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.
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:
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 Dr. Marios Phylactides.
Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA.
The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa.
In this research study, instead of using the standard myeloablative conditioning, the study doctor is using reduced-intensity conditioning (RIC), in which lower doses of chemotherapy will be used. Although the lower doses may not eradicate every single stem cell in the bone marrow, nevertheless in the presented combination it still intends to eliminate already formed immune cells, paving the way to successful engraftment of donor cord blood cells. Engrafting cord blood cells can outcompete and reject the patients' few surviving stem cells. With reduced-intensity conditioning, the side effects on brain, heart, lung, liver, and other organ functions are usually less severe, and the patients can have a better long-term recovery. There is also realistic hope that after lower doses of chemotherapy many patients will avoid becoming sterile.
The purpose of this study is to collect data from the patients undergoing reduced-intensity conditioning before UCBT, so that the study doctor can compare it to the standard myeloablative conditioning with the expectation that there will be full therapeutic benefits paired with better survival rate and improved quality of life following the reduced intensity approach compared to myeloablative regimen.
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the time to neutrophil and platelet engraftment, to determine the time to immune reconstitution (including normalization of T, B and natural killer (NK) cell repertoire and Immunoglobulin G production), and to establish the incidence of infectious complications including bacterial, viral, fungal and atypical mycobacterial and other infections following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant disease.
We are pleased to announce the official program and the book of abstracts for the ITHANET meeting at the 13th International Conference on Thalassaemia and Haemoglobinopathies (TIF World Congress 2013). The meeting will be held on 23rd October 2013 at the Abu Dhabi National Exhibition Centre, UAE, between 8:30am and 10:30am, in room Capital Suite 5.
The ITHANET meeting aims to bring together experts in the field of haemoglobinopathies in order to discuss new research ideas and possible collaborations. Existing research projects will be presented, specifically on aspects of epidemiology, novel therapeutic approaches and clinical characterization of patients, as examples of international collaborations and in order to facilitate possible contribution by the meeting participants. This will be followed by a discussion on funding opportunities and future collaborations. Furthermore, the redesigned ITHANET Community Portal and its databases will be presented, inviting suggestions and discussion for the further development of the portal and its role in promoting new collaborations will also be discussed.
Download / View the Book of Abstracts from here (PDF format)