Sickle Cell Drug Saves Lives

Join Our Community of Science Lovers!

On supporting science journalism

If you're enjoying this article, consider supporting our award-winning journalism by subscribing. By purchasing a subscription you are helping to ensure the future of impactful stories about the discoveries and ideas shaping our world today.

Sickle cell anemia, a blood disorder characterized by abnormally shaped red blood cells, is an inherited disease that occurs most frequently in people of African, Indian, Middle Eastern and Mediterranean descent. In the U.S., about 72,000 African Americans currently have the disease. In 1995, the FDA approved the first drug to treat the disorder, hydroxyurea, after trials showed that it reduced the number of painful episodes sufferers experienced and decreased the number of hospitalizations and blood transfusions needed. Now a new report indicates that the drug also saves lives. The findings, published today in the Journal of the American Medical Association, suggest that expanded use of the drug may be warranted.

The original study of hydroxyurea's effects, which was randomized, double-blind and tested for the placebo effect, took place between 1992 and 1995. In the new work, Martin H. Steinberg of the Boston University School of Medicine conducted a follow-up survey of 299 of these patients, who were given the option to continue, stop or start taking the drug. The team determined that among individuals who took the drug over a nine-year period there was a 40 percent reduction in deaths. "Hydroxyurea's ability to reduce complications in the sickest patients is strongly connected to lower death rates," notes study co-author Duane Bonds of the National Heart, Lung, and Blood Institute. "These results also confirm that the benefits of hydroxyurea persist. After the original trial ended, we didn't know if the benefits of the drug would be long term."

It is thought that the drug's beneficial effects stem from its ability to stimulate the production of so-called fetal hemoglobin. In people with sickle cell anemia, molecules of sickle hemoglobin clump together and form long rods that cause red blood cells to become rigid and take on a sickle shape. Increasing the level of fetal hemoglobin inside red blood cells helps to rein in this "sickling." In an accompanying editorial, Debra L. Weiner and Carlo Brugnara of Harvard Medical School call for increased use of hydroxyurea. They note that it "provides hope and help for improving quality of life and lifespan for patients with this devastating disease."

It’s Time to Stand Up for Science

If you enjoyed this article, I’d like to ask for your support. Scientific American has served as an advocate for science and industry for 180 years, and right now may be the most critical moment in that two-century history.

I’ve been a Scientific American subscriber since I was 12 years old, and it helped shape the way I look at the world. SciAm always educates and delights me, and inspires a sense of awe for our vast, beautiful universe. I hope it does that for you, too.

If you subscribe to Scientific American, you help ensure that our coverage is centered on meaningful research and discovery; that we have the resources to report on the decisions that threaten labs across the U.S.; and that we support both budding and working scientists at a time when the value of science itself too often goes unrecognized.

In return, you get essential news, captivating podcasts, brilliant infographics, can't-miss newsletters, must-watch videos, challenging games, and the science world's best writing and reporting. You can even gift someone a subscription.

There has never been a more important time for us to stand up and show why science matters. I hope you’ll support us in that mission.

Thank you,

David M. Ewalt, Editor in Chief, Scientific American