Verteporfiinia

Verteporfiinia is a synthetic compound belonging to the class of drugs known as heat shock protein 90 (HSP90) inhibitors. It was initially developed as an anticancer agent and has been studied extensively for its potential therapeutic applications in oncology. The drug functions by binding to the ATP-binding pocket of HSP90, a molecular chaperone that assists in the proper folding and stabilization of various client proteins, including several oncogenic kinases. By inhibiting HSP90, verteporfin disrupts the function of these client proteins, leading to their degradation and ultimately inducing cell cycle arrest or apoptosis in cancer cells.

Beyond its role in cancer treatment, verteporfin has gained attention for its effects on non-cancerous biological processes. It has been shown to modulate the activity of the transcription factor YAP (Yes-associated protein), which plays a critical role in the Hippo signaling pathway—a key regulator of organ size, cell proliferation, and tissue homeostasis. By inhibiting YAP, verteporfin influences cellular behaviors such as migration, invasion, and stemness, making it a subject of interest in regenerative medicine and fibrosis research.

In clinical practice, verteporfin is most notably used in the form of Visudyne, a photosensitizing agent approved for the treatment of subfoveal choroidal neovascularization (CNV) associated with age-related macular degeneration (AMD). When activated by visible light, verteporfin generates reactive oxygen species that damage abnormal blood vessels, leading to their occlusion and subsequent regression. This photodynamic therapy approach is minimally invasive and targets specific pathological vessels without affecting healthy tissue.

While verteporfin shows promise in multiple therapeutic areas, its clinical use is currently limited by challenges such as off-target effects, resistance mechanisms, and formulation constraints. Ongoing research continues to explore its potential in combination therapies and broader applications, including inflammatory diseases and degenerative conditions.