Is Gene Therapy Our Next Best Hope for an Alzheimer’s Cure?


The cure for Alzheimer’s is one of the holy grails for the medical research community as well as for the estimated 5.4 million Americans who suffer from the disease and their family members. Drug companies have poured billions of dollars into the search for a cure, but so far they’ve only been able to produce drugs that can slow the progression of the disease. There have been big hopes with new drugs in the past followed by big failures during clinical trials. It’s enough to make anyone wary when they hear about an exciting new study that offers tantalizingly positive results. However, put your cynicism aside, because gene therapy might just lead us to a future free of Alzheimer’s.

A Gene Called PGC-1ɑ

In early October, 2016, the Proceedings of the National Academy of Sciences published fascinating research on gene therapy to conquer Alzheimer’s disease. A research team at the Imperial College of London identified a gene called PGC-1ɑ, which stops the production of beta amyloid peptides.

If you’ve done your Alzheimer’s research, then you know that beta amyloid peptides are the things that produce plaque deposits in the brains of people with Alzheimer’s. It’s these plaque deposits that cause so much trouble by triggering inflammation in the brain and destroying neurons that lead to significant damage in the hippocampus (the memory center of the brain) and the cortex (which supports thinking and reasoning). If PGC-1ɑ could really disrupt the production of beta amyloid peptides, that would mean no more brain plaque (or at least a lot less of the stuff) and possibly no Alzheimer’s!

The researchers set out to put PGC-1ɑ to the test.

Very Hopeful Results

Before human trials could start, the researchers needed to test the gene therapy on a smaller scale. Small as in whiskers, long tail, and big round ears. That’s right, a group of mice were volunteered for the task. Not just any group of mice. The researchers used a strain of mice that have been engineered to produce higher levels of amyloid beta peptides (the stuff that produces plaque in the brain). One group of these mice, let’s call them Group A, were given the PGC-1ɑ to see if it would prevent the formation of plaque. They were compared to two test groups. Group B mice also have higher levels of amyloid beta peptides but were not given the gene therapy. Two control groups of normal mice was also used (one received the gene therapy and one did not) as a comparison.

When the researchers put the mice through observational memory tests, they found that the mice in Group A (those that received the gene therapy) performed just as well as the control group of normal mice. The poor mice in Group B showed definite signs of memory decay on the tests.

The researchers then looked directly at the brains of each group of mice. They found that the brains of the Group A showed a 19.1% reduction in beta amyloid peptides in the cortex and a 30% reduction in the hippocampus compared to their unfortunate cousins in Group B. Most impressively of all, Group A mice showed 43% less plaque buildup in the cortex and 51% less buildup in the hippocampus.

This is big news, but does it mean that Alzheimer’s is history?

Not exactly. Mice are not humans. This study basically provided a “proof of concept,” or evidence that PGC-1ɑ is worth studying further. The researchers will need to move forward to see if this same result can be replicated in humans. Even if that occurs, we will still need to determine how to turn the results into a safe, wide-scale therapy.

Despite all of these logistical hurdles, this type of research should provide hope that researchers are making some very promising discoveries in the fight against Alzheimer’s. While these therapies are still on the horizon and won’t be able to provide much help to those already in the throes of the disease, it does offer hope to future generations. There may come a day when Alzheimer’s is a distant memory!

Of course, if you have a loved one suffering from Alzheimer’s now, then you may need guidance, support, or simply someone to listen right now. If you live in Poway or San Diego County, we invite you to attend our monthly support group for caregivers and families affected by Alzheimer’s and Dementia.

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