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Case Report

Case Study Case Rep. 2012; 2(2): 38-45

CPI-613, an investigational novel anti - cancer agent, provides long- term stable disease without significant adverse effects in a patient with stage IV relapsed hepatocellular carcinoma

Neil Senzer; Cynthia Bedell; Claudia Maturo; John Luddy; Robert Rodriguez; Robert Shorr; King Lee.

The current case study describes the safety and prolonged therapeutic effect of an investigational drug (CPI-613) in a patient with advancing relapsed hepatocellular carcinoma. The patient is a 73-year-old Caucasian male diagnosed with advancing Stage IV metastatic hepatocellular carcinoma. He had been treated with, and relapsed from, intra-arterial intrahepatic chemotherapy infusion therapy, sorafenib (Nexavar®), and an investigational drug CUDC-101. At the time of study entry, his disease was progressing. The liver was ~16 cm in size, had multiple heterogeneous abnormal signal lesions throughout, and had near complete replacement of parenchyma with tumor. There was a discrete mass (~5.2 cm) in the inferior right hepatic lobe, and another (~8.3 x 4.2 cm) in the inferior tip of the right hepatic lobe. There was one lesion (~2.4 cm) in the inferior spleen, and two (~2 cm and ~4.5 cm) in the superior spleen. There was also a left mid-abdominal mesenteric mass (~6.9 x 6.7 cm). During the 10 months of treatment with CPI-613, he maintained his performance status and the disease was stable. CPI-613 related adverse events were transient and of mild-to-moderate severity, and included fatigue, nausea, weakness, elevation of troponin I, anemia and elevation of hepatic enzymes.
CPI-613 therapy provided long-term Stable Disease and with minor transient adverse events in a patient with advancing T2 Stage IV relapsed hepatocellular carcinoma. This is an uncommon case where chemotherapy with an investigational drug is providing long-term stabilization of advanced metastatic relapsed hepatocellular carcinoma without significant adverse events.

Key words: Metastatic relapsed hepatocellular carcinoma;CPI-613;cancer cell mitochondrial energy metabolism;prolonged Stable Disease

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