Experimental Drug May Induce Effective Responses in Patients With Liver Cancer, Early Study Findings Show

A small group of patients with early- or intermediate-stage hepatocellular carcinoma, the most common type of primary liver cancer, that cannot be surgically resected, derived a benefit from treatment with an experimental drug, according to findings from an early-phase trial.

“The current standard of care for unresectable liver cancer is conventional transarterial chemoembolization (TACE) with the chemotherapeutic agent doxorubicin,” Dr. Chang-Hsien Liu, the primary investigator of the study, said in a press release announcing the findings. “Though well established, TACE with doxorubicin has limited efficacy because chemotherapy does not effectively kill cancer stem cells, a root cause for tumor recurrence after TACE. Additionally, embolization-induced tumor hypoxia (low levels of oxygen) is thought to dampen the cytotoxic (cell-damaging) effects of doxorubicin.”

According to the National Cancer Institute, a transarterial chemoembolization is a minimally invasive procedure in which the blood supply to a tumor or an abnormal area of tissue is blocked. Members of the patient’s health care team make a small call in the inner thigh. Then, a thin, flexible tube is inserted into the incision and guided into an artery near the tumor or abnormal tissue and the treatment is subsequently delivered.

In this phase 1 study, 17 patients with early- or intermediate-stage hepatocellular carcinoma were all given treatment with an experimental anti-cancer drug known as tirapazamine delivered via transarterial chemoembolization. Patients enrolled onto the trial were allowed to have received previous treatment.

The findings — which were published in The Journal of Vascular and Interventional Radiology — showed that treatment with the experimental anti-cancer drug tirapazamine resulted in a complete response (meaning that the cancer completely responded to treatment) in eight of the 17 patients, or 47.1% of the study population.

Additionally, three patients achieved a partial response. The early findings also showed that this treatment was associated with a six-month progression-free survival (percentage of patients alive without disease progression) rate of 72.6% and a median overall survival (length of time a patient is alive without death from any cause) of 29.3 months.

In the press release, Liu noted that these results show that the experimental anti-cancer drug tirapazamine could be a viable alternative to the chemotherapy doxorubicin.

“The results of this study support tirapazamine as an alternative to doxorubicin in trans-arterial chemoembolization,” he said. “A future randomized study is warranted to compare tirapazamine and doxorubicin under embolization.”

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