Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Beyond Hydroxyurea: New and Old Drugs in the Pipeline for Sickle Cell Disease
source: Blood
year: 2016
authors: Telen MJ
summary/abstract:Despite Food and Drug Administration (FDA) approval of hydroxyurea to reduce the frequency of vaso-occlusive episodes, sickle cell disease (SCD) has continued to be treated primarily with analgesics for pain relief. However, elucidation of the multiple pathophysiologic mechanisms leading to vaso-occlusion and tissue injury in SCD has now resulted in a burgeoning effort to identify new treatment modalities to prevent or ameliorate the consequences of the disease. Development of new drugs as well as investigation of drugs previously used in other settings have targeted cell adhesion, inflammatory pathways, upregulation of hemoglobin F, hemoglobin polymerization and sickling, coagulation, and platelet activation. Although these efforts have not yet yielded drugs ready for FDA approval, several early studies have been extremely encouraging. Moreover, the marked increase in clinical pharmaceutical research addressing SCD and the new and old drugs in the pipeline make it reasonable to expect that we will soon have new treatments for SCD.
organization: Division of Hematology, Department of Medicine, and Duke Comprehensive Sickle Cell Center, Duke University, Durham, NC, USADOI: 10.1182/blood-2015-09-618553
read more full text
Related Content
-
SCDAA Gene Therapy Town Hall – Webinarhttps://www.youtube.com/watch?v=J77Gq9yI...
-
What’s Inside My Medicine Cabinet?Living with sickle cell disease, I rely ...
-
Cannabinoid receptor-specific mechanisms to alleviate pain in sickle cell anemia via inhibition of mast cell activ...Sickle cell anemia is a manifestation of...
-
Don’t Let Sickle Cell Disease Limit Youhttps://www.onescdvoice.com/wp-content/u...
-
Use of telemedicine to improve access to clinical services and treatment for children with sickle cell diseasePatients with sickle cell disease (SCD) ...
-
Early clinical trial data show gene therapy reversing sickle cell anemiaAfter over a decade of preclin...
-
CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell DiseaseThe California Institute for Regenerativ...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.