TIF was excited to participate in the 25th European Hematology Association (EHA) Annual Virtual Congress, held this year virtually on 11 – 21 June 2020. Dr Androulla Eleftheriou (TIF Executive Director) and Ms Lily Cannon (TIF Operations Manager) have attended the following satellite symposia on 11 June:
- MDS AND β-THALASSEMIA: MINIMIZING TRANSFUSIONS AND IRON OVERLOAD TO OPTIMIZE PATIENT OUTCOMES discussed by Prof. Ali Taher (Lebanon) and Prof. John Porter (UK)
- TARGETING GLYCOLYSIS WITH A PYRUVATE KINASE ACTIVATOR AS AN INVESTIGATIONAL ORAL TREATMENT FOR PK DEFICIENCY, THALASSEMIAS, AND SICKLE CELL DISEASE discussed by Dr Kevin Kuo (Canada)
- β-THALASSEMIA GENE THERAPY FROM RESEARCH TO REAL WORLD: APPLYING LESSONS LEARNED discussed by Dr Janet Kwiatkowski (USA), Dr Andreas Kulozik (Germany) and Dr Evangelia Yannaki (Greece).
MDS AND β-THALASSEMIA: MINIMIZING TRANSFUSIONS AND IRON OVERLOAD TO OPTIMIZE PATIENT OUTCOMES
The wider benefits of reducing transfusions and iron overload in β-thalassaemia patients were discussed by Prof. Ali Taher (Lebanon) and Prof. John Porter (UK), international experts in the field. Prof. Porter furthermore presented information demonstrating the reduction of serum ferritin levels in patients that had received luspatercept whilst comparing this novel therapeutic agent with other curative approaches. Pending authorisation of luspatercept outside the USA, the impact of minimizing transfusions will be felt more emphatically in many parts of the world, where thalassaemia patients are required either to travel long distances for transfusions or indeed where blood is not readily available and its safety questionable, Prof. Porter commented. Prof. Taher and Prof. Porter agreed that the full meaningful benefit to patients of the drug and its impact on quality of life, has yet to completely transpire, but in the coming years a possible paradigm shift in the management of thalassaemia patients may be on the cards, changing the natural history of the disease.
TARGETING GLYCOLYSIS WITH A PYRUVATE KINASE ACTIVATOR AS AN INVESTIGATIONAL ORAL TREATMENT FOR PK DEFICIENCY, THALASSEMIAS, AND SICKLE CELL DISEASE
The results of the phase 2 clinical trial investigating Mitapivat (AG-348) in thalassaemia were presented by Dr Kevin Kuo (Canada). Mitapivat, a pyvrate kinase (enzyme) metabolizer, works by increasing the energy levels in RBCs thus reducing oxidative damage, thus reducing hemolysis and ineffective erythropoiesis and better regulates iron metabolism. This leads to an increase of haemoglobin. The study, conducted with NTDT patients, has shown an increase of haemoglobin at least 1.0g/dl. Based on this proof-of-concept Agios Pharmaceuticals Inc. is progressing to the development of a phase 3 clinical trial in TDT and NTDT patients.
β-THALASSEMIA GENE THERAPY FROM RESEARCH TO REAL WORLD: APPLYING LESSONS LEARNED
A comparison between the conventional treatment of thalassaemia, gene therapy and gene editing was discussed by the panel of experts, comprised of Dr Janet Kwiatkowski (USA), Dr Andreas Kulozik (Germany) and Dr Evangelia Yannaki (Greece), highlighting the continued necessity of long-term monitoring of patients who have thus far (with over 5 years follow up) presented positive results. Most remarkably, patients who have undergone gene therapy, even with more severe forms of thalassaemia, still maintain higher haemoglobin levels and remain transfusion free for at least 12 months. The experts furthermore commented on the importance of patent adherence to conventional treatment regimens and low iron levels which will allow them to be in a good physical condition and thus eligible for innovative therapies as they become available. The discussion furthermore explored the German experience with gene therapy in thalassaemia, as the first country where it is commercially available, including the criteria and process of becoming a qualified treatment centre.