WARF: P09011US02

Treating Alexander Disease by Reducing Expression of Glial Fibrilary Acidic Protein (GFAP)


Albee Messing, Woosung Cho, Jon Thorson, Randal Goff

The Wisconsin Alumni Research Foundation (WARF) is seeking commercial partners interested in developing methods for treating Alexander disease.
OVERVIEWAlexander disease is an uncommon but fatal central nervous system disorder. This disease usually affects children and results in seizures, spasticity, hydrocephalus and psychomotor developmental delay. No cure exists and no standard treatment has been developed.

Most patients with Alexander disease have mutations in the gene for glial fibrilary acidic protein, or GFAP, which is the major structural protein in astrocytes. The GFAP mutations occur within an amino acid sequence that is highly conserved among intermediate filament proteins. Mutations at the homologous sites of other intermediate filament proteins also are associated with human diseases; however, in contrast to those mutations, mutations within the GFAP sequence appear to cause disease by producing a new and deleterious activity rather than by reducing or eliminating normal GFAP function.
THE INVENTIONUW-Madison researchers have developed methods of treating Alexander disease by down-regulating the expression of GFAP. They identified several compounds, including known antidepressants, antipsychotics, serotonin inhibitors and antihistamines, which are capable of reducing GFAP expression.

When administered to a patient with Alexander disease, the compounds should lessen the symptoms of the disease. They may reduce the intensity of the megalencephaly, or abnormally large brain, associated with Alexander disease; the amount of Rosenthal fibers, which are widely deposited in astrocytes in patients with the disease; and/or the intensity or frequency of seizures.
  • Treatment of Alexander disease
  • Provides a method of treating Alexander disease
  • Provides new uses for known, FDA-approved drugs
For More Information About the Inventors
Contact Information
For current licensing status, please contact Rafael Diaz at or 608-960-9847.
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