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Related donor stem cell transplantation using the alemtuzumab/ TBI platform has been shown to be a safe strategy to cure severe sickle cell disease. However, due to a lack of suitable donors, many patients cannot benefit from this strategy. Alternative donor sources are desperately needed to fill this gap. Nearly all patients will have a haploidentical family member who would be able to donate. The use of post transplantation cyclophosphamide has greatly improved the outcome of haploidentical stem cell transplantation. The investigators propose to combine this with alemtuzumab/TBI conditioning.

More information:, ITHANET Clinical Trials


Propanolol is a beta blocker which has been found to inhibit the ability of epinephrine to upregulate sickle red cell adhesion to laminin and endothelial cells in vitro. The purpose of this pilot study is to administer one dose of propanolol to children with sickle cell disease and to measure pre and post dose red cell adhesion. The hypothesis is that a single dose of propanolol will decrease red cell adhesion to laminin and endothelial cells as compared to baseline.

More information:, ITHANET Clinical Trials

bluebird bio

Bluebird Bio today announced that the first subject with beta-thalassemia major has been enrolled in its phase 1/2 HGB-205 study in France and has undergone infusion with bluebird bio’s LentiGlobin drug product in an autologous hematopoietic stem cell transplantation.

“We believe gene therapy represents a potentially new and exciting treatment option for patients with severe forms of beta-thalassemia and sickle cell disease,” stated Marina Cavazzana, MD, Professor of Medicine at Paris Descartes University and Research Director at the Centre for Clinical Research in Biotherapy, Necker Hospital, Paris France. “The beta-hemoglobinopathies are the most prevalent inherited disorders worldwide, and they result in substantial morbidity and mortality. The HGB-205 study builds on early and encouraging clinical data from the LG001 study based on the pioneering work of Professor Philippe Leboulch and colleagues at the Universities of Paris and Harvard, Inserm and the French Atomic Energy and Alternative Energies Commission (CEA) research center in Paris.” "It is very gratifying for our research, manufacturing and development teams to see their efforts to improve the potency and scalability of our product platform finally reach the clinic for patients with this life threatening disease. This milestone brings us closer towards realizing our vision of making hope a reality for patients with limited therapeutic options,” stated Dave Davidson, MD, bluebird bio’s Chief Medical Officer.

More information: Bluebird Bio Website, Press Release


Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions. The purpose of this study is to investigate FibroScan readings in patients with sickle cell disease and iron overload secondary to blood transfusions, and to correlate the FibroScan results with MRI R2*. The primary hypothesis is that the results of FibroScan will correlate with the results of the abdominal MRI R2*.

More information:, ITHANET Clinical Trials

ITHANET logo trans600

Following the presentations and discussion at the ITHANET Meeting at the TIF World Congress 2013, we contact you regarding a collaborative project entitled "Genotype/phenotype correlation studies for β-thalassaemia patients", which is coordinated through the ITHANET portal. The project aims to develop a universal set of markers and techniques for stratification of β-thalassaemia and SCD patients into treatment subgroups for:

  1. onset and frequencies of blood transfusions,
  2. choice of iron chelation,
  3. induction of foetal haemoglobin,
  4. prospective efficacy of gene therapy.

For genotype/phenotype correlation studies and patient stratification, detailed clinical data for patients with different genotypes will be collected. Currently more than 1000 patients with 14 different genotypes, as combinations of HbS and four β-thalassaemia mutations common in the Mediterranean region,have been included in the study. In order to expand the genotypic repertoire of the study, we are calling for new collaborators who are eager to have their own patients analysed and subcategorised and who are thus willing to contribute to this study with samples and data.

For more information, please contact This email address is being protected from spambots. You need JavaScript enabled to view it..

More information about the project: ITHANET project, ITHANET Forum.