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Official PDF TranslationStem Cell Research & Therapy

A phase I, open-label study of intravenous human dental pulp stem cells (NestaCell®) at two dose levels in patients with Huntington’s disease

Authors: Joyce Macedo Sanches Fernandes; Eduardo Pagani; Cristiane Valverde Wenceslau; Leandro Hideki Ynoue; Luciana Ferrara; Irina Kerkis

DOI: 10.1186/s13287-025-04703-wStatus: Verified Translated Edition
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Key Findings in This Report

• First-in-human trial demonstrates that intravenous hDPSCs (NestaCell®) are safe and well-tolerated in Huntington’s disease patients over five years. • No serious adverse events were directly attributed to the stem cell product; the only serious event (lung cancer) was unrelated to the cells, with no evidence of engraftment. • Treatment-related adverse events were limited to transient hair pigmentation changes, indicating a favorable safety profile. • The study supports further clinical development of hDPSC therapy for neurodegenerative disorders, with preliminary evidence of potential efficacy.