The nation's first ethnic drug -- a pill for black patients with heart disease -- may soon be heading to a pharmacy near you.

The drug, called BiDil, is the brainchild of Dr. Jay Cohn, a heart specialist at the University of Minnesota. And it's being hailed as a major breakthrough for a deadly disease that, some have claimed, strikes blacks at higher rates than others.

But as the drug speeds toward approval, it has run afoul of critics. They say that it's irresponsible and unscientific to imply that BiDil works better on people of one race than another.

"There's no evidence that the results for any therapy are significantly different for African-Americans," said Jay Kaufman, an epidemiologist at the University of North Carolina who studies racial disparities in health care.

On Nov. 8, Cohn and his colleague, Dr. Anne Taylor, will unveil the results of a major trial of BiDil, involving more than 1,000 black patients, that could pave the way for approval by the Food and Drug Administration (FDA).

For Cohn, it's the culmination of a 30-year struggle to prove that his idea to combine two old drugs -- now repackaged as a single pill called BiDil -- can save lives. At the same time, the research has placed him in the middle of a touchy debate about the role of race in medicine.

The drug's manufacturer, NitroMed Inc., has asked the FDA's approval to market BiDil specifically as a heart drug for blacks.

Cohn believes that the drug also works in whites and other groups. For almost 20 years, he has been giving the generic equivalent to patients without regard to race.

But he agrees that the company had good reasons to pursue BiDil as an ethnic drug. The sponsor chose to test it only in black patients, he said, because earlier studies suggested they were more likely to respond than others.

"We've proved that BiDil is an effective treatment for heart disease," Cohn said. "Now, who is it effective in? In African-Americans, I now know that."

The study was halted in July after early results showed a "significant survival benefit" from the drug, according to NitroMed of Lexington, Mass. A scientific panel concluded "it would be unethical" to keep giving some of the participants a placebo. That news sent the company's stock up 73 percent in one day. The official results will be released Nov. 8 at the annual meeting of the American Heart Association.

But critics such as Jonathan Kahn, a law professor at Hamline University in St. Paul, say that no matter how successful, the study doesn't justify attempts to promote BiDil as an African-American drug. "The trial itself says nothing about whether it works better in blacks," he said, because no one else was tested.

Kahn said the race angle was largely a marketing strategy to salvage a drug that previously had been rejected by the FDA. "BiDil did not begin as an ethnic drug," he wrote in a critique in the Yale Journal of Health Policy, Law and Ethics in February. It only became one five years ago, he said, after researchers struck out at efforts to win wider approval.

Packaging it this way, he and others argue, could have dangerous consequences, misleading patients and reinforcing old myths about racial differences.

Yet the research has been embraced by many black organizations, including the Association of Black Cardiologists and the Congressional Black Caucus.

"When we see something that can close the gap, we need to use it, we need to celebrate it," said Gloria C. Lewis, director of Minnesota's Office of Minority and Multicultural Health. "This is not a race issue; this is a life-and-death issue."

A long search

Cohn wasn't thinking about race in the early 1970s, when he began searching for a new way to treat heart failure. At the time, there weren't many good treatments for the disease, a slow deterioration of the heart that can cause fatigue, swelling, difficulty breathing and other symptoms.

He decided to test two drugs -- hydralazine and isosorbide dinitrate -- that had been used for years on other heart-related conditions.

Since the patents on both had long expired, no drug company was interested in paying for studies. Then in 1980, Cohn launched the first of two studies with money and patients from the Veterans Administration. And it showed that the combo did, in fact, lower the death rate from heart failure.

As it happened, about a third of the 600 patients in that study were black. But he wasn't looking for racial differences at the time.

Over the next 10 years, two drug companies asked the FDA for permission to promote the drug combo as a treatment for heart failure. Both times, the FDA said there wasn't enough proof.

In 1999, Cohn took a fresh look at his old study, this time through a racial prism. "There was this striking difference," he said. The effect "was dramatic in the black subgroup, and was barely discernible [in whites]."

Armed with this new insight, Cohn and his new corporate partner, NitroMed, asked the FDA for approval once again, this time for pill called BiDil combining both drugs. Once again, the FDA said it wanted more proof. So in 2001, NitroMed launched the African American Heart Failure Trial, which eventually enrolled about 1,050 black patients at 170 medical centers.

Researchers don't envision BiDil as a single cure-all. It is one more drug in the array of pills that heart-failure patients take every day. In the study, everyone got all the standard medications, plus either BiDil or a placebo.

Taylor, who headed the study, is preparing for a "media circus" when the results are released Nov. 8. "The addition of this drug had an exceptional benefit," said Taylor, a cardiologist and associate dean of the University of Minnesota Medical School.

Fears over race

Many, though, fear that BiDil will lead to racial profiling in medicine.

"If it's approved as a race-specific drug, it means that at a minimum, a lot of non-African-Americans who might benefit from it won't get it," said Kahn, of Hamline University. At the same time, he said, black patients might assume it will help them even if it won't.

Kaufman agrees. "Race has never been shown to be an adequate proxy for use in choosing a drug," he and two colleagues wrote in an essay called "Race and Genomics" in the New England Journal of Medicine last year. There is, he says, too much genetic variation within each race to lump everybody together.

Beyond that, some say that looking for ethnic drugs could distract attention from the root causes of most health disparities, such as poverty or discrimination. "We're utilizing a biological bullet [for] a problem that is largely social and economic in its origins," said Laurie Nsiah-Jefferson, a health policy researcher at Brandeis University in Waltham, Mass.

BiDil's supporters say the fears are overblown.

"I don't think we serve the medical or social community by trying to disregard differences," said Cohn. "I think putting blinders on is a disservice certainly to the medical community. ...We now know that that population, on average, has a dramatic benefit from BiDil. End of story," he said.

Other supporters see a larger story.

"You can look at this and say it's taken us a very long time to truly come up with a truly definitive statement on treating heart failure in African-Americans," said Dr. Clyde Yancy, a cardiologist at the University of Texas Southwestern Medical Center in Dallas. This drug, he said, could be a significant step toward improving their care.

At the same time, he believes that in the future, scientists will find a more precise way to pinpoint who benefits from drugs such as BiDil.

Taylor, meanwhile, says the drug may have a wider reach than first appears. "What we have shown is we can actually save lives," she said. "It works in blacks, but it doesn't mean that's the only group it works on."

She hopes that message doesn't get lost.

"I think that the public's reaction to this is probably going to be more sensible than the philosophers'," she said. "We have to keep in mind that we are saving lives. What's wrong with that?"

Maura Lerner is at mlerner@startribune.com.

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