Neurosteroid: A New Treatment for Alzheimer's

Sherry Christiansen
By Sherry ChristiansenMarch 19, 2018

When a person has Alzheimer’s disease, they lose a large number of brain cells – or neurons – in specific regions of the brain. The process starts in the area called the hippocampus, where memories are processed for long-term storage.

Scientists have been trying to figure out just how to restore these lost brain cells for some time, through a process called neurogenesis.


Neurogenesis is the process by which brain cells, known as neurons, are produced by neural stem cells. The assumption is that if the neurons are replaced, cognitive function will be restored. But in the past, neurogenesis involving stem cell therapy has failed.

Stem cells reside within the body waiting to be used to regenerate tissues and cells in a continuous process, which occurs throughout the lifespan. Without stem cells, people wouldn’t be able to live very long, because as old cells die, there would be no way of replacing them.

In addition to replacing old cells and tissue that have lived out their lifespan, the inflammatory process stimulates cells to flood into an area that has been damaged or injured, to repair the wreckage.

Neurosteroid: A New Treatment for Alzheimer’s

Most recently, through funding from the Alzheimer’s Drug Discovery Foundation (ADDF), Dr. Roberta Diaz Brinton, Professor of Pharmacology and Neurology at the University of Arizona, in Tucson, has discovered that a neurosteroid, called allopregnanolone, helps to multiply and stimulate stem cells.

Dr. Brinton specializes in bioenergetics and regenerative systems in the brain, a part of biochemistry concerned with the energy required to create (and break) chemical bonds in the molecules found in organisms. Regenerative systems involve the scientific discovery that many of the organs of the body (including the brain) harbor stem cells which can regenerate dead cells.  

Neurosteroids are molecules synthesized in the brain, that modulate brain excitability. In a recent phase 1 clinical research trial, Dr. Brinton demonstrated that:

The allopregnanolone promoted neurogenesis, restoring cognition while reducing symptoms of Alzheimer’s in the brain.

This is very inspiring news for the hope of a future cure for the disease. An all-new, larger, phase 2 clinical trial is now being planned to support the manufacture of allopregnanolone, funded by the ADDF.

“Dr. Brinton is among the most prominent researchers working on Alzheimer’s today,” said Howard Fillit, MD, Founding Executive Director and Chief Scientific Officer of the ADDF. “Her research output, including her development of what could become the first regenerative therapy for Alzheimer’s, is impressive. I have known Robbie for many years and am pleased that the Committee chose to honor her immense contribution to our field with the third annual Goodes Prize.”

The Goodes Prize Awarded to Neurosteroid Researchers

ADDF researchers make important contributions toward the availability of effective treatment for Alzheimer’s. The Goodes Prize was awarded to Dr. Brinton this year, for her groundbreaking work with neurosteroids, as an innovative new treatment approach for the disease. According to the panel of experts, “Dr. Brinton’s “integrative research strategies span discovery to translation to clinical science. She is one of the few investigators in Alzheimer’s disease applying systems approaches to drug development to enable precision medicine in the field.”

“The recognition of our innovative therapeutic approach by the ADDF’s Goodes Prize is deeply gratifying. I am honored to be among those who continue the legacy of Melvin and Nancy Goodes to cure Alzheimer’s disease and am confident that together we will achieve the goal” said Dr. Brinton.

Dr. Brinton’s goal is said to be an example of “precision therapeutics to delay, treat, and cure Alzheimer’s disease.” Dr. Brinton is also involved in a second clinical trial involving a ground-breaking treatment for age-related cognitive decline and menopausal symptoms in women at risk for Alzheimer’s.

Are you hopeful that neurosteroid research may result in a treatment for Alzheimer’s? We’d like to hear your thoughts in the comments below.

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Sherry Christiansen

Sherry Christiansen

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