Perspectives on Development and Regulation of Therapeutic Products for CED-Based Therapy of Neurodegenerative Diseases.

Neurodegenerative diseases (NDs) caused by the progressive degeneration of brain neurons affect millions of people worldwide and pose significant public health challenges. Despite therapeutic advances in the treatment of these diseases, drug delivery to desired brain targets is difficult due to the intrinsic resistance of the blood-brain barrier (BBB). Many research groups are investigating novel strategies of transporting drug molecules across this barrier. Convection-enhanced delivery (CED) is a neurosurgical administration technique that has been recognized as an effective means of bypassing the BBB to deliver drugs to their clinical targets in pharmacologically relevant quantities. Currently, the drugs and devices being used in CED therapy remain at an investigational stage and have not been approved for use in actual treatment. This article provides a brief introduction of the etiologies of NDs, technical insights into CED and perspectives on the development and regulation of therapeutic products for the CED-based therapy of NDs.