Memorial Sloan Kettering Researcher Recognized for Stem Cell Therapy Trial for Thalassemia Patients

Pictured: His Highness Sheikh Sultan Bin Khalifa Bin Zayed Al Nahyan & Michel Sadelain

His Highness Dr. Sheikh Sultan Bin Khalifa Al Nahyan awarding Dr. Michel Sadelain the Sultan Bin Khalifa International Thalassemia Award for Innovative Medical Research

Stem cell therapist Michel Sadelain was awarded the Sultan bin Khalifa International Award for Innovative Research in Thalassemia in September 2013.

Other individuals selected to receive awards for excellence in the field of hemoglobinopathies are David Nathan (USA) Prowase Wasi (Thailand), and Phaedon Fessas (Greece) – three co-winners of the Sultan bin Khalifa Grand International Award for Scientific Achievement.

A Global, Hereditary Blood Disorder

Beta-thalassemia is found in people of Mediterranean, Asian, and African descent. It is characterized by the inability of red blood cells to make a protein called beta-globin. In sickle cell disease, a related disorder affecting the same gene, the red blood cells make an abnormal form of beta-globin.

Together, beta-thalassemia and sickle cell disease are the most common, severe hereditary blood disorders worldwide, with several million people affected and more than 50,000 born with these conditions every year.

The current treatment for beta-thalassemia is a lifetime of regular red blood cell transfusions, which are lifesaving but fraught with serious secondary complications. Bone marrow or stem cell transplants can offer a cure, but more than three-quarters of patients are unable to find a matched donor.

Hope for Patients

Dr. Sadelain is leading research at Memorial Sloan Kettering Cancer Center to evaluate a new stem-cell-based gene therapy for patients with the inherited blood disorder beta-thalassemia. The clinical trial is the first to receive US Food and Drug Administration approval to treat this disease with genetically engineered cells.

The potential new treatment is a culmination of more than two decades of research led by Memorial Sloan Kettering investigators. If the trial proves to be successful, the treatment could offer hope of a cure for patients with this severe blood disease, as well as related conditions such as sickle cell disease. Only a handful of diseases are currently treated with this type of gene- transfer therapy, and all but one of them are rare immune disorders.

The trial will eventually be extended to patients at other institutions, including the National Institutes of Health and the University of Washington. Dr. Sadelain’s group is coordinating with investigators in India, Italy, Greece, and Thailand — areas where beta-thalassemia is much more prevalent — to offer the treatment to patients there as well.

In 2012, His Highness Dr. Sheikh Sultan Bin Khalifa Al Nahyan announced his intention to establish the Sultan Bin Khalifa International Thalassemia Award, in partnership with the Thalassemia International Federation, to recognize individuals and groups whose efforts have contributed to the advancement of care for patients with thalassemia and other hemoglobinopathies.

This major new award for the significant contribution of outstanding scientists in the field has been established by the His Highness Dr. Sheikh Sultan Bin Khalifa Al Nahyan Humanitarian & Scientific Development Foundation, and the selection and awarding of candidates was made in partnership with Thalassaemia International Federation, which is based in Cyprus.

The Award winners have been invited to Abu Dhabi to receive their prize at the 13th International Thalassemia Conference on October 20-23, 2013.