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Neuroblastoma patients at Floating Hospital get access to promising experimental drug

It’s a mouthful to say—difluoromethyl-ornithine (DFMO)—but it is shaping up to be a genuine lifesaver for families across New England. The Neuroblastoma and Medulloblastoma Translational Research Consortium is providing access to this promising new experimental drug for children suffering from a rare but often terminal cancer.

In joining the consortium, which is known as the NMTRC, Floating Hospital for Children at Tufts Medical Center has become the only institution in New England outside of Connecticut that offers enrollment in this study, which seeks to prevent relapse for those with high-risk neuroblastoma.

Just two dozen hospitals and universities in the US belong to the consortium. "Children take DFMO after they’ve been treated for neuroblastoma," says Michael Kelly, MD, MPH, MS, a pediatric hematologist/oncologist at Floating Hospital. Neuroblastoma, a cancer with a mortality rate of about 50 percent, develops from nerve cells that can be found in different areas of the body. The drug, which is taken orally, seeks to prevent recurrence. "It’s really well tolerated, and we are optimistic that it may lower the incidence of relapse," Dr. Kelly says.

Floating Hospital’s Pediatric Oncology group is involved in other drug trials, too, offering a range of breakthrough treatments to kids who have various forms of cancer. In fact, 44 percent of Floating Hospital’s cancer patients from the past two years are enrolled in a clinical trial, and 13 percent are enrolled in more than one.

To learn more about the NMTRC and involvement in the Neuroblastoma study, please contact the Division of Pediatric Hematology/Oncology at 617-636-5535.