DFMO was developed in 1979 but has never been tested in combination with cytotoxic drugs in tumour types such as neuroblastoma where it is likely to be impactful.
Neuroblastoma is a common solid tumour of childhood, often presenting with advanced disease. Despite maximally-intensified treatment (surgery, chemo, radiotherapy, and biotherapy) most patients will ultimately die. An international team of researchers has focused on understanding the mechanisms of tumorigenesis in neuroblastoma to develop innovative therapeutic strategies that target key components of neuroblastoma progression.
DFMO is an approved drug for clinical use in trypansomic encephalitis (“sleeping sickness”) and has been studied extensively in the paediatric population where it is well tolerated. Further, there has been a resurgence of interest in DFMO as a preventative agent in adult cancers when used at low dosages. However, there is no data describing either the efficacy or toxicity of DFMO in children with cancer. Given dramatic effects demonstrated in extensive preclinical testing, there is compelling rationale to test these findings in the clinic.
This study is therefore the ideal means to translate years of research into a new, exciting and potentially safer treatment approach for children with relapsed neuroblastoma who are otherwise destined to die of their disease.