Drug development and novel therapeutics to ensure a personalized approach in the treatment of systemic sclerosis

ABSTRACT

Introduction

Systemic sclerosis (SSc) is a systemic disease encompassing autoimmunity, vasculopathy, and fibrosis. SSc is still burdened by high mortality and morbidity rates. Recent advances in understanding the pathogenesis of SSc have identified novel potential therapeutic targets. Several clinical trials have been subsequently designed to evaluate the efficacy of a number of new drugs. The aim of this review is to provide clinicians with useful information about these novel molecules.

Area covered

In this narrative review, we summarize the available evidence regarding the most promising targeted therapies currently under investigation for the treatment of SSc. These medications include kinase inhibitors, B-cell depleting agents, and interleukin inhibitors.

Expert opinion

Over the next five years, several new, targeted drugs will be introduced in clinical practice for the treatment of SSc. Such pharmacological agents will expand the existing pharmacopoeia and enable a more personalized and effective approach to patients with SSc. Thus, it will not only possible to target a specific disease domain, but also different stages of the disease.

KEYWORDS:

• Biologic agents
• interleukins
• interstitial lung disease
• systemic sclerosis
• targeted therapies

Article highlights

• The pathogenesis of systemic sclerosis encompasses vasculopathy, immune system dysregulation and fibrosis, all of which are potential therapeutic targets.

• Despite recent advances, there are still a significant unmet needs in regard to the treatment of systemic sclerosis, particularly in refractory and more severe forms.

• Several compounds are currently under investigation for the treatment of systemic sclerosis with promising results and will possibly implement the available pharmacopoeia in the coming years.

• The constant identification of new molecular targets and pharmacological agents is pivotal for clinical to have at their disposal an adequate choice when deciding how to treat patients with systemic sclerosis, especially in more difficult-to-treat cases.

• Ideally, five years from now, clinicians will be able to treat patients with systemic sclerosis with different, targeted thereapeutic schemes according to the stage of the disease and the type of organ involvement.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers in this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.