Metastatic retinoblastoma and L-phenylalanine mustard-dianhydrogalactitol

Retinoblastomas heterotransplanted to nude mice were shown to be sensitive to a combination of phenylalanine mustard and dianhydrogalactitol. A patient with metastatic retinoblastoma was treated with this combination and had a significant response with complete clinical clearance of her bone marrow and metastatic tumors. However, the patient's response was similar to that observed in nude mice; recurrences of tumors developed after complete clinical remission.