Breakthrough Treatments and Clinical Trials Coming to Pediatric Neuro-Oncology
Hackensack Meridian Health Joseph M. Sanzari Children’s Hospital is Pioneering New Treatments with Multi-Disciplinary Expertise
With 25 clinical trials open for neuro-oncology patients, Hackensack Meridian Health Joseph M. Sanzari Children’s Hospital’s specialists are leading several key research initiatives, including:
- Along with Dana-Farber Cancer Institute in Boston, Derek Hanson, M.D. at Joseph M. Sanzari Children’s Hospital is leading the first clinical study with 2-hydroxyoleic acid (2OHOA) in children with advanced cancer. Goals for the FDA-approved IND (Investigational New Drug) trial include testing the safety and tolerability of 2OHOA in the pediatric population (under 18 years); characterizing the pharmacokinetic profiles in this population; and assessing the preliminary anti-tumor efficacy of the product. While used in Europe for solid tumors in adults, 2OHOA has not been used in the U.S. or in pediatrics.
- Derek Hanson has developed a protocol for embryonal tumor with multilayer rosettes (ETMR), a rare and highly aggressive brain tumor which occurs almost exclusively in young children. Dr. Hanson has created ETMR One, an international registry and research platform for children diagnosed with ETMR. The project has the following aims:
- Collect epidemiologic, clinical, and molecular data of patients with ETMR.
- Evaluate tumor responses and patient outcomes using expert consensus therapy.
- Procure tumor tissue for research of new biology-based therapies for children with ETMR.
- Provide guidance and access to clinical trials for ETMR.
Management of patients with ETMR is complex and requires multidisciplinary expertise in pediatric neuro-oncology, pediatric neurosurgery, neuropathology, neuroradiology, and radiation oncology. The hospital’s medical advisory board reviews the case of every registry participant and provides expert guidance for the clinical management of each individual patient.
ETMR One aims to be a platform for both laboratory and clinical research. To date, the majority of ETMR laboratory research has been performed using a single cell line that is unlikely to be representative of all ETMR cases. Using tumor specimens collected from the registry, researchers are aiming to produce multiple ETMR cell lines that will allow potential therapies to be tested against a range of ETMR models. The data obtained from studies will be used to improve the consensus protocol therapy or used to generate additional biology-based phase I/II trials for ETMR.
- A pediatric trial for Optune® is underway. Optune is a device that creates Tumor Treating Fields (TTFields) to slow or stop glioblastoma tumor cells from dividing, and may destroy them. The device is worn on the head and delivers an electrical shock into the tumor cells without using chemo or radiation. It has been FDA-approved for adults. Joseph M. Sanzari Children’s Hospital is the only hospital in New Jersey participating.