Federal health advisers yesterday endorsed the approval of a drug to treat heart failure in African Americans, which would make the controversial pill the first medicine targeted at a specific racial group.
The Food and Drug Administration advisory panel voted unanimously to recommend that the agency approve a request by NitroMed Inc. of Lexington, Mass., to sell the drug BiDil for patients with severe heart failure, and a majority agreed with the company that its label should say it is specifically intended for African Americans. The agency is not bound by the panel's decision but usually follows the advice.
The closely watched vote marked a crucial step for the drug, which has triggered intense debate, coming amid intensifying efforts to tailor "personalized" treatments to the genetic makeup of individual patients and groups of patients. Supporters say the drug would represent one of the first steps in that direction, offering an urgently needed treatment to a group that suffers more from many health problems and has been long neglected by medical research.
Opponents say marketing the drug this way would be an alarming development that would promote racial stereotyping and the discredited idea that there are fundamental genetic differences among races. They also condemn the plan as an attempt to exploit race for economic reasons. The company has two patents for BiDil, and the one covering its use for African Americans extends an additional 13 years.
"This development with BiDil is a foot in the door -- the first step of what could be seen as racialized medicine," said Troy Duster, a New York University sociologist. "Race is too crude a measure. We should be looking at the individual and his and her biochemical makeup -- not whether he or she is black or white."
But the company and its supporters applauded the decision.
"If approved by the FDA, BiDil will provide new hope to black heart-failure patients," said NitroMed's Manuel Worcel.
More than 700,000 U.S. blacks suffer from heart failure, in which the heart is progressively weakened by heart attacks, high blood pressure, diabetes or some other factor, leaving victims increasingly weak, struggling for breath and eventually dying. Blacks are especially prone to it, are often younger when the condition is diagnosed, tend to respond more poorly to existing treatments, and are more likely to die from it.
BiDil combines two drugs -- isosorbide dinitrate and hydralazine -- that have been used separately for years to treat chest pain and high blood pressure. The combination appears to boost levels of nitric oxide in the blood, helping to expand blood vessels.
Studies have suggested that blacks tend to have lower levels of nitric oxide, and researchers noticed during studies in the 1980s that the drug combination, while appearing to offer no benefit in the general population, may be useful among black patients. A follow-up study involving 1,050 patients who identified themselves as African American was stopped early and released in November when it concluded that the drug significantly improved the quality of life, reduced the likelihood of being hospitalized by 39 percent and cut the chance of dying by 43 percent.
During yesterday's hearing, NitroMed officials and scientists and several outside researchers detailed that study and other evidence supporting BiDil's use among black patients.
"It is all consistent with the hypothesis that there is a better outcome in the black patients," said Jay N. Cohn of the University of Minnesota Medical School.
Panel members peppered the researchers and company representatives with questions about how the studies were conducted. Vivian Wang of the National Institutes of Health's National Human Genome Research Institute noted that the patients in the primary study "self-identified" themselves as African Americans, leaving their race open to question.
"If we are going toward genomic science, we have to look very carefully at the issue of self-identified populations," Wang said. "We may be satisfied with that, but I am not."
But the panel chairman, Steven E. Nissen, a Cleveland Clinic cardiologist, said that until genetic tests become more widely available, race is a useful substitute.
"What we're doing here . . . is using self-identified race as a surrogate for genomic-based medicine," Nissen said. "I wish we had a gene chip, but in the absence of that we have some evidence that African Americans have a robust response to the drug."
Several advocates for improving health among blacks endorsed the drug, saying the treatment would mark a landmark advance.
"This is the most important advance in the treatment of black people that I have seen in my lifetime," said Charles L. Curry, president of the International Society on Hypertension in Blacks.
But several health advocates and researchers voiced concern about labeling BiDil as a treatment only for "African Americans," saying that would discourage its use by many people who might benefit even if they are not black.
"There is no . . . scientific basis on which to claim race-specific efficacy for BiDil," said Jonathan D. Kahn of Hamline University, a law professor specializing in racial issues in health.
Several speakers argued that approving BiDil for blacks would perpetuate the misconception that blacks and whites differ significantly on a biological level, which historically has been used to justify discrimination. Race tends to represent differences in social characteristics rather than meaningful physiological attributes, given that there are greater genetic differences within racial groups than among them, they said.
"The effort to stereotype this drug as a race drug needs to be universally decried," said Gary Puckrein of the National Minority Health Month Foundation, part of a coalition of black health organizations that endorsed BiDil's approval but rejected labeling it as exclusively for African Americans.