Printer Friendly

Drug shows promise in sickle cell anemia.

Drug shows promise in sickle cell anemia

Early clinical studies suggest that a drug normally used to treat leukemia may be effective in treating sickle cell anemia, a genetic disorder in which patients produce defective hemoglobin, the oxygen-carrying molecule within red blood cells. Scientists pinpointed the mutation responsible for sickle cell anemia more than 20 years ago, but that finding has not led to an effective therapy. The recent development stems from researcher's observation that the mutation causes mild or no symptoms in people who naturally produce fetal hemoglobin -- a form normally produced only in the womb.

Hydroxyurea, the drug under study, has been used so far to treat about 60 patients with full-blown sickle cell anemia, according to study coauthor George J. Dover, a pediatrician at Johns Hopkins University School of Medicine. Among the 23 patients for whom the researchers have compiled complete data, the drug induced five to produce fetal hemoglobin at levels representing more than 20 percent of their total hemoglobin. In eight patients, fetal hemoglobin rose to a level of more than 10 percent. It represented less than 10 percent of total hemoglobin in five patients,, while another five showed no response. At about the 20 percent level, disease symptoms begin to lessen, Dover says. The researchers do not understand fully how the drug works, but it appears to activate a gene controlling fetal hemoglobin production, he says.

Now hydroxyurea is undergoing a multicenter, 38-patient trial to determine maximum dose levels and the magnitude of the fetal hemoglobin response. But researchers must show more than an increase in fetal hemoglobin, Dover says. They need to demonstrate that this treatment improves patients' lives, which are interrupted by frequent hosptital visits for painful episodes and infections resulting from blocked blood flow to vital organs. So far, it does not seem to produce any serious adverse effects, says Dover, who hopes to begin a controlled clinical trial of hydroxyurea in 1990.
COPYRIGHT 1989 Science Service, Inc.
No portion of this article can be reproduced without the express written permission from the copyright holder.
Copyright 1989, Gale Group. All rights reserved. Gale Group is a Thomson Corporation Company.

Article Details
Printer friendly Cite/link Email Feedback
Title Annotation:from the Johns Hopkins Centennial Science Writers Seminar
Author:Wickelgren, Ingrid
Publication:Science News
Date:Jun 3, 1989
Words:322
Previous Article:Got a light? This carrot keeps going out.
Next Article:Genetic evidence for autism.
Topics:


Related Articles
Sudden death tied to sickle-cell trait.
Breaking the sickle cycle: potential treatments emerge for sickle cell anemia.
Scientists create sickle-celled mouse.
Sickle mice turn anemic.
Ultrasound reveals sickle-cell stroke risk.
Awakened gene aids inherited anemias.
Drug wards off sickle-cell attacks.
Gas therapy for sickle-cell anemia.
GYNOB2 Effect of hydroxyurea on in-vitro embryo development. (Gynecology & Obstetrics).
Zoology and Entomology.

Terms of use | Copyright © 2016 Farlex, Inc. | Feedback | For webmasters