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A New Way to Unclog the Arteries
The New York Times,
November 9, 2003

Last week’s report of an experimental treatment that seems to remove plaque from clogged arteries is potentially good news for legions of people threatened with cardiovascular disease. If the findings can be verified in larger and longer studies, the medical profession may have entered a new area in treatments to ward off heart attacks and strokes. Yet this therapy might never have been pursued were it not for a fluke discovery that made patenting possible. Otherwise there would have been little financial incentive for any company to develop the treatment for clinical use.  

Although the results announced last week are more suggestive than conclusive, they clearly have scientists excited. Intravenous infusions of a genetically engineered protein actually caused fatty deposits on artery walls to diminish in volume and thickness. The rate of reversal, after just five weekly infusions, far exceeded anything previously achieved with drugs or diets used for much longer periods. The substance infused contained a genetically engineered variant of the key protein in high-density lipoprotein cholesterol, the so-called good cholesterol.  

For some time now, pharmaceutical companies have been trying to develop pills that might stimulate the body to produce its own H.D.L. cholesterol, thus far with no great success. An alternative approach, infusing H.D.L. cholesterol directly into the body, was shown effective in animals more than a decade ago, but the industry never really pursued it. One reason was that companies saw little economic incentive in using a normal body protein for therapeutic purposes, since it would be hard to gain patent protection. A medicine that could be made and sold by anybody had little potential for profit. That problem was circumvented in this case by using a mutant form of protein discovered among some 40-plus inhabitants of a small Italian village. That made the drug unique, and patentable.  

Several companies are exploring different approaches to develop their own H.D.L. pills or infusion therapy, increasing the likelihood that science may find a new weapon against clogging of the arteries. That’s good news. But the fact that such a promising treatment was widely ignored because there was no immediate profit potential is disturbing. In theory, the nation’s great web of government-financed medical research institutions should step in to promote development of the kinds of drugs and therapy that industry regards as unprofitable. This story makes one wonder how many similar gaps exist in the vaunted American research establishment.

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