Year: 2024
Type: conference-paper
Abstract: Background Huntington’s disease (HD) leads to progressive neural dysfunction and degeneration. Striatal degeneration is an early and prominent consequence of this process. Most experimental cell therapy research has focused on replacing striatal cells lost in HD, with the goal of repairing neural circuits. Studies in animal models using various cell types have shown that transplanted cells can survive in the brain for extended periods and improve some function. Transplantation of human fetal striatal cells also demonstrated long-term graft survival and that these cells may improve function, although the conditions for consistent success are not yet defined. Aims To develop cell-based Advance Therapy Medicinal Product (ATMPs) for cell replacement strategies in HD. Methods The Stem Cells for HD (SC4HD) and the EHDN Advanced Therapies Working Group (ATWG) aim to propose guidance for the safe development of stem cell transplantation studies for regenerative medicine in humans. The SC4HD ‘Cell production’-Task Force (TF) focuses on addressing the challenges and potential benefits of cell-based ATMPs for HD, including neural progenitor, telencephalic neuroblasts, or astrocytes transplants. Results/Conclusions Cell production-TF is currently working on several solutions for the identified challenges which include: 1) defining biomarkers based on cell surface markers or gene expression signatures to characterize the cell-based ATMP; 2) scaling-up the manufacturing process with Good Manufacturing Practices (GMP) compliance; 3) establishing the right quality controls to guaranty the standardization of the manufacturing process; and 4) creating strategies to assess their functionality and therapeutic potential on in vitro tests and animal models of HD.
Authors: Josep M Canals, Anselme Perrier, Unai Perpiña, on behalf of the EHDN Advanced Therapies Working Group and Stem Cells for Huntington’s Disease (SC4HD)
Institutions: Universitat de Barcelona, Université Paris-Saclay
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
OpenAlex ID: W4402373767