LAUSR

* Abbreviation: FDA — : Food and Drug Administration I sat across from my patient with recently confirmed relapsed pulmonary osteosarcoma. Having previously endured multiple rounds of chemotherapy, an amputation, and debilitating and, at times, recalcitrant phantom limb pain, he had slowly pieced his life back together and had returned to some semblance of normalcy; he now had a job, was living on his own, and was attending university. Although osteosarcoma is the most common primary bone cancer affecting children and young adults, it is still a rare disease, with only ∼400 new cases each year in the United States. Osteosarcoma has a predilection for spreading to the lungs and, once there, is considerably more difficult to cure. Treatment of osteosarcoma includes combination chemotherapy and surgical resection. Survival rates have remained stagnant for >30 years.1 If there ever were a disease in need of novel approaches and new therapies, osteosarcoma is it. In discussing with my patient how best to move forward, I explained that traditional cytotoxic chemotherapy no longer seemed like a viable option and suggested that he consider a clinical trial investigating an innovative approach. We discussed various novel therapies before settling on a phase II study investigating an immunotherapeutic administered with cytokine support.2 In discussing the trial, I explained that phase II trials seek to determine if an experimental therapy is effective and safe in larger numbers of patients, and that there exists the possibility for both known and unforeseen harms. I shared my concerns regarding his existing phantom limb pain and the experimental therapy’s penchant to … Address correspondence to Yoram Unguru, MD, MS, MA, Division of Pediatric Hematology/Oncology, The Herman and Walter Samuelson Children’s Hospital at Sinai, 2401 West Belvedere Ave, Baltimore, MD 21215-5271. E-mail: yunguru{at}lifebridgehealth.org