Therapy for Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a rapidly progressive, uniformly fatal neurodegenerative disease. It is characterized by the loss of motor neurons in the spinal cord, brainstem and cerebral cortex, leading to a decline in muscular function. It eventually results in weakness, speech deficits and difficulty swallowing. ALS is almost always fatal within two to three years.

We are developing h-MSCs as a delivery platform for therapeutic factors in ALS where they may have an intrinsic therapeutic effect.

Mensenchymal stem cells (MSCs) are pluripotent, self-renewing cells with the potential for tissue regeneration. These cells also have the potential for gene delivery and to transdifferentiate into cells of mesodermal origin. MSCs can be used as vectors of cytokines and trophic factors to prevent cell death, tissue inflammation and damage.

Project Personnel

  • Greg W. Butler
  • Allan B. Dietz, Ph.D.
  • Douglas J. Padley
  • Eric J. Sorenson, M.D.
  • Nathan P. Staff, M.D., Ph.D.
  • Anthony J. Windebank, M.D.