Penn State College of Medicine, Beat Childhood Cancer Research Consortium leading clinical trial to test new drug combination in neuroblastoma
Penn State College of Medicine, the Beat Childhood Cancer Research Consortium and Four Diamonds are set to begin a clinical trial testing a new drug combination to treat relapsed and refractory neuroblastoma that has shown promising results in preclinical studies.
Valerie Brown, MD, PhD, professor of pediatrics at the College of Medicine, is the national clinical study chair. Researchers project to enroll more than 90 participants through the Beat Childhood Cancer Research Consortium hospitals nationwide.
“We hope to gain a deeper understanding of the molecular mechanisms involved in neuroblastoma,” said Brown. “This could lead to a big step forward in the ongoing battle against neuroblastoma and offers hope for improved treatment outcomes for affected children and their families.”
The trial aims to evaluate the efficacy of a novel drug combination in treating neuroblastoma, a rare and challenging pediatric cancer. The drug combination involves the use of tipifarnib. This investigational agent blocks the enzyme farnesyl protein transferase which increases immunity against the tumor, in combination with an immunotherapy anti-GD2 monoclonal antibody, naxitamab, already utilized in patients with relapsed and refractory neuroblastoma.
The preclinical research, led by H.G. Wang, PhD, the Lois High Berstler professor of pediatrics and pharmacology at the College of Medicine and national research study chair, uncovered a novel mechanism by which the addition of tipifarnib enhances the efficacy of anti-GD2 monoclonal antibodies. Four Diamonds and Lois High Berstler Research Endowment Fund supported this early investigative work.
“This drug combination helps the immune system fight neuroblastoma in children, particularly in addressing tumor cells that can evade standard treatments,” Wang said. “Early studies show that tipifarnib can reduce the tumor’s ability to weaken the immune system and make anti-GD2 immunotherapy work more effectively.”
With the College of Medicine serving as its home, the Beat Childhood Cancer Research Consortium is a worldwide network of more than 50 universities and children’s hospitals focused on saving lives and helping make a lasting difference around the world. The Beat Childhood Cancer Foundation and Four Diamonds are providing funding for this clinical trial. Kura Oncology, Inc. is supplying tipifarnib for this clinical trial.
“We are excited to advance this critical research on a broader scale,” said Dr. Giselle Sholler, division chief of pediatric hematology/oncology, director of pediatric oncology research at the College of Medicine and chair of the Beat Childhood Cancer Research Consortium. “The knowledge we gain from this trial has the potential to pave the way for improvement in immune therapies that can impact children across the country and worldwide.”
Four Diamonds has supported cancer care for qualifying pediatric cancer patients and their families and funded support services and innovative research at Penn State Health Children’s Hospital for more than five decades.
“Scientific discovery to change the future of cancer treatments takes time and dedication,” said Suzanne Graney, executive director of Four Diamonds. “Our generous donors have helped to fund years of preclinical work that has led us to move this research into the exciting clinical trial phase.”
“Kura remains committed to developing new treatment options so that patients with cancer may lead better, longer lives,” said Mollie Leoni, MD, executive vice president of clinical development at Kura Oncology. “The combination of tipifarnib and naxitamab has shown promising results in preclinical studies and we are proud to support this important study to further our understanding of its potential in the treatment of neuroblastoma.”
For more information about the trial, contact Beat Childhood Cancer Research Consortium at BCCEnroll@pennstatehealth.psu.edu.
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