Etarfolatide

Etarfolatide is an investigational antifolate drug developed for the treatment of cancer. It is a folate-analog conjugate designed to target cancer cells that overexpress folate receptors. These receptors are commonly found on the surface of various types of tumors, including ovarian, breast, lung, and brain cancers. By binding to these receptors, etarfolatide aims to deliver a cytotoxic payload specifically to cancerous cells, sparing healthy tissues and potentially reducing side effects.

The drug's mechanism of action involves inhibiting dihydrofolate reductase (DHFR), an enzyme crucial for DNA synthesis and cell replication. Cancer cells, due to their rapid proliferation, often have a higher demand for folate and thus express more folate receptors. Etarfolatide's design leverages this difference to achieve selective tumor targeting. It is typically administered intravenously.

Clinical trials have been conducted to evaluate the safety and efficacy of etarfolatide in patients with advanced or recurrent solid tumors. These studies have explored its use as a monotherapy and in combination with other anti-cancer agents. While early research showed promise, further investigation is ongoing to determine its definitive role in cancer treatment. Regulatory approval for etarfolatide has not yet been granted.