Phase 1 Neuroblastoma Treatment: High-Dose DFMO with Chemotherapy

A recent study has shed light on the potential benefits of high-dose DFMO treatment combined with chemotherapy for patients with neuroblastoma, a type of cancer commonly associated with poor outcomes. The research, conducted by Panbela Therapeutics, Inc., a biopharmaceutical company at the forefront of developing innovative treatments for unmet medical needs, has been published in a reputable journal, demonstrating the tolerability and efficacy of the treatment.

The study focused on the use of CPP-1X, also known as α-Difluoromethylornithine (DFMO) or Eflornithine, in treating neuroblastoma. It is well-documented that children with this condition, especially those whose cancer has relapsed or is refractory, face a grim prognosis. The new data indicates that the treatment not only has a high tolerance rate but also extends the median time-to-progression to 19.8 months, a significant improvement over existing treatments.

The Phase 1 clinical trial results revealed a notable overall response rate, with some patients experiencing partial or minor responses, and stable disease in others. Remarkably, three patients have shown no signs of disease progression or events for over four years post-treatment, suggesting the potential for long-term benefits from the therapy.

This research builds on the FDA's recent approval of CPP-1X to reduce the risk of relapse in high-risk neuroblastoma patients, marking a significant milestone in the treatment of this disease. The findings have also prompted the initiation of the ANBL-1821 Phase 2 trial, which aims to further investigate the safety and effectiveness of the treatment.

Elizabeth Bruckheimer, PhD, Vice President & Chief Scientific Officer at Panbela, expressed enthusiasm for the study's outcomes, particularly in light of the FDA's approval for maintenance therapy. She highlighted the potential of high-dose DFMO as a viable treatment option for this patient population with high unmet needs.

The study's first author, Michael Hogarty, MD, Professor of Pediatrics at the University of Pennsylvania, emphasized the importance of understanding the biological mechanisms behind neuroblastoma, particularly the role of MYC signaling and the polyamine pathway. His work has contributed to the recognition of deep polyamine depletion as a promising therapeutic target.

Panbela Therapeutics continues to drive forward in its mission to develop groundbreaking treatments for patients with urgent medical needs. With its lead assets, Ivospemin (SBP-101) and Flynpovi, the company is well-positioned to make significant contributions to the field of oncology.

It is important to note that while the study's findings are promising, they are part of ongoing research and clinical trials. The ultimate goal is to develop effective novel therapies that can address the critical needs of patients with neuroblastoma and potentially other cancer types. The ongoing Phase II trial by the Children’s Oncology Group is a testament to the commitment to advancing the clinical management of neuroblastoma and improving patient outcomes.