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We are living in an era of precision medicine, where scientific innovations have improved drug targeting across various patient populations and most therapeutic areas. However, the growing unmet needs, everchanging landscape, and complex biology of neurological and neuropsychiatric disorders have made precision medicine difficult to apply in clinical neuroscience. Researchers at Janssen are pioneering multiple approaches in neuroscience to address these challenges to reduce the burden and disability caused by serious neuropsychiatric, neurological, and neurodegenerative diseases, while harnessing — and extending — scientific advances in precision medicine. Janssen intends to reconceptualize the diagnosis and treatment of nervous system disorders with the aim to reduce the burden and disability experienced by patients worldwide.

The neuroscience field is poised to leverage data to refine disease categories, such as Alzheimer’s disease and serious forms of depression, and identify the biological drivers of disease in different subpopulations. Together, these data-driven insights enable the design of more precise therapeutic interventions in targeted patient populations. Janssen is focusing on three precision pillars to guide the science behind development of innovative therapies.

  1. Precision in patient identification

From pharmacogenetics in the 1960’s to the Human Genome Project in the 2000’s, precision medicine has come in waves. Over the long history of scientific and conceptual advances in the understanding of disease, the notion of matching a treatment to a patient — with any degree of precision — has been elusive in neuroscience, as diseases of the neurological system are inherently difficult to access and study. Clinical studies must involve the right subset of patient participants to streamline clinical development for neurological diseases.

Advances in data sciences, digital health, and — in particular — biomarkers across diagnostic categories have promise to translate into an ability to select the patients most likely to respond to therapy — or perhaps deselect those unlikely to respond. Biomarkers can exist across many dimensions, including genetic, molecular, histologic, and physiologic characteristics. Janssen’s biomarker team is working to impact precision patient identification by producing powerful diagnostic tools for screening and diagnosis, while improving disease staging — a framework for measuring the severity of disease based on a reliable set of identifiable points1 — monitoring progression and measuring therapeutic effect across its development programs.

For example, the team is evaluating a biomarker-based blood test for diagnosis of Alzheimer’s disease pathology that has the potential to transform disease research by providing a simplified way to identify the right patients for clinical trials.

  1. Precision in target identification

Precision in target identification examines the use of various large datasets and a broad array of high-throughput tools, computation, and sequencing, to understand the molecular basis of disease in different ways, therefore providing greater understanding into patient-specific mechanisms that cause or contribute to disease, ultimately enabling precise targeting of these mechanisms. Guided by a greater understanding of nervous system diseases, engaging biology that may be present across disease states and indications may enable “pipeline in product” approaches.

Janssen is working to match novel mechanisms of diseases to people with subtypes of illnesses to address significant unmet needs. For example, nearly 70% of patients with major depressive disorder (MDD) experience residual symptoms with first line standard of care.2 These may include anxiety, impaired cognition, fatigue, and anhedonia.

It is believed that these symptoms can be mediated by changes in distinct brain circuits and can be improved by targeting proteins expressed within these circuits. With sleep disturbance, for instance, orexin-producing neurons are thought to be involved in the regulation of sleep and waking activities, as well as emotions.3 Janssen’s latest research is evaluating this kind of target identification as a potential adjunctive therapy for such individuals, particularly for those who have been inadequately helped by existing treatments.

  1. Precision in therapeutic focus

Precision in therapeutic focus aims to determine the best approach to prevent, diagnose, and treat disease, in which what is measured is linked to outcomes and relevant clinical unmet needs. Janssen recently acquired a molecule with the potential to address a range of serious autoantibody-mediated autoimmune diseases, from neurologic to hematologic diseases, including generalized myasthenia gravis. It is expected to lower the levels of autoantibodies circulating in the bloodstream and ease the symptoms associated with such disorders.

In building on its 60-year legacy in neuroscience, Janssen remains committed to advancing science across a wide range of nervous system disorders. By delivering differentiated therapies in areas of critical unmet need and by creating and leveraging advances in precision medicine across several key areas, Janssen aims to lead the precision revolution in neuroscience to reduce the burden and disability caused by serious nervous system disorders. Learn more.

Bill Martin, Ph.D., is Global Therapeutic Area Head, Neuroscience, Janssen Research & Development, LLC.


1 Gonnella, J. S., Hornbrook, M. C. & Louis, D. Z. Staging of disease: a case-mix measurement. JAMA 251, 637–644 (1984).

2 Rush AJ, et al. Acute and longer-term outcomes in depressed outpatients requiring one or several treatment steps: A STAR*D report. Am J Psychiatry. 2006;163(11):1905-17. doi: 10.1176/ajp.2006.163.11.1905.

3 Shariq AS, et al. Evaluating the role of orexins in the pathophysiology and treatment of depression: A comprehensive review. Prog Neuropsychopharmacol Biol Psychiatry. 2019; Jun 8;92:1-7. doi: 10.1016/j.pnpbp.2018.12.008.