Opioid growth factor (OGF) for hepatoblastoma: a novel non-toxic treatment

• Published in: Investigational new drugs 2013-08, Vol.31 (4), p.1066-1070
• Main Authors: Rogosnitzky, Moshe, Finegold, Milton J., McLaughlin, Patricia J., Zagon, Ian S.
• Format: Article
• Language: English
• Subjects: Abdomen; Adjuvants; Age; Antineoplastic agents; Biological and medical sciences; Biopsy; Cancer therapies; Chemotherapy; Chemotherapy, Adjuvant; Child; Child, Preschool; Drug dosages; Drug therapy; Enkephalin, Methionine - therapeutic use; Female; Gastroenterology. Liver. Pancreas. Abdomen; General aspects; Growth factors; Hearing loss; Hepatoblastoma - diagnostic imaging; Hepatoblastoma - drug therapy; Histology; Humans; Hypertension; Infant; Infant, Newborn; Kidney diseases; Liver; Liver cancer; Liver. Biliary tract. Portal circulation. Exocrine pancreas; Magnetic resonance imaging; Medical sciences; Medicine; Medicine & Public Health; Multiple tumors. Solid tumors. Tumors in childhood (general aspects); Narcotics; Neutropenia; Oncology; Opioid Peptides - therapeutic use; Other diseases. Semiology; Parents & parenting; Patients; Pediatrics; Pharmacology. Drug treatments; Pharmacology/Toxicology; Pneumonia; Pregnancy; Sepsis; Short Report; Studies; Surgeons; Toxicity; Tumors; Ultrasonic imaging; Ultrasonography
• ISSN: 0167-6997; 1573-0646
• DOI: 10.1007/s10637-012-9918-3

Summary Hepatoblastoma is the most common liver malignancy 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 ultrasound. After subsequent diagnosis of hepatoblastoma, she was treated with one course of neoadjuvant chemotherapy at approximately 1 week of age. Following significant complications from the chemotherapy (neutropenic fever, pneumonia and sepsis), the patient’s parents declined further chemotherapy, 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 hepatoblastoma. 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 neoadjuvant 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 promising safe and therapeutic avenue for effective treatment of hepatoblastoma.