Urea normalizes cells – Science News, December 19, 1970
Intravenous infusions of urea now seem to promise successful treatment of patients with sickle cells … Urea causes sickle cells to return to their normal donut shape. To date, intravenous infusions of urea have alleviated sickle cell crises in 22 patients tested.
Sickle disease causes painful clots that can cause strokes and damage organs. Bone marrow transplants, which began in the 1980s for sickle cell patients, are a cure, but finding a donor can be a challenge. In 1998, a urea-based compound called hydroxyurea became the first drug approved by the U.S. Food and Drug Administration to treat sickle cell disease. Until then, without a transplant, doctors could only relieve symptoms with painkillers and blood transfusions.
Hydroxyurea is also used today. Because the compound can be toxic at high doses, scientists have continued to look for better treatments. Ongoing clinical trials in the United States are using the CRISPR / Cas9 gene editing tool to edit the DNA of patients with sickle cell cells in an effort to definitively overcome the disease (SN: 31/08/19, p. 6).