A new clinical trial, led by Penn State College of Medicine and the Beat Childhood Cancer Research Consortium, will evaluate the efficacy of combining tipifarnib with naxitamab for treating relapsed and refractory neuroblastoma. The trial, supported by Four Diamonds and the Beat Childhood Cancer Foundation, seeks to improve outcomes for children battling this aggressive cancer.
Novel Drug Combination Targets Immune Evasion
The clinical trial will investigate a novel drug combination designed to enhance the immune system's ability to fight neuroblastoma. Tipifarnib, an investigational agent supplied by Kura Oncology, blocks the enzyme farnesyl protein transferase, which can increase immunity against the tumor. Naxitamab, an anti-GD2 monoclonal antibody, is already used in patients with relapsed and refractory neuroblastoma. Preclinical research indicates that tipifarnib can reduce the tumor's ability to weaken the immune system, making anti-GD2 immunotherapy more effective.
Preclinical Evidence and Mechanism of Action
H.G. Wang, PhD, the Lois High Berstler professor of pediatrics and pharmacology at the College of Medicine, led the preclinical research that uncovered the mechanism by which tipifarnib enhances the efficacy of anti-GD2 monoclonal antibodies. According to Wang, "This drug combination helps the immune system fight neuroblastoma in children, particularly in addressing tumor cells that can evade standard treatments."
Trial Design and Enrollment
The trial aims to enroll more than 90 participants through the Beat Childhood Cancer Research Consortium hospitals nationwide. Valerie Brown, MD, PhD, professor of pediatrics at the College of Medicine, serves as the national clinical study chair. The study will provide critical insights into the molecular mechanisms involved in neuroblastoma and potentially pave the way for improved immune therapies.
Collaboration and Support
The Beat Childhood Cancer Research Consortium, based at the College of Medicine, is a global network of over 50 universities and children’s hospitals. Funding for this clinical trial is provided by the Beat Childhood Cancer Foundation and Four Diamonds. Kura Oncology, Inc. is supplying tipifarnib for the trial. 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, expressed excitement about advancing this critical research on a broader scale, emphasizing its potential impact on children across the country and worldwide.
Future Implications
Suzanne Graney, executive director of Four Diamonds, highlighted the dedication and time required for scientific discovery in cancer treatment, noting that years of preclinical work have led to this exciting clinical trial phase. Mollie Leoni, MD, executive vice president of clinical development at Kura Oncology, stated Kura's commitment to developing new treatment options and supporting this study to further understand the potential of the tipifarnib and naxitamab combination in treating neuroblastoma.
