J03 Clinical Translation of Stem Cell Therapies for Huntington’s Disease (HD)

Background The underpinning concept of regenerative medicine is restoration of structure and function. This can be achieved through several approaches, including implantation of cells to either provide support for vulnerable host cells or to integrate and adopt the function of cells lost to the disease process. HD represents an excellent prospect for regenerative medicine, but there are multiple challenges along the translational pipeline, many of which are common across diseases and pertinent to multiple donor cell types. Aims Provide a consensus document that sets out the challenges for advancing stem cell therapies to the clinic for HD. Methods Stem cells for Huntington’s disease (SC4HD) and the EHDN Advanced Therapies Working Group (ATWG) provide a combined platform for discussion and to share experience in order to generate a robust clinical development plan across a range of stem cell-based therapies for HD. Results Over a series of meetings, SC4HD/ATWG have produced a consensus white paper (submitted for publication) that identifies the challenges of cell therapy for HD. These include defining criteria for transitioning to clinical studies; scale-up, characterization, quality control and validation of the cell product; design, validation and approval of surgical devices; operative procedures for safe and effective delivery of cell product to the brain; designing clinical trials that incorporate principles of efficient design and disease specific outcomes. All processes must be adaptable in a dynamic regulatory environment. Conclusions Here we set out the challenges associated with the clinical translation of cell therapies, using HD as a specific example, and suggests potential strategies to address these challenges. This provides the starting point for developing guidance, and highlighting future directions, with the aim to expedite progress towards therapies for clinical benefit in HD.