Hepatocellular carcinoma (HCC) is a deadly tumor for which the incidence is increasing in
the United States, primarily due to prevalence of hepatitis C infection. An important
aspect of the development of HCC is that it occurs in patients who have underlying cirrhosis
of the liver, thereby limiting the therapeutic options. There is potential curative
treatment for these patients, such as resection of the tumor lesion and liver
transplantation, but these treatments are feasible in a small percent of patients only.
Furthermore, the majority of the patients with HCC are also not candidates for palliative
treatments such as percutaneous ablation of the tumor, chemotherapy or radiation.
Additionally, it has been shown that these palliative treatment modalities do not alter
survival, and are associated with significant risks. Therefore, there are no treatment
options for most patients with HCC. A new theory has emerged in the fight against cancer
through inhibition of angiogenesis (development of new blood vessels). The hypothesis being
that if there is no blood supply "feeding" the tumor cells cannot divide or survive. One
such approach, pioneered in this institution by Drs. George Brewer and Sofia Merajver, is
the anticopper approach using the medication tetrathiomolybdate (TM). By creating a mild
copper deficiency state, several pathways required for angiogenesis are inhibited. They
performed a Phase I trial in which patients with metastatic cancer were treated with TM
resulting in decrease tumor vascularity. TM had excellent safety profile in this patient
population. HCC is well known to be a hypervascular tumor. An antiangiogenesis approach
might provide a novel treatment for this HCC. This is a pilot study of 10 patients with HCC
who are not candidates for curative surgical therapy with resection or liver
transplantation, nor for ablative techniques. Patients seen in the General Liver clinic and
Liver Transplant clinic who have an overall good performance status, with an expected
survival of more than 6 months will be enrolled. After an initial evaluation, they will be
given 120 mg/day of TM in divided doses for one year. The size and vascularity of the tumor
will be evaluated by magnetic resonance imaging (MRI). The primary outcome of this study is
to prevent tumor progression.
- Patients with definite diagnosis of HCC based on a Dynamic MRI revealing a vascular
mass in the liver and histologic confirmation of HCC.
- Patients who are not candidates for surgical resection, OLT or radiofrequency
- Patients with no prior treatment for HCC.
- Patients with extrahepatic spread and/or vascular invasion are permissible.