According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma Inc., recently announced the release of results from a Phase 2 study which tested the drug luspatercept as a therapy for patients with the blood disorder beta thalassemia. The trial included patients that were transfusion dependent and those that were not. Luspatercept is an experimental therapy that has been developed collaboratively between Acceleron and Celgene.
About Beta Thalassemia
Beta thalassemia is a group of genetic disorders which affect the blood. These disorders are characterized by the abnomal production of hemoglobin, the substance in red blood cells which allows them to transport oxygen. The severity of the disorder can vary widely from person to person and depends on the present mutation. These mutations affect the HBB gene which is found on chromosome 11. These mutations can be easily passed down within families. Symptoms of beta thalassemia major, the most severe form, include spleen problems, skeletal abnormalities, poor growth, anemia, liver problems, diabetes, osteoporosis, and heart failure. Treatment for beta thalassemia may include blood transfusions, surgery, and bone marrow transplant, which can be curative for some children. There is a need for improved treatments for patients with severe cases. To learn more about beta thalassemia, click here.
About The Study and Luspatercept
In the open label Phase 2 study the patient participants received subcutaneous treatments of luspatercept every three weeks. The five year extended portion of the study, which will evaluate long term outcomes of treatment with this drug, is ongoing.
Luspatercept has displayed the potential to be of significant benefit for beta thalassemia patients. Currently, many patients are forced to get frequent blood transfusions, which can be problematic because of the inevitable iron overload that eventually occurs after receiving several transfusions. Luspatercept may allow patients to reduce the frequency of their transfusions because it allows patients to produce more normal hemoglobin on their own.
Luspatercept is also being tested in ongoing clinical trials as a therapy for myelofibrosis and low risk myelodysplastic syndromes. The companies are hoping to submit marketing applications for this experimental drug by the middle of 2019.
Check out the original study findings here.