Emerging drugs and therapeutics for systemic sclerosis

Abstract

Treatment of systemic sclerosis (SSc) is challenging despite advances in medical therapeutics for other rheumatologic diseases. Significant disease modifying therapy is lacking for most patients with SSc, due to the heterogeneous multisystem nature of SSc and its complex pathophysiology. The emergence of organ based management strategies has provided guidance in patient care as well as research and drug development. Areas covered: Design and development of new compounds focused on the underlying fibrotic disease processes have been sparse. Therefore, organ based strategies with targeted approaches have been directed towards the most devastating and life threatening features of systemic sclerosis. These include pulmonary arterial hypertension, interstitial lung disease, peripheral vasculopathy and skin thickening. In this context, new treatment regimens using older drugs as well as discovery of novel compounds based on recent insights of the disease pathophysiology are discussed. Expert opinion: Systemic sclerosis is a heterogeneous rare disease that carries a high burden of morbidity and mortality. Organ based management strategies have improved the natural history of systemic sclerosis using targeted interventions or strategies, particularly vascular features. However, more research is required to better understand disease mechanisms, including an ultimate unifying pathway that explains the multisystem nature of systemic sclerosis.

Keywords: Systemic sclerosis; autoimmunity; cytokine signaling; fibrosis; interstitial lung disease; microRNA; pulmonary arterial hypertension; tyrosine kinase inhibitors; vascular dysfunction.