Stem Cell Therapy for Acute Cerebral Injury

What Do We Know And What Will the Future Bring?

Robin Lemmens; Gary K. Steinberg


Curr Opin Neurol. 2013;26(6):617-625. 

In This Article

Abstract and Introduction


Purpose of review The central nervous system has limited capacity for regeneration after acute and chronic injury. An attractive approach to stimulate neural plasticity in the brain is to transplant stem cells in order to restore function. Here, we discuss potential mechanisms of action, current knowledge and future perspectives of clinical stem cell research for stroke and traumatic brain injury.

Recent findings Preclinical data using various models suggest stem cell therapy to be a promising therapeutic avenue. Progress has been made in elucidating the mechanism of action of various cell types used, shifting the hypothesis from neural replacement to enhancing endogenous repair processes. Translation of these findings in clinical trials is currently being pursued with emphasis on both safety as well as efficacy.

Summary Clinical trials are currently recruiting patients in phase I and II trials to gain more insight in the therapeutic potential of stem cells in acute cerebral injury. A close interplay between results of these clinical trials and more extensive basic research is essential for future trial design, choosing the optimal transplantation strategy and selecting the right patients.


Stroke causes one in 18 deaths in the United States, and stroke incidence is expected to increase over the next decades.[1] The only effective treatment, intravenous tissue plasminogen activator, has a narrow time window, thereby limiting its application to a small subset of patients with ischemic stroke.[2,3] Unfortunately, many patients are disabled after stroke and require ongoing medical care, so that most yearly costs occur in this chronic setting.[4] These findings have led to research focused on the mechanisms of neural repair as these relate to patterns of human recovery after stroke.

Traumatic brain injury (TBI) is a major cause of mortality and morbidity, especially among young adults, and lifelong disability is common in those who survive.[5] With no effective therapy available, interest in the potential of stem cell therapy for TBI patients is emerging.

Stem-cell-based approaches, therefore, could provide new therapeutic avenues to restore neurological function in both patients with stroke[6] and those with TBI.[7]