Updates on neonatal cell and novel therapeutics: Proceedings of the Second Neonatal Cell Therapies Symposium (2024)

Abstract: Cell therapies as treatments for neonatal conditions have attracted significant research and parent interest over the last two decades. Mesenchymal stromal cells, umbilical cord blood cells and neural stem cells translate from lab, to preclinical and into clinical trials, with contributions being made from all over the world. Effective and timely translation involves frequent reflection and consultation from research-adjacent fields (i.e. cell therapies for cerebral palsy, adult neurology, companies, and regulatory bodies) as well as meaningful involvement of people with lived experience. Progress to date suggests that aligning outcome and data reporting in later phase clinical trials will support our sector, as well as involving industry partners for streamlined solutions in cell manufacturing, commercialisation and regulatory processes. Importantly, our field can also benefit from resource sharing and research collaboration in novel drug therapies, small molecules and extracellular vesicles as we attempt to bridge preclinical and clinical research. In this review, we present highlights and learnings from the second Neonatal Cell Therapies Symposium (2024), held in Sydney, Australia. Impact: Multiple cell therapy candidates have advanced through preclinical and clinical trials in neonatology, showing promising feasibility, safety and efficacy. Effective and timely translation is enabled by collaboration across research-adjacent fields, commercial partnerships, harmonising research outcomes and meaningful involvement of people with lived experience. Progress on the potential utility of cell therapies for neonatal conditions and further translational considerations are discussed in this paper.