Drug intelligence / Profile preview

valproic acid + expanded cord blood stem cells

Development stage
Phase 1
Lead developer
University of Illinois Chicago
Modality
Small Molecules, Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

Valproic acid + expanded cord blood stem cells refers to an experimental cell therapy product where umbilical cord blood (UCB) is cultured ex vivo with the small molecule valproic acid (VPA) to increase the population of hematopoietic stem and progenitor cells (HSPCs). Valproic acid acts as a histone deacetylase (HDAC) inhibitor, which modifies the epigenetic state of the cells to promote self-renewal and inhibit differentiation, effectively expanding the number of CD34+ cells available for transplantation. This approach aims to overcome the significant limitation of cord blood transplantation—the low absolute number of stem cells in a single unit—thereby accelerating hematopoietic recovery and improving clinical outcomes for patients with high-risk hematologic malignancies who lack a matched bone marrow donor.

Other names
VPA-expanded cord bloodvalproic acid-expanded hematopoietic stem cellsVPA-expanded CD34+ cellsVPA-UCB
02

Targets

HDAC1 (Histone Deacetylase 1)HDAC8 (Histone Deacetylase 8)GSK3 (Glycogen synthase kinase 3 beta)

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