SHORT REPORT Opioid growth factor (OGF) for hepatoblastoma: a novel non-toxic treatment Moshe Rogosnitzky & Milton J. Finegold & Patricia J. McLaughlin & Ian S. Zagon Received: 26 November 2012 / Accepted: 17 December 2012 / Published online: 30 December 2012 # Springer Science+Business Media New York 2012 Summary Hepatoblastoma is the most common liver malig- nancy in children, typically diagnosed before age 2. The survival rate for hepatoblastoma has increased dramatically in the last 30 years, but the typical chemotherapeutic agents used for treatment are associated with significant toxicity. In this report, the authors present two cases of hepatoblastoma treated with surgical resection and a novel biotherapeutic regimen that included opioid growth factor (OGF). Case #1 is an infant diagnosed with a large mass on prenatal ultra- sound. After subsequent diagnosis of hepatoblastoma, she was treated with one course of neoadjuvant chemotherapy at ap- proximately 1 week of age. Following significant complica- tions from the chemotherapy (neutropenic fever, pneumonia and sepsis), the patient’ s parents declined further chemother- apy, and the infant was treated with surgical resection and opioid growth factor (OGF)/low dose naltrexone (LDN). She is currently at close to 10 years disease–free survival. Case #2 is a child diagnosed with a liver mass on ultrasound at 20 months of age, later biopsy-proven to represent hepato- blastoma. Due to existing co-morbidities including autosomal recessive polycystic kidney disease and hypertension, and indications from the biopsy that the tumor might be insensitive to chemotherapy, the parents elected not to proceed with neo- adjuvant chemotherapy. The patient was treated with surgical resection and OGF/LDN, and is currently at more than 5 years disease-free survival. This case series highlights the need for less toxic treatment options than conventional chemotherapy. Modulation of the OGF-OGF receptor axis represents a prom- ising safe and therapeutic avenue for effective treatment of hepatoblastoma. Keywords Hepatoblastoma . Opioid growth factor . Toxicity . Naltrexone . LDN . Disease-free survival Introduction Malignant liver tumors account for slightly greater than 1 % of pediatric malignancies. Unlike the adult population, where the predominant histology is hepatocellular carcinoma, two-thirds of pediatric liver malignancies are hepatoblastomas, arising from embryonal cells [1]. The median age for diagnosis of hepatoblastoma is 18 months [2]. The initial clinical presen- tation varies, but usually involves the detection of a painless abdominal mass by a physician or parent. In more advanced cases, decreased appetite, failure to thrive, and jaundice may also be present [2]. Most cases of hepatoblastoma are sporad- ic, but associations do exist with multiple genetic syndromes, including Beckwith-Wiedemann syndrome, familial adeno- matous polyposis and trisomy 18 [3]. The 5-year overall survival rate for hepatoblastoma has improved dramatically, from approximately 35 % three dec- ades ago to 75–80 % currently [4]. This increase in survival may reflect the shift away from isolated surgical resection to a combination of surgical resection and chemotherapy [4, 5]. The mainstay of chemotherapy for hepatoblastoma is cis- platin, usually combined with either vincristine or doxoru- bicin. Depending on the treatment protocol and the initial staging of disease, the patient may receive both neoadjuvant and adjuvant chemotherapy, or adjuvant chemotherapy alone following surgical resection [2]. Chemotherapy regimens incorporating cisplatin result in response rates of up to 93 % 6 , but treatment can also result M. Rogosnitzky (*) MedInsight Research Institute, P.O. Box 386, Telz Stone 90840, Israel e-mail: moshe@medinsight.org M. J. Finegold Department of Pathology, Texas Children’ s Cancer Center at Baylor College, Houston, TX 77030, USA P. J. McLaughlin : I. S. Zagon Department of Neural & Behavioral Science, The Pennsylvania State University, College of Medicine, Hershey, PA 17033, USA Invest New Drugs (2013) 31:1066–1070 DOI 10.1007/s10637-012-9918-3