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Pharmacological approach to myelin repair

Value Proposition

The white matter of the central nervous system (CNS) is made up of myelin and myelin-producing glial cells that are essential for efficient neurotransmission. White matter damage is associated with several prevalent diseases and disorders characterized by neurobehavioral and cognitive impairments, including multiple sclerosis, cerebral palsy, traumatic brain injury, schizophrenia, stroke, various age-related neurodegenerative diseases (e.g. Alzheimer’s and Parkinson’s disease), and many others. These conditions affect several tens to hundreds of thousands of individuals in the United States alone each year and are frequently associated with an extensive economic impact, not only in terms of direct costs (e.g. drugs, healthcare) but also indirect costs, like burden to family members and loss of income. While the etiology of these diseases/disorders differs significantly, there is great potential for the amelioration of these conditions through the reestablishment of white matter via the formation of new oligodendrocytes (myelin-producing glial cells). However, to date, there are no approved therapies for the regeneration of white matter.


Dr. Eric Benner MD, PhD, a neonatologist at Duke University, has recently discovered that oxysterols can promote the differentiation of neural stem cells into oligodendrocytes. Additionally, in a sepsis-induced brain injury model, treatment of affected mice with oxysterols significantly enhanced the number of oligodendrocytes within the corpus callosum (Fig. 1). These data have encouraging implications for diseases and disorders that are associated with white matter damage.

Dr. Benner’s discoveries also have ramifications in the domain of memory and cognition. One of Dr. Benner’s findings was that human breast milk contains oxysterols, and breast feeding has long been associated with cognitive development in infants. This finding further strengthens the link between white matter integrity and cognitive function. Thus, oxysterols may be useful as cognitive enhancers, otherwise known as nootropics – a component of the $12-$37 billion per year supplement industry.

Duke File (IDF) Number



  • Benner, Eric

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School of Medicine (SOM)

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