UW researchers working to combat fatal Alexander disease

UW-Madison campus

MADISON, Wis. – Alexander disease has no cure, no standard course of treatment, and is typically fatal. UW researchers are working to change that.

Waisman Center senior scientist Tracy Hagemann is leading a study alongside Albee Messing of the Alexander Disease Lab.

Messing and his colleague Michael Brenner discovered the gene responsible for Alexander disease. He has been conducting research on the disease for over 20 years.

This new study, which is exploring a potential treatment for Alexander disease, is moving from a rat model to human clinical trials.

People born with the disease can develop an enlarged brain and head, have intellectual disabilities, or experience seizures.

The disease destroys the white matter of the brain.

Researchers used antisense oligonucleotides in rats who suffered from the disease as part of a treatment to combat these symptoms.

Antisense oligonucleotides are small pieces of DNA. In the rat model, these pieces targeted mRNA cells to stop the creation of harmful types of proteins.

Alexander disease can cause a gene mutation that creates a harmful protein called GFAP.

The study showed that when rats were treated with the antisense oligonucleotides before they developed any major symptoms from Alexander disease they remained in a nearly indistinguishable state from their healthy littermates.

When treated after symptoms developed, not only did symptoms improve, some of the damage to the brain’s white matter was reversed.

The team hopes to replicate similar success in humans.

“We’ll be happy if we can stop the disease from progressing,” Hagemann said in a statement Wednesday. “But if you can actually see some reversal of symptoms that have already occurred, that would be wonderful.”

The study is co-authored by researchers across the globe and is supported by the National Institutes of Health.