A new international consortium to promote Stem Cell-Based Therapy for Huntington's disease

The Stem Cells for Huntington's Disease (SC4HD) is a new international consortium created to promote advanced therapy medicinal product (ATMP) through cell transplantation studies on Huntington’s Disease

The entity, made up of twenty-eight renowned researchers from ten countries, has been officially presented in a recent publication in the Journal of Huntington’s Disease.

Josep M. Canals and Unai Perpiñá, researchers of the IDIBAPS research group Pathophysiology and treatment of neurodegenerative disorders, are part of the SC4HD.

The main goals of SC4HD researchers are to work together with the HD community, develop criteria and guidance and address the main challenges to rapidly and safely bring potentially beneficial stem cell-based therapies to patients with this devastating disease. 

According to Josep M. Canals, who is also director of the UB Creatio center and member of the steering committee of SC4HD, “there is an exciting opportunity to apply stem cell-based therapies in Huntington’s disease (HD), a devastating neurodegenerative disease that mainly affects neurons in the brain nucleus known as striatum”. 

To date, most experimental cell therapies  have focused on the striatum aiming to replace the lost striatal neurons during the disease process. Therefore, this aims to repair the neuronal circuit and increase the survival of the remaining neuronal and glial striatal cells. Technological advances aimed to direct the differentiation of stem cells to desired neural types have opened new strategies for restoring damaged neuronal circuits in Huntington's.

The SC4H consortium brings together experts in stem cell biology, cell transplantation, clinical grade cell production, neurosurgery, and clinical evaluation. Its aim is to accelerate the safe clinical translation of stem cell therapy and to provide a comprehensive standardization and guidance for the field, spanning the full range of therapeutic considerations.

Article reference:

Stem cells for Huntington’s disease (SC4HD): an international consortium to facilitate stem cell-based therapy for Huntington’s disease Canals, J. M.; Perpiñá, U. et al. ". Journal of Huntington’s Disease, February, 2021. DOI: 10.3233/JHD-210473