Phospholipidosis Biomarkers
Drug-induced phospholipidosis represents a concern in risk assessment. There is the absence of information related to the prevalence and time course of the condition in humans. A readily accessible biomarker in the urine or blood is urgently needed for routine phospholipidosis assessment. Di-docosahexaenoyl (22:6)-bis(monoacylglycerol) phosphate (di-22:6-BMP) was identified and patented by Nextcea Inc (Woburn, MA) as a non-invasive biomarker to evaluate DIPL in animals and humans. BMP is a phospholipid increased in the tissues of patients with DIPL. BMP is localized within the intra-vesicular vesicles of late endosomes and lysosomes where it plays a role in phospholipid and cholesterol trafficking. Di-22:6-BMP in the blood or urine may provide a non-invasive marker for routine diagnosis/screening and research on the role of phospholipidosis in the etiology of drug-induced toxicities. The FDA has formed a PL working group to address concerns related to DIPL and develop policy recommendations. FDA has published a paper to support the use of di-22:6 BMP as an effective biomarker to predict drug-induced phospholipidosis. Di-22:6-BMP may be used as a biomarker of DIPL to support development of guidance for industry and regulatory reviewers on how to proceed with drug development when DIPL is observed in preclinical and clinical regulatory studies.
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