Investigational drugs for the treatment of scleroderma: what's new?

Abstract

Introduction: Systemic sclerosis (SSc) is an orphan, chronic, autoimmune, fibrotic disease with unknown etiology characterized by progressive fibrosis of the skin and internal organs. SSc has the highest mortality, the deadliest among the connective tissue diseases, despite the introduction of new treatment options in the past decades.

Areas covered: The aim of the current systematic review was to investigate new targeted therapy and their impact on disease progression, mainly focusing on phase I and II clinical trials within the past three years.

Expert opinion: Despite recent groundbreaking advancements in understanding SSc pathophysiology, early diagnosis and early introduction of effective targeted treatments within the optimal window of opportunity to prevent irreversible disease damage still represents a significant clinical challenge. Ongoing significant research for new molecular and epigenetics pathways is of fundamental importance to offer new perspectives on disease phenotype and for the development of personalized treatment strategies.

Keywords: clinical trials; investigational drugs; pathogenesis; systemic sclerosis; targeted therapy.