Nearly 50% of patients with high-risk neuroblastoma suffer a relapse of their disease despite intensive upfront treatment. Unfortunately, relapsed high-risk neuroblastoma remains incurable in the large majority of patients. We have initiated a phase 1/1b investigational trial for patients with relapsed/refractory neuroblastoma, which provides patients with treatment that matches the genetic profile of their tumor. The NEPENTHE trial; NExt generation PErsonalized Neuroblastoma THErapy. In part one of this trial, a patient's tumor is sent for next generation sequencing-based cancer genomic profiling (NGS). Patients who have mutations in the anaplastic lymphoma kinase oncogene (ALK), RAS/MAPK pathway or who have TP53 wild-type neuroblastomas are eligible for this treatment (TP53 wild-type).
Participation in such an innovative clinical trial requires a significant commitment from patients and their families. The majority of patients are required to undergo biopsies of their tumors and frequent disease re-evaluations/testing throughout the trial. They need to spend a large amount of time away from their home, family, and employment. We have a multidisciplinary team dedicated to the NEPENTHE Trial that supports the patient and family as they manage the medical, social, emotional, and financial consequences associated with a potentially complicated treatment course and schedule. We utilize various foundations/organizations to cover family's travel and other out-of-pocket expenses incurred during the patient's treatment course. Ongoing education is provided to both the patient and family as well as the referring medical team to maintain a focus on collaboration and on-going patient support.
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