. These trials are based on the research from a group of closely collaborating investigators who are linked with laboratory programs developing novel therapies for high-risk neuroblastoma and medulloblastoma. NMTRC’s mission is to create a national collaborative effort of researchers, oncologists and family advocates to bring forward new therapies for children with relapsed neuroblastoma and medulloblastoma with the goal of improving quality of life and survival of children with neuroblastoma and medulloblastoma. More information on NMTRC can be found at

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Andrea LePain
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