Stem Cells for Huntington's Disease (SC4HD): An International Consortium to Facilitate Stem Cell-Based Therapy for Huntington's Disease

Romina Aron Badin, Anne Catherine Bachoud-Lèvi, Gerhard Bauer, Monica Busse-Morris, Josep M. Canals, Philipp Capetian, Elena Cattaneo, Jefferson Chen, Emanuele Cozzi, Zdenka Ellederova, Steven A. Goldman, William Gray, Liangxue Lai, Meng Li, Anna Morenkova, Guangjin Pan, Zhong Pei, Unai Perpina Martin, Anselme Perrier, John C. ReidlingAnne E. Rosser, Jiwhan Song, Leslie M. Thompson, Vicki Wheelock

Research output: Contribution to journalArticlepeer-review

Abstract

Huntington's disease (HD) research is entering an exciting phase, with new approaches such as huntingtin lowering strategies and cell therapies on the horizon. Technological advances to direct the differentiation of stem cells to desired neural types have opened new strategies for restoring damaged neuronal circuits in HD. However, challenges remain in the implementation of cell therapy approaches for patients suffering from HD. Cell therapies, together with other invasive approaches including allele specific oligonucleotides (ASOs) and viral delivery of huntingtin-lowering agents, require direct delivery of the therapeutic agents locally into the brain or cerebrospinal fluid. Delivering substances directly into the brain is complex and presents multiple challenges, including those related to regulatory requirements, safety and efficacy, surgical instrumentation, trial design, patient profiles, and selection of suitable and sensitive primary and secondary outcomes. In addition, production of clinical grade cell-based medicinal products also requires adherence to regulatory standards with extensive quality control of the protocols and cell products across different laboratories and production centers. Currently, there is no consensus on how best to address these challenges. Here we describe the formation of Stem Cells For Huntington's Disease (SC4HD: https://www.sc4hd.org/), a network of researchers and clinicians working to develop guidance and greater standardization for the HD field for stem cell based transplantation therapy for HD with a mission to work to develop criteria and guidance for development of a neural intra-cerebral stem cell-based therapy for HD.

Original languageEnglish (US)
Pages (from-to)221-226
Number of pages6
JournalJournal of Huntington's Disease
Volume10
Issue number2
DOIs
StatePublished - 2021

Keywords

  • Huntington's disease
  • Neuroscience
  • stem cells
  • transplantation

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience

Fingerprint

Dive into the research topics of 'Stem Cells for Huntington's Disease (SC4HD): An International Consortium to Facilitate Stem Cell-Based Therapy for Huntington's Disease'. Together they form a unique fingerprint.

Cite this