Emerging therapies for juvenile arthritis: agents in early clinical trials

ABSTRACT

Introduction

Juvenile idiopathic arthritis (JIA) is the most common chronic inflammatory rheumatic condition in childhood. The management of JIA has been revolutionized thanks to the development of new powerful drugs and the possibility to conduct controlled clinical trials with support from legislative initiatives and availability of international collaborative networks. Trials are still needed in children because we now have new drugs related to specific JIA category.

Areas covered

The review is centered on the latest achievements in the field, focusing on new investigational drugs which are currently or have been recently tested for JIA treatment, encompassing agents in early phase of clinical development.

Expert opinion

Despite the tremendous improvement witnessed in the field of JIA treatment in the past 20 years, there are still many unmet needs to be prioritized. Studies on disease pathogenesis will hopefully help in the identification of new treatment targets for individual JIA categories, that could possibly favor a stricter disease control and contribute to solve the issue of refractory JIA. Novel strategies aimed at the prevention of the risk of long-term joint damage are also desirable, as well as the discovery of predictive biomarkers for treatment efficacy and safety in the individual patient.

KEYWORDS:

• Biologics
• clinical trials
• investigational drugs
• JAK inhibitor
• juvenile idiopathic arthritis
• small molecules

Article highlights

• JIA treatment has improved enormously thanks to appropriate legislative initiatives in paediatric drug development, the implementation of large collaborative research networks, and the introduction of new potent therapeutic agents

• Biological drugs that selectively block target pathogenetic cytokines have tremendously improved JIA management, and many paediatric investigation plans are in place for new investigational agents

• Agents in early clinical trials consist in both new anti-cytokine drugs and oral small molecules

• New treatment options are under evaluation for a more personalized management of JIA in order to improve disease outcome and to address the unmet medication need of a portion of JIA population

• Well-established biomarkers may help foster more rational and efficacious therapeutic strategies and minimize the risk of treatment-related adverse events

• A new approach to define biological categories within JIA may enable the partition of patients into subsets amenable to mechanism-based intervention

• Ethical issues, trials on biosimilars and me-too drugs, clinical study design, and greater involvement of patients and parents are some of the points to be targeted by future research

Acknowledgments

The authors thank Chiara Pallotti for the editorial assistance (coordination among co-authors, bibliography, English revision).

Declaration of interest

N Ruperto has received honoraria for consultancies or speaker bureaus from the following pharmaceutical companies in the past 3 years: Ablynx, Amgen, Astrazeneca-Medimmune, Aurinia, Bayer, Bristol Myers Squibb, Cambridge Healthcare Research (CHR), Celgene, Domain therapeutic, Eli-Lilly, EMD Serono, GlaxoSmithKline, Idorsia, Janssen, Novartis, Pfizer, Sobi, and UCB Pharma. The authors have no other 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 apart from those disclosed

Reviewer disclosures

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

Additional information

Funding

This paper was not funded.