Compositions for the Treatment of Blood Disorders

Abstract

The invention relates to compositions containing chemical compounds and compositions containing steel factor which stimulate the expression of hemoglobin or globin protein such as embryonic or fetal globin, or the proliferation of hemoglobin expressing and other cells. These compositions can be used to treat or prevent the symptoms associated with anemia, sickle cell diseases, thalassemia and other blood disorders. The invention also relates to methods for administering these compositions to patients and to medical aids for the treatment and prevention of blood and other disorders.

1 . An oral pharmaceutical formulation comprising:
 (1) a pharmaceutically effective amount of a derivative of butyric acid, or a pharmaceutically acceptable salt thereof; and   (2) a pharmaceutically acceptable carrier or diluent, wherein said oral pharmaceutical formulation is in solution as a dispersion, mixture, liquid, spray, capsule or as a dry solid as a powder or pill.   
     
     
         2 . The formulation of  claim 1 , which further comprises a flavoring agent. 
     
     
         3 . The formulation of  claim 1 , which is in the form of a pill, capsule, or tablet. 
     
     
         4 . The formulation of  claim 1 , wherein the pharmaceutically acceptable salt is selected from the group consisting of sodium, potassium, magnesium, calcium, ammonium, and lithium. 
     
     
         5 . A method for treating a hemoglobin disorder without significantly decreasing viability of a hemoglobin-expressing cell and for stimulating proliferation of a hemoglobin-expressing cell comprising the steps of:
 (a) administering an effective amount of an oral pharmaceutical composition comprising:
 (1) a pharmaceutically effective amount of a compound that does not significantly decrease the viability, and stimulates proliferation, of a hemoglobin-expressing cell, wherein the compound is a derivative of butyric acid, or a pharmaceutically acceptable salt thereof; and 
 (2) a pharmaceutically acceptable carrier or diluent, wherein the oral pharmaceutical composition is in solution as a dispersion, mixture, liquid, spray, capsule or as a dry solid as a powder or pill; and 
   (b) monitoring the hemoglobin-expressing cell to ensure that cell proliferation is stimulated and that cell viability is not significantly decreased, thus treating the hemoglobin disorder without significantly decreasing viability of the hemoglobin-expressing cell and stimulating proliferation of the hemoglobin-expressing cell.   
     
     
         6 . The method of  claim 5 , wherein the composition further comprises a flavoring agent. 
     
     
         7 . The method of  claim 5 , wherein the composition is in the form of a pill, capsule, or tablet. 
     
     
         8 . The method of  claim 5 , wherein the hemoglobin disorder is sickle cell anemia. 
     
     
         9 . The method of  claim 5 , wherein the hemoglobin disorder is a thalassemia. 
     
     
         10 . The method of  claim 5 , wherein the pharmaceutically acceptable salt is selected from the group consisting of sodium, potassium, magnesium, calcium, ammonium, and lithium. 
     
     
         11 . A method for stimulating white blood cells comprising administering to a patient a pharmaceutically effective amount of a composition comprising a derivative of butyric acid or a pharmaceutically acceptable salt thereof, wherein the pharmaceutically effective amount of the composition increases proliferation of white blood cells in the patient. 
     
     
         12 . The method of  claim 11 , wherein the method is used to treat a blood disorder. 
     
     
         13 . The method of  claim 12 , wherein the blood disorder is caused by radiation therapy or chemotherapy. 
     
     
         14 . The method of  claim 12 , wherein the blood disorder is aplastic anemia. 
     
     
         15 . The method of  claim 11 , wherein the pharmaceutically acceptable salt is selected from the group consisting of sodium, potassium, magnesium, calcium, ammonium, and lithium.