Neuroscience

Business World: Protein Misfolding and FoldRx, Satori, & Zyentia

From The Boston Globe:

Crumpled proteins a new frontier
By Scott Kirsner
The Boston Globe
May 30, 2005

Inside your body, at this very moment, a class in intracellular origami is taking place.

Long strands of protein are being twisted into intricate shapes within your cells. To work, they've got to be in exactly the right formation -- imagine a piece of paper folded in the shape of a perfect giraffe. When something goes awry, and the protein winds up in a crumpled ball, it doesn't act the way it's supposed to. It tends to cluster with other malformed proteins over in the corner of the classroom.

In large numbers, these crumpled proteins can lead to more than two dozen nasty diseases, from Alzheimer's to Parkinson's to diabetes. Scientists call it ''protein misfolding."

So little is known about what proteins do in the human body, what causes them to fold correctly, and what causes them to misfold, that trying to develop drugs to deal with protein misfolding is a bit like trying to teach origami to a goat. You might not want to brand it impossible; you'd just be surprised if anything came of it.

But in the biotechnology field, venture capital money sometimes pours into start-ups trying to commercialize promising research in fields like protein misfolding and RNA interference, both of which attempt to rip up the root causes of disease, rather than just hack away at symptoms. This funding process, which has a lot in common with betting the long shot at the track, discovers lots of scientific dead ends, but it occasionally results in important new drugs. In the case of protein misfolding, many of these will be what the industry calls ''first-in-class drugs," as opposed to ''me too" drugs, like the fourth pill on the market to treat erectile dysfunction or reduce cholesterol.

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Anthony H. Risser | neuroscience | neuropsychology | brain

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