eNeuro: Protein-based therapeutic therapies are helpful for patients with amyotrophic lateral sclerosis

Researchers at the USF Health Morsanne School of Medicine at the University of South Florida have successfully tested a protein that has the potential to help develop a protein-based therapy for patients with ALS, a progressive neurological disorder, also known as Lou Gale's disease, that affects nerve cells
in the brain and spinal cord.

The study, published in eNeuro, examined the effects of apolipoprotein A1, a "good cholesterol," on endothelial cells, which are the lining of blood vessels that provide a barrier
between the brain, spinal cord tissue, and blood circulation.

In a dish reminiscent of amyotrophic lateral sclerosis (ALS), the team found that the protein activates a unique pathway inside cells that can improve survival and protect endothelial cells from toxic substances in the blood
.
This pathway enhances cell survival and prevents further damage
to blood vessels by ALS.

Svitlana Garbuzova-Davis, professor and principal investigator of the Department of Neurosurgery and Brain Repair, said: "With the functional barrier, we hope that the environmental toxicity of the central nervous system will be reduced and the progression of the disease can be slowed down
.
"

While this protein has been shown to protect endothelial cells from diseases such as diabetes and atherosclerosis, the role of als-damaged endothelial cells was previously unclear
.

To test the effect of this protein on amyotrophic lateral sclerosis, the team studied how this protein affects endothelial cell signaling
.
Garbuzova-Davis and co-researchers USF Professor Alison Willing and USF Distinguished University Professor Cesario Borlongan found that injured cells absorb proteins, significantly reducing endothelial cell damage
.

"It's too early to predict the eventual impact on patients," Willing said
.
"In this study, we used a cell culture model in which we can control every
aspect of what environment the cells are exposed to.
Humans cannot have the same level of control
.
”

Garbuzova-Davis said the study lays the groundwork for further study of animal models of amyotrophic lateral sclerosis to fully determine the therapeutic effects
of this protein.
If the results are successful, apolipoprotein A1 may be considered for clinical trials to assess its safety and efficacy in patients with ALS
.

In the near future, apolipoprotein A1 may be considered a potential new therapy for endothelial cell repair, restoring the central nervous system barrier
in patients with ALS.
It may also help relieve symptoms of amyotrophic lateral sclerosis, such as imbalanced antioxidant levels and inflammation
.