Preclinical Evidence for Cellular Therapy as a Treatment for Neurological Disease

Abstract

There continues to be extraordinary anticipation that stem cells will advance the current therapeutic regimen for both acute and degenerative neurological disease. Given the limited ability of the nervous system for regeneration and repair, combined with the devastating consequences that result from minimal neural tissue damage or cell death, any emerging therapy showing potential for cellular rescue or cell replacement represents a significant opportunity. This chapter will detail the preclinical progress using adult stem cells as a therapy for neurological diseases, both acute and degenerative. The basic adult stem cell types, including bone marrow–derived cells, umbilical cord blood–derived cells, and neural stem cells, will be discussed. The preclinical research using these cells to treat acute neurological diseases, such as traumatic brain injury and cerebrovascular disease, along with degenerative neurological disease, including Parkinson’s, Alzheimer’s, Huntington’s, and multiple sclerosis, will be detailed. Potential mechanisms of action and important limitations of current work will be addressed. Finally, the details of initiating a cellular therapy clinical trial, along with a brief discussion of ongoing clinical trials using cell therapy, with a focus on neurological disease trials, will be provided. Overall, early work using adult stem cell therapy for acute and degenerative neurological disease has provided some promising results, leading the way for the translation to clinical trials. The future of applying stem cell therapy to current treatment regimens of neurological diseases requires continued discovery at the molecular, cellular, tissue, and organism levels, alongside well-designed clinical trials.KeywordsCellular therapyAdult stem cellsNeurological diseaseTranslational research