Scientists at Northwestern University used a novel compound to arrest early stages of Alzheimer’s disease in mice — setting the stage, if all goes well, for a groundbreaking treatment in which the debilitating condition can be controlled like high cholesterol.
The researchers discovered an unknown subtype of a protein in the brain which can lead to Alzheimer’s disease, according to a new paper the team published in the journal Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association, and they demonstrated that their new novel compound, dubbed NU-9, can attack this brain protein and stop the disease from taking hold — in mice, at least.
“Most people are used to monitoring their cholesterol levels,” said Richard Silverman, a chemistry professor at Northwestern who coauthored the paper and invented NU-9, in a university statement. “If you have high cholesterol, it doesn’t mean that you will have a heart attack soon. But it’s time to take drugs to lower your cholesterol levels to prevent that heart attack from happening down the road. NU-9 could play a similar role. If someone has a biomarker signaling Alzheimer’s disease, then they could start taking NU-9 before symptoms appear.”
For this particular experiment, the team took lab mice that were predisposed to develop Alzheimer’s disease but hadn’t developed the condition yet, and started giving them an oral dose of NU-9 for 60 days. They were especially curious about how NU-9 would impact the protein amyloid beta oligomers, which accumulate in the brain hand in hand with signs of Alzheimer’s.
After closely examining the mice brains, scientists observed that NU-9 essentially decreased the presence of a previously unknown subtype of amyloid beta oligomers that they dubbed ACU193+ AβOs. This specific type of protein was associated with brain inflammation that shows up before people are diagnosed with Alzheimer’s. When the team introduced NU-9 into these mice brains, they found that NU-9 tamped down the presence of this toxic protein and hence decreased inflammation.
“These results are stunning,” William Klein, a Northwestern neuroscience professor and the paper’s principal investigator, summed up in the university statement.
“There are a couple early diagnostic blood tests for Alzheimer’s disease in development,” he said. “The promise of better early diagnostics — combined with a drug that could stop the disease in its tracks — is the goal.”
The next step now is to experiment and analyze the effectiveness of NU-9 in the later stages of Alzheimer’s disease, according to the scientists.
Silverman originally invented NU-9, a synthetic compound with the technical name of cyclohexane-1,3-dione, in an effort to identify chemicals that can tackle neurological diseases. Besides Alzheimer’s disease, the NU-9 has proved effective against Amyotrophic lateral sclerosis (ALS) and possibly frontotemporal degeneration, all three of which involve the appearance of toxic protein in brains.
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