December 12, 2013

New drugs -- "pharmacoperones" -- for Alzheimer's and Parkinson's?

Can a new drug that targets “misfolded” proteins cure neurodegenerative diseases -- like Alzheimer’s, Parkinson’s, and Huntington’s – as well as diabetes, cystic fibrosis, and inherited cataracts?

Scientists have known about misfolded proteins for years. Until recently, they assumed these misfolded proteins just didn’t work, accumulated, and caused disease.

Healthy protein molecules assume very precisely folded 3-D shapes. Mutations mess up those folds, and problems develop. The cell’s “early warning system” then moves the misfolded proteins to a different location within the cell. The misfolded protein isn’t destroyed in the move, only disabled.

Dr. P. Michael Conn and his team at Oregon Health & Science University have apparently developed a new type of drug – a pharmacoperone – that essentially rescues misfolded proteins, returning them to their original location and thus enabling them to resume their proper health-sustaining work inside cells.

From Mice to Men
Conn has had success with mice, and is eager to learn if clinical trials can duplicate his achievements with humans. As we’ve seen time and again, it’s a daunting task making that leap. He said:
The opportunity here is going to be enormous, because so many human diseases are caused by misfolded proteins. The ability of these drugs . . . to rescue misfolded proteins and return them to normalcy could someday be an underlying cure to a number of diseases. Drugs that act by regulating the trafficking of molecules within cells are a whole new way of thinking about treating disease.
On Tuesday, I wrote about a newly discovered compound -- NT219 -- that may treat brain diseases – Alzheimer’s, Parkinson’s, Huntington’s – by disabling a particular neural channel associated with aging.

The Research Funding Challenge
Now, we hear about the promise of pharmacoperones to treat these same diseases. It’s hard NOT to feel we’re making some progress, slow though it may be. I wish we could direct more funding toward these important efforts, but don’t have faith in this Congress to do the right thing.

As I mentioned in yesterday’s post, I’m willing to pay higher taxes and receive fewer benefits to pay for this research. But I don’t think higher taxes are coming, and the chorus to maintain (or increase!) benefits seems very loud indeed.

The report on Conn’s work should appear this week in the online edition of the "Proceedings of the National Academy of Sciences." The December 9 edition "Science Daily" provided this preview.

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