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The Boston Globe


Don't Handcuff Scientists

October 20, 2008

By Thomas P. Stossel

IT'S Nobel Prize season to reward medical breakthroughs, and the Lasker Foundation recently conferred its "American Nobel," the DeBakey-Lasker Award for Clinical Research, on biochemist Akira Endo for his discovery in 1976 of the first statin, a chemical that prevents heart attacks and strokes by reducing the concentration in blood of "bad" (LDL) cholesterol.

An underappreciated dimension of Endo's accomplishment, which ultimately saved millions from death and disability, is the risk of drug development undertaken in the private sector. Absent industry's commitment to medical research and a few executives willing to tolerate enormous financial risk, Endo would not have been honored with the Lasker-DeBakey Award.

Endo made his discovery as an employee of Sankyo, a pharmaceutical company, negating the accusation that companies simply expropriate discoveries of publicly funded academic researchers to make profits. Moreover, only companies have the skills to transform chemicals such as Endo's statin into drugs safe and effective enough for human consumption.

However, events almost kept statins from clinical use. When Endo published his findings in scientific journals, other companies started searching for statins. One of these companies was Merck, whose researchers demonstrated by the early 1980s that a statin could lower LDL cholesterol in the blood of healthy volunteers without side effects.

But Merck discontinued statin development upon learning that Sankyo abandoned its statin program after discovering what seemed to be cancerous changes in experimental animals fed large statin doses. Despite a well-established association between high LDL cholesterol levels and cardiovascular complications, researchers worried that reducing blood cholesterol would cause side effects, because cholesterol is an essential component of body cells.

Statin development stalled for three years until Edward Scolnick assumed a research leadership role at Merck. Scolnick devoted a large fraction of Merck's research budget to overcoming concerns about statin toxicity, and the results convinced the Food and Drug Administration that the findings that killed Sankyo's program were not really cancers and that proceeding with human trials of stain therapy was reasonable.

Prior to the discovery of statins, the only way to lower blood LDL cholesterol was to combine an unpalatable diet with medications causing unpleasant side effects. A clinical trial had concluded that this regimen could reduce cardiovascular complications of high LDL cholesterol, but few believed patients would stomach it. When the FDA approved Merck's statin in 1987 simply on the basis of its ability to lower cholesterol safely, the stage was set for a revolution in the treatment of heart disease.

A subsequent clinical trial completed in 1994 confirmed that Merck's statin also reduced cardiovascular events. This watershed study set the stage to demonstrate subsequently that more potent statins could lower LDL cholesterol even further, with greater clinical benefits.

The success of statins required luck, perseverance, and great expense; but, above all, a passionate champion and a willingness to take risks.

Over 30 years after Endo's pioneering research, it is sobering to speculate whether it would come to fruition today. The controversy surrounding Merck's painkiller Vioxx so empowered anti-industry critics that imagining any company (or the FDA) taking similar risks today is difficult, given the toxicity data that came close to killing the industry's work on statins. Critics vilified the same Edward Scolnick and Merck for developing Vioxx after it unexpectedly promoted the very same cardiovascular problems prevented by statins. One has to wonder if medical product companies are shelving potentially life-saving products because industry critics are pushing policymakers and regulators to near zero tolerance for rare adverse events.

These decisions are made outside of the public eye - as opposed to the media frenzy that attends every drug side effect, real or imagined - so we'll never know how many lives excess caution costs. Also unclear is whether now the FDA would approve a statin based on its LDL cholesterol-lowering ability alone, without definitive cardiovascular event data. Had the FDA taken that approach in the 1980s, many patients would not have had their lives saved by statins during the seven years required to obtain such information.

Thankfully, Akira Endo and the Lasker Award remind us of what is possible - and how much there is to lose.

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