{SUPER: Marc Diamond, M.D./Professor, Neurology & Neurotherapeutics/Director, Center for Alzheimer’s and Neurodegenerative Diseases}

Alzheimer’s is probably the single biggest looming threat to our healthcare system.

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There’s currently about 5 million people who have the disease. That number is expected to triple in the next 30 years or so…and if you triple that number, you’ve basically would exhaust the entire Medicare budget just that one disease if you don’t come up with something to slow this down.

In order to stop Alzheimer’s disease, we think we have to intervene as early as possible before any neurological problems have occurred and to stop the first molecular events that underlie this disease. We think we’ve identified these events in determining that the critical protein that causes neurodegeneration called tau undergoes a conformational change from an inert form to one that is pathological.

The protein has two general shapes that it can be in.

We were able to figure out that there is a core region in the protein that is collapsed on itself in the inert form. It is basically two hairpins that are kind of tight and they encapsulate some amino acids that enable the protein to self-assemble.

So in this inert form, these bad amino acids, if you will, are hidden, but in the pathological forms of tau that we could produce in a test tube or that we could pull from human brain, the tau molecule was opened up and these reactive amino acids were now exposed.

And what we found was that when it was in this bad form, if we left it alone, it would quickly build up on itself.

So what we’ve done then is to find two different structures of a protein which was previously thought to be without an intrinsic structure.

We have a lot of insight now into the structure that discriminates this good from bad form of tau and that should allow us to make new therapies that prevent the formation of the bad or clear it away or to detect the bad form of tau at the earliest stage of disease long before anyone experiences symptoms.

It might someday be possible to extract tiny amounts of this tau protein that’s floating around in our spinal fluid and by determining its sub-structures, actually predict classes of diseases that a person might be at risk for long before they actually get sick.

There’s a lot of other disease besides Alzheimer’s. There’s a lot of other proteins that cause neurodegenerative disease…if this works for tau then you can immediately start to think about how to scale these ideas across other proteins like the protein that cause Parkinson’s disease…proteins that are linked to Lou Gehrig’s disease. Things like that.

I’ve been working in this field for 15 years and this is perhaps the most exciting new discovery I have made in these efforts.