Neurorestoration therapeutics for neurodegenerative and psychiatric disease

Abstract

Objective: Neurorestoration within the human central nervous system (CNS) is a concept that is barely a decade old. Despite this, there is significant clinical activity in this area, although there has not been any attempt to systematically identify these trials and organize them by disease area and phase of development. The objective of this investigation is to broadly review the current state of neurorestorative clinical trial activity ongoing worldwide.

Methods: Iterative searches of the databases clinicaltrials.gov, EU Clinical Trials Register and ADIS Insight were used to locate clinical trials identified as involving neurorecovery/restoration strategies or stem cells for central nervous system diseases.

Results: A wide range of neurorestorative clinical trials (N = 106) are ongoing or planned. Nearly three-fourths of all clinical trials (75/106) are targeting one of four disease areas: multiple sclerosis, stroke, Parkinson’s disease, and amyotrophic lateral sclerosis. Nearly two-thirds (63%) of 106 ongoing and planned clinical trials focus on cell-based therapy, although these are almost exclusively in phase 1 or 2 of development.

Discussion: Neurorecovery is an emerging field that is currently focused on earlier stages of clinical development, primarily in four disease areas. As the field matures, it is expected that there will be a greater balance of studies across a wider spectrum of CNS diseases as well as in late-stage development.

Keywords:

• Gene therapy
• Neurorestoration
• Stem cells
• Trophic factors