Innovative therapies for red cell and iron related disorders


Synthetic report

made by Pr Clara Camaschella (Milan, Italy) for the EHA-ESH-ENERCA Scientific Workshop

The EHA-ESH Scientific Workshop “Innovative Therapies for red cell and iron related disorders” organized in collaboration with ENERCA took place in Cascais, Portugal, April 16-18, 2010. The aim of the workshop was to discuss recent and original data that may result in new therapeutic strategies for red cell and iron metabolism disorders.  

Hot topics discussed were: HSC transplantation for beta-thalassemia with both related and unrelated donors, the controversial indication to transplantation in severe sickle cell anemia, the problem of gene therapy for beta-thalassemia, including the possibility of ex vivo HSC expansion, genetic modification and modalities of overcoming insertional adverse effect of integrating vectors. The molecular basis of the hemoglobin switching by BCL11A was also discussed in the light of fetal hemoglobin reactivation in hemoglobinopathies.  

In the field of iron special emphasis was given to the regulation of the hepcidin pathway as a potential target of therapeutic manipulation to treat iron overload by hepcidin agonists and to treat anemia of inflammation by hepcidin antagonists. The use of low dose iron chelators was discussed to relocate misdistributed iron in neurodegenerative disorders and anemia of inflammation. The relevance of Genome Wide Association Studies to identify genetic modifiers of beta-thalassemia phenotype and new approaches to the molecular pathophysiology of sickle cell syndromes were addressed. The potential protective effect of heme oxigenase-1 in conferring the well known selective protection against malaria in hemoglobinopathies and other congenital hemolytic disorders raised great interest. The ENERCA network and aims were presented in the Poster Session.  

Notwithstanding the Workshop took place during the volcano ash cloud it was unanimously considered a success. Several new advances and ideas are now available to the ENERCA community.

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