Biomarker-driven development of new therapies for autoimmune diseases: current status and future promises

ABSTRACT

Introduction

Auto-immune diseases are complex and heterogeneous. Various types of biomarkers can be used to support precision medicine approaches to autoimmune diseases, ensuring that the right patient receives the most appropriate therapy to improve treatment outcomes.

Areas covered

We review the recent progress made in modeling several autoimmune diseases such as Systemic Lupus Erythematosus, primary Sjogren Syndrome, and Rheumatoid Arthritis following extensive molecular profiling of large cohorts of patients. From this knowledge, BMKs are being identified which support diagnostic as well as patient stratification and prediction of response to treatment. The identification of biomarkers should be initiated early in drug development and properly validated during subsequent clinical trials. To ensure the robustness and reproducibility of biomarkers, the PERMIT Consortium recently established recommendations highlighting the importance of relevant study design, sample size, and appropriate validation of analytical methods.

Expert opinion

The integration by AI-powered analytics of massive data provided by multi-omics technologies, high-resolution medical imaging and sensors borne by patients will eventually allow the identification of clinically relevant BMKs, likely in the form of combinatorial predictive algorithms, to support future drug development for autoimmune diseases.

KEYWORDS:

• Autoimmune diseases
• biomarker
• disease modeling
• precision medicine
• targeted treatment

Article highlights

• Various types of candidate biomarkers have been identified to facilitate drug development in Systemic Lupus Erythematosus, primary Sjogren Syndrome and Rheumatoid Arthritis.

• Biomarkers can potentially be used in autoimmune diseases to document susceptibility/risk, diagnostic, monitoring, prognostic, treatment response and safety.

• Many challenges are being faced in the identification and validation of candidate biomarkers, most particularly to establish their clinical relevance in informing therapeutic decisions.

• There is an emerging interest in digital BMKs capturing patient-reported outcomes via connected digital devices borne by patients with AIDs.

• Within ten years, the combined use of biotechnologies, high-resolution imaging and advanced computational analytics will yield numerous clinically relevant BMKs increasing dramatically the success rate in the development of innovative treatments for AIDs.

Declaration of interest

All authors are employees of Servier. 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.

Abbreviations

AI: Artificial intelligence; AIDs: Autoimmune diseases; BMK: Biomarker: PM: CDU: Context of use; IFN: Interferon; IFNGS: Interferon gene signature; Precision medicine; pSS: Primary Sjogren syndrome; RA: Rheumatoid arthritis; SLE: Systemic Lupus Erythematosus; RF: Rheumatoid factor

Additional information

Funding

This paper was not funded.