children hospital philadelphia

The combination of deferoxamine and deferiprone has been shown to be particularly beneficial for reducing cardiac iron, but it requires a painful injection/infusion, which hinders adherence. This pilot study aims to investigate the safety of an oral-only combination chelator regimen (deferasirox and deferiprone) in individuals with thalassemia major with poorly controlled iron overload and to assess how well this chelator combination lowers iron stores over one year.

 

This study is sponsored by the Children's Hospital of Philadelphia in collaboration with the Cooley’s Anemia Foundation and the Toronto General Hospital.

 

More information: clinicaltrials.gov

bluebird bio

Gene therapy firm Bluebird Bio awarded $9.3 million from the California Institute for Regenerative Medicine (CIRM) to support a Phase I/II study evaluating bluebird’s LentiGlobin® treatment for β-thalassemia. The trial is scheduled to start in the U.S. next year. The CIRM grant is one of the first to have been made through the Institute’s new Strategic Partnership Awards initiative.

 

Read the News Release for more information

Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body. Patients suffer from poor growth, fatigue, heart failure, endocrine deficiencies, and eventually, many require chronic blood transfusions. There is no approved therapeutic for the deficiency of beta globin chains in beta thalassemia.

 

This trial will study an oral therapeutic which stimulates production of fetal globin, an alternate type which is produced by all humans, but is normally switched off in infancy. This type of globin can compensate for the missing protein in beta thalassemia.

 

More Information: clinicaltrials.gov

Sultan Bin Khalifa International Thalassemia Award

H.H. Dr. Sheikh Sultan Bin Khalifa Al Nahyan, the son and also the current Advisor for the President of the United Arab Emirates, has been deeply committed for the welfare of Thalassemia Community since a long time, having initiated several magnanimous projects for the benefit of Thalassemia Patients and their families.

 

As an extension of his vision, H.H. Dr. Sheikh Sultan Bin Khalifa Al Nahyan has now taken another noble initiative in recognizing these individuals and group efforts, for first time in the World, by establishing Sultan Bin Khalifa International Thalassemia Award in partnership with Thalassemia International Federation.

 

For more information, please visit the official website.

memorial sloan kettering cancer center

Memorial Sloan-Kettering Cancer Center will begin evaluating a new stem-cell-based gene therapy for patients with the inherited blood disorder β-thalassemia. The clinical trial is the first to receive US Food and Drug Administration approval to treat this disease with genetically engineered cells and is a culmination of more than two decades of research led by Memorial Sloan-Kettering investigators.

 

This study is being done to see if the investigators can make the stem cells produce normal red blood cells and hemoglobin. The investigators do this by collecting the stem cells. The genes with mistakes are removed from the cells. These cells are then treated so they have the corrected gene for making normal hemoglobin. These treated cells are given back to the patient through an injection (shot) in the vein. This is also known as gene transfer. In order for the body to accept these cells, the patient will need to receive a low dose of a drug called busulfan. It is a drug that will prepare the body to receive the new stem cells.

 

This study will let the investigators know:

  • If it is safe to give the patient the treated stem cells
  • If the treated stem cells will go into the bone marrow without causing side effects.

 

More Information: clinicaltrials.gov, Cooley's Anemia Foundation