Related Experiment Video
Updated: Apr 15, 2026
Novel Passive Clearing Methods for the Rapid Production of Optical Transparency in Whole CNS Tissue
Published on: May 8, 2018
From mice to mind: Strategies and progress in translating neuroregeneration
Terry C Burns1, Catherine M Verfaillie2
1Department of Neurosurgery and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, USA.
Abstract:
Decisions about what experimental therapies are advanced to clinical trials are based almost exclusively on findings in preclinical animal studies. Over the past 30 years, animal models have forecast the success of hundreds of neuroprotective pharmacological therapies for stroke, Alzheimer׳s disease, spinal cord injury, traumatic brain injury and amyotrophic lateral sclerosis. Yet almost without exception, all have failed. Rapid advances in stem cell technologies have raised new hopes that these neurological diseases may one day be treatable. Still, how can neuroregenerative therapies be translated into clinical realities if available animal models are such poor surrogates of human disease? To address this question we discuss human and rodent neurogenesis, evaluate mechanisms of action for cellular therapies and describe progress in translating neuroregeneration to date. We conclude that not only are appropriate animal models critical to the development of safe and effective therapies, but that the multiple mechanisms of stem cell-mediated therapies may be particularly well suited to the mechanistically diverse nature of central nervous system diseases in mice and man.
Insights
Preclinical animal models have consistently failed to predict effective neuroprotective therapies for neurological diseases. Stem cell therapies offer promise, but require better animal models for successful translation to human clinical trials.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Translational Research
Background:
- Preclinical animal studies are the primary basis for advancing experimental therapies to human clinical trials.
- Despite decades of research, neuroprotective therapies for stroke, Alzheimer's disease, and other neurological conditions have largely failed in clinical translation.
- Advances in stem cell technology present new opportunities for treating neurological diseases.
Purpose of the Study:
- To evaluate the limitations of current animal models in predicting human neurological disease outcomes.
- To explore the potential of stem cell-based therapies for neurological conditions.
- To discuss the translation of neuroregeneration strategies from preclinical research to clinical application.
Main Methods:
- Comparative analysis of human and rodent neurogenesis.
- Evaluation of the mechanisms of action for cellular therapies.
- Review of current progress in translating neuroregeneration research.
Main Results:
- Animal models have historically been poor predictors of therapeutic success for neurological diseases.
- Stem cell therapies possess multiple mechanisms of action that may be advantageous for complex central nervous system diseases.
- The mechanistic diversity of stem cell therapies aligns well with the heterogeneity of neurological disorders.
Conclusions:
- Appropriate animal models are essential for developing safe and effective neuroregenerative therapies.
- Stem cell-mediated therapies hold significant potential for treating a range of neurological diseases due to their versatile mechanisms.
- Addressing the limitations of animal models is crucial for successful clinical translation of neuroregenerative treatments.
