Does Mitotane have a therapeutic effect on liver cancer?

Mitotane(Mitotane) is a synthetic derivative originally derived from the chemical structure of the pesticide DDT, but was developed in medicine as a drug with specific anti-tumor effects. It mainly targets adrenocortical cells and can reduce the synthesis of adrenal hormones by damaging mitochondrial function and inhibiting related enzyme activities. Therefore, it is approved by the US FDA and the European EMA for the treatment of functional or non-functional adrenocortical carcinoma (ACC; adrenal cancer), especially in patients with unresectable or postoperative recurrence, mitotane is regarded as the first-line standard drug.

In response to the question of "whether mitotane has a therapeutic effect on liver cancer" needs to be analyzed from the perspective of drug mechanism and clinical application. Mitotane's selectivity is primarily focused on adrenocortical cells, rather than targeting specific pathways in hepatocytes or liver cancer. It works by inhibiting the production of adrenocortical hormones and killing adrenal cells, which is inconsistent with the common molecular mechanisms of liver cancer. Therefore, mitotane is not recommended for the treatment of hepatocellular carcinoma (HCC) in international clinical guidelines. Current standard treatments for liver cancer mainly include surgical resection, local ablation, transarterial chemoembolization (TACE), targeted therapy, and immune checkpoint inhibitors, but mitotane does not appear in relevant guidelines or drug lists.

However, from a pharmacological perspective, mitotane is metabolized by the liver in the body and has certain effects on the liver. Some scholars speculate that its metabolic characteristics may indirectly affect the pathological environment of the liver, but this effect is not sufficient to serve as a basis for the treatment of liver cancer. There are currently no large randomized clinical trials confirming the efficacy of mitotane in patients with liver cancer, so it cannot replace existing liver cancer treatment options.

In summary, the therapeutic indications of mitotane are clearly limited to adrenocortical cancer, not liver cancer. For patients with suspected liver cancer, international standard treatment paths should be followed and mitotane should not be used by oneself.

Reference materials:https://go.drugbank.com/drugs/DB00648