December 10, 2013

NT219: New Promise for Treating Neurodegenerative Diseases?

Can a newly created compound that inhibits an element of aging really treat brain diseases like Alzheimer’s, Parkinson’s, and Huntington’s?

Scientists at the Hebrew University of Jerusalem, working with a new company that – no surprise – has already filed for a patent on the potentially profitable product, are betting, “Yes it can.”

Their findings were published last week in the journal Aging Cell ("A novel inhibitor of the insulin/IGF signaling pathway protects from age-onset, neurodegeneration-linked proteotoxicity"). The compound in question has a name: NT219. Its creators claim it impedes a particular aging process – without extending lifespan -- to protect the brain from neurodegenerative diseases.

Protein Build-ups in People Only as They Age
These brain diseases share several features: they seem to result from protein accumulations, and happen to people later in life. They don’t occur in much younger people, the way cancer does. So what is it exactly about the process of aging that makes people vulnerable?

Dr. Ehud Cohen at Hebrew University discovered he could create a defense against toxic protein build-ups – in worms – by reducing the activity of a signaling mechanism conveyed through insulin and the growth hormone IGF1, a key channel that regulates aging. Cohen later found he could also protect Alzheimer’s-afflicted mice against behavioral impairment by restraining that same signaling process.

Enter TyrNovo, the start-up company apparently created to develop a compound to inhibit the growth hormone IGF1, whose natural activity seems to set in motion the processes that sometimes result in debilitating neurodegenerative diseases.

TN219 Best Disrupted the Aging Channel
Researchers created several compounds, and identified TN219 as the most promising. Since then, they report having demonstrated that TN219 has inhibited the IGF1 signaling mechanism in worms and human cells.

The next step? Cohen has secured “ethical approval” to test the effectiveness of TN219 on AD-afflicted mice.

It’s all very interesting. And there are some exciting new elements in these reports. But there's an issue that strikes me as puzzling. We hear repeatedly that -- at least in the case of Parkinson's -- by the time most people receive their diagnoses, most of the damage to dopamine-producing cells has already occured, and the patient begins the long and difficult journey -- through drugs and exercise, mostly -- of damage control. It seems TN219 -- even if all the bugs are worked out and approvals come -- would have to be administered well before people even know they're candidates for PD. As always, the issue of early detection, BEFORE people develop symptoms, is paramount.  

And if we’ve learned anything over the past few years, as we've followed other “breakthrough” developments, it’s this: there’s a vast distance between mice and men.

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