Technology ID

Ionophores as Treatment for Sickle Cell Disease

Lead Inventor
Eaton, William (NIDDK)
Hofrichter, Harry (NIDDK)
Henry, Jeffrey (NIDDK)
Smith, Jeffrey (McKinsey & Company)
Research Materials
Therapeutic Areas
Infectious Disease
This technology includes a method using ionophores to reduce sickling in patients with sickle cell disease. Sickle cell disease is caused by polymerization of a hemoglobin mutant, and the only approved treatment acts by replacing sickle hemoglobin with fetal hemoglobin, thereby increasing the delay time prior to polymerization. This drug is only partially successful because it does not induce fetal hemoglobin synthesis in all cells. The discovery of a drug that inhibits sickling in all cells has been hampered by the lack of a sensitive, quantitative and automated cellular assay for screening that is directly related to the pathogenesis. We have developed such a method and used it to discover that decreasing the intracellular-sickle hemoglobin concentration by increasing the volume of the red cell with ionophores is a powerful method to reduce sickling. Because of the enormous dependence of the delay time prior to polymerization on concentration, the resulting increase in sickling time from small a concentration decrease will be therapeutic because it will allow more cells to escape the narrow vessels of the tissues before fibers can form and result in vaso-occlusion.
Commercial Applications
Treatment of sickle cell disease.

Competitive Advantages
Novel mechanism of action; there are no current drugs that have been discovered to treat sickle cell disease that act by inhibiting polymerization of deoxyhemoglobin, the basic cause of the disease.
Licensing Contact:
Tong, Betty
Phone: 301-451-7836