Novel investigational agents for the treatment of scleroderma

Zsuzsanna Hortobagyi McMahan, Fredrick M. Wigley

Research output: Contribution to journalReview articlepeer-review

11 Scopus citations


Introduction: The purpose of this article is to highlight novel therapies that are being used in scleroderma (SSc). Therapeutic interventions in SSc generally target at least one of three ongoing biological processes characteristic of the disease: vasculopathy, autoimmunity and tissue fibrosis. Treatment decisions in SSc are determined by the level of disease activity and the degree of specific organ involvement. Traditional therapy has primarily focused on organ-specific management without clear evidence of overall disease modification. Areas covered: The authors provide a review of a variety of agents, which are already used for other autoimmune diseases, that are now being used to treat active SSc skin or lung disease, including rituximab, tocilizumab and IVIG. Several agents studied in vitro and in animal models of fibrosis have shown promise, including bortezomib, LPA-1 antagonists, anti-CCN2 therapy, anti-IL-13 and thrombin antagonists. The authors also provide details on targeting intracellular molecular pathways and matricellular proteins, which is another novel area of investigation. Expert opinion: Combination therapy may be necessary to control the complex biological network active in SSc. Most of the current evidence that suggest benefit of these agents is based on small population studies. Ultimately well-designed clinical trials are required to define the role of these agents in treating SSc.

Original languageEnglish (US)
Pages (from-to)183-198
Number of pages16
JournalExpert Opinion on Investigational Drugs
Issue number2
StatePublished - Feb 2014


  • Drugs
  • Immunomodulatory
  • Novel therapies
  • Scleroderma
  • Systemic sclerosis
  • Treatment

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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