The TransBioLine consortium has been working for the past five years on discovery, validation and regulatory qualification of a variety of safety biomarkers for drug-induced organ injury.

On Friday, February 9, 2024, the first Biomarker Qualification Plan from TransBioLine on Drug Induced Nervous System Injury was submitted to the FDA.

Work Package 5, focused on drug-induced nervous system toxicity, has validated several sensitive serum biomarkers on the Quanterix platform in multiple clinical cohorts and nonclinical models. The team made a data-driven decision to focus on Neurofilament Light Chain (NFL) as a promising biomarker to bring forward into regulatory qualification. If NFL can be fully qualified for its intended context of use, it would be the first of its kind as a blood-based monitorable endpoint to prevent serious nervous system injury and facilitate advancing potential breakthrough therapies safely into the clinic.

Other outputs with contributions from this team have included two key publications highlighting NFL as a biomarker of drug-induced nervous system injury. The first, published in JCI Insight, demonstrated a correlation of serum NFL with chemotherapy-induced polyneuropathy in patients with breast or ovarian cancer (Neurofilament proteins as a potential biomarker in chemotherapy-induced polyneuropathy). In January, an evaluation of NFL and other biomarkers of nervous system toxicity in 15 different nonclinical models was published in Frontiers in Neuroscience (Performance of biomarkers NF-L, NSE, Tau and GFAP in blood and cerebrospinal fluid in rat for the detection of nervous system injury).

This key milestone will contribute to increasing patient safety and to streamline drug-development processes, the main goal of TransBioLine.