Brian Kaspar, Ph.D.

Associate Professor, Dept. of Pediatrics; Nationwide Children's Research Institute


Current Research Description

The main focus of the Kaspar laboratory is centered on the mechanism(s) of neurodegeneration in Amyotrophic Lateral Sclerosis (ALS) and the design and utilization of novel therapies to combat this debilitating, lethal disease. The underlying cause for ALS remains highly elusive and there is no known cure. There have been few effective therapies that have emerged to impede the progression of the disease in the ALS mouse model, and ironically, only a few of these therapies have translated their beneficial effects to human beings. Specifically, the lab is skilled in utilizing adeno-associated viral (AAV) gene therapy to deliver genes to all areas of the central nervous system (CNS). Dr. Kaspar has demonstrated that AAV-mediated delivery of IGF-1 to the spinal cord can prolong survival of a mouse model of ALS that is equivalent to adding years to the lives of ALS patients. Currently, clinical trials are being developed to translate this discovery into a therapeutic for ALS patients. Other neurotrophins are being investigated for their neuroprotective characteristics as well as novel delivery strategies are being pursued to target the CNS more efficiently in the treatment of ALS.

In addition to delivering neurotrophic support such as IGF-1 to the spinal cord by AAV, the Kaspar laboratory is developing strategies to differentiate motor neurons from adult neural progenitor cells. Adult neural progenitors exhibit stem cell characteristics as demonstrated by their multipotentiality and self-renewal. Unlike embryonic stem cells, there is no controversy related to these cells since they are isolated from the adult nervous system. We are currently designing procedures to transplant these differentiated motor neurons into the spinal cords of ALS mice in efforts to establish a cell replacement therapeutic for ALS.

Areas of Expertise
  • Neurotrauma, Neurological Disorders, and Gene Therapy
  • PhD: University of California, San Diego
  • Postdoctoral Training: Salk Institute for Biological Studies

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614) 722-2700
614) 355-5247
700 Childrens Drive, Room WA3022
Columbus, OH 43205