Planning The Way Forward In Neurodegenerative Disease Studies

By Ewan Campbell, Advanced Therapies Director, Veristat LLC

Clinical trials involving gene therapies for the treatment of Central Nervous System (CNS) indications are some of the more complex in the field of gene and cell therapy (G&CT) development.  During the lockdown, the Food and Drug Administration (FDA) released specific draft guidance titled, Human Gene Therapy for Neurodegenerative Diseases (ND).   This guidance provides recommendations for Sponsors who are developing human gene therapy (GT) products for neurodegenerative diseases, which affect adult and pediatric patients. 

Neurodegenerative diseases like Alzheimer's, Huntington's, and Parkinson's represent a heterogeneous group of disorders, characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system.  These diseases vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases.

The FDA guidance focuses on considerations for product development, preclinical testing, and clinical trial design. The guidance also discusses marketing approval pathways for investigational gene therapy products. Communicating early with regulatory agencies like the FDA and EMA is particularly advantageous in ND.  Several areas to consider when preparing for agency interactions are outlined below.