Stem Cell Alzheimer’s Cure? Dr. Larry Goldstein’s Promising ‘Disease-in-a-Dish’

Stem cell ‘disease-in-a-dish’ shows promise for Alzheimer’s cure: Alzheimer’s, a progressive brain disorder that often strikes late in life, is a terrible disease. Sufferers slowly but irreversibly lose their memory, language skills and perception of time and space. Eventually, they can’t even take care of themselves.

On both patients and those who love and care for them, the disease takes a devastating toll. People with Alzheimer’s often feel great frustration and even fear as they struggle with everyday tasks and slowly lose their independence. Witnessing how the disease takes their loved one from them bit by bit, friends and family feel deeply hurt. Those who provide daily care for Alzheimer’s sufferers tend to get burned out.

About 5.4 million Americans and 30 million people across the world suffer from Alzheimer’s, but the disease is nearly impossible to diagnose before symptoms develop — and a drug to cure it or change its course remains elusive.

In this neurodegenerative disease, the physical changes in the brain are difficult to detect before clinical symptoms appear.

Striving to understand the causes of Alzheimer’s, scientists have probed animals as varied as fruit flies and fish, blood samples of patients, and even brain biopsies of patients who died.

And, until researchers from the University of California, San Diego, came out with their innovative technique, it’s been impossible to probe the neurons of Alzheimer’s patients before they show symptoms.

“By the time you can see dementia in a person, their brain cells have been behaving in an abnormal way for years — perhaps decades or longer,” says Dr. Larry Goldstein, a UCSD neuroscientist who led the team that used stem cells to create an “Alzheimer’s-in-a-dish” lab model to simulate what goes wrong in diseased brain cells of people with the disease. The findings of Dr. Goldstein’s study were published online in January in the journal Nature.

Reprogrammed cells = ‘Alzheimer’s in a dish’
In their study, researchers took fibroblasts or skin cells from dead patients, four who had died from Alzheimer’s and two who did not have dementia when they were alive.

Then, employing newly developed stem-cell technology, Dr. Goldstein and his team created induced pluripotent stem (iPS) cells or iPSCs. iPS cells were made by treating the fibroblasts with reprogramming factors to revert them to an embryonic-like state. Like the stem cells in early embryos, iPS cells can form any tissue in the body — including neurons — but their use for experiments isn’t controversial.

Finally, the team turned the iPCs into neurons that closely replicated those found in living Alzheimer’s patients.

The resulting cells of diseased neurons are in effect “Alzheimer’s-in-a-dish models” that can be manipulated in the lab by researchers, enabling them to study the disease more effectively. Being able to conduct more thorough and extensive studies on Alzheimer’s may, in the long run, help early diagnosis and even lead to the discovery of new drugs to treat the disease.

Breakthrough methodology
“We developed a true human neuronal model that accurately replicates early stages of the disease in true human brain cells,” said Dr. Goldstein, who is also professor of cellular and molecular medicine at UCSD’s medical school.

But, he tells Nature, the real innovation lies in his team’s designing of a procedure to purify neurons from a solution with other cells, making it possible to grow these neurons in enough amounts for study.

Some doctors who were not involved in the study praised the new technique, according to ABC News, which interviewed them.

According to Dr. George Grossberg of Missouri’s Saint Louis University School of Medicine, the “unique methodology” could be used to identify “why cells die in Alzheimer’s disease, and what therapeutic interventions might be useful.”