Background: The intense focus on biomarkers is a response to the increasing importance of research focus for ALS diagnosis, disease monitoring, prognosis, prediction of treatment response, and pharmacodynamic response in clinical development programs of novel therapies. The NEALS biorepository organizes the assembly and storage of high-quality, annotated, cross-sectional, and longitudinally collected biofluids and the broad sharing of these samples.
Objectives: The NEALS biorepository provides a reliable framework for collecting, storing, and distributing biofluids and the associated clinical data to ALS researchers, both domestically and internationally.
Results: Between June 2023 and June 2024, NEALS reviewed 20 sample request applications, including 12 from academic investigators and 8 from industry representatives. During this period, the NEALS Biorepository distributed samples for 38 projects, some of which had been reviewed earlier but were fulfilled within this timeframe. A total of 9,943 sample aliquots were distributed, consisting of 5,982 plasma, 1,069 serum, 324 whole blood, and 2,571 CSF aliquots. Currently, the NEALS Biorepository holds 102,933 plasma aliquots, 26,968 serum aliquots, 9,584 CSF aliquots, 14,839 whole blood aliquots, and 1,762 urine aliquots in storage.
Conclusions: The NEALS Biorepository aims to provide high-quality human biofluids to researchers to advance ALS research. Expanding the collection of natural history samples and acquiring samples from phase II and III trials will further support vital efforts to identify prognostic and predictive biomarkers linked to early symptom onset and treatment responses.