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Scientists Test A Potential Treatment Of Alzheimer's In Vitro

This article is more than 10 years old.

A Team of Researchers in Japan have been reprogramming cells from patients with Alzheimer's into neurons to model the progress of the disease and develop potential therapies that can aid at least a subset of patients suffering from it.

As I've written before, many researchers expect that the biggest breakthroughs with stem cells are in the short term more likely to be in the realm of drug discovery rather than tissue or organ regeneration. (Not to take away from the crucial progress being made there.)

A major cause of Alzheimer's is a protein called amyloid-β (beta) peptide, which builds up in the brain as a form of plaque.

In certain Alzheimer's patients with a familial mutation in amyloid proteins, the build up of Aβ molecules, or oligomers, in neurons and astrocytes causes stress on a particular organelle of the cell called the endoplasmic reticulum.

Among other functions, this organelle is responsible for the transporting of proteins within the cell.

The Japanese team, which included 2012 Nobel laureate Shinya Yamanaka, differentiated cells from these Alzheimer's  patients into neurons and then treated them with docosahexaenoic acid (DHA), a solution that alleviated the stress response in the neurons.

According to the paper in the April 4th issue of Cell Stem Cell:

Using patient neurons and astrocytes, we addressed the accumulation and possible pathological roles of intracellular Aβ oligomers in familial and sporadic AD. We found that Aβ oligomers were not proteolytically resistant and that docosahexaenoic acid (DHA) treatment attenuated cellular phenotypes of AD neural cells with intracellular Aβ oligomers in both familial and sporadic AD patients.

Granted that this is just one study looking at a small set of people that suffer from a disease that effects millions, I followed up with one of the paper's authors, Haruhisa Inoue of the Center for iPS Cell Research and Application at Kyoto University, to see if their research might lead to a concrete treatment.

"So far several companies have showed an interest in our work," he told me in an email. "But not specific to DHA."

Inoue pointed out that DHA clinical trials have been reported in the past few years, and the treatment was effective only for a group of patients with mild to moderate symptoms, and largely but not completely ineffective in another study.

"Our data might suggest that, if we identify individual patient perspective by iPSC-based analysis," he said, "and classify the phenotypes of patients... DHA treatment might be effective to a part of the moderate-symptoms group in addition to the mild-symptoms group."

This is provisional, to be sure. But as the iPSC process itself improves and the neurons of thousands of Alzheimer's patients can be studied more speedily, we may soon see many niche therapies that can be customized for different groups of Alzheimer's sufferers.

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