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Content archived on 2024-04-30

Re-activation of fetal haemoglobin synthesis as a means to improve the quality of life of patients with haemoglobinopathies


- To create a network of Clinical Units and Laboratories which deal with thalassemia and sickle cell syndromes in order :
- To exchange expertise and technology with regards to the agents or drugs which may induce fetal hemoglobin synthesis.
- To obtain valid evaluations of several pending therapeutic questions by pooling results in common registries and to develop reliable pre-clinical tests systems.
- To upgrade research with regards to the underlying mechanisms.
- To reduce efforts and unnecessary expenses, and
- To collaborate effectively, but also to compete successfully, with other non-European Centers which carry out similar research.

The present proposal aims to upgrade the scientific content and to increase the safety of various clinical trials which are about to start in several European Centres with the goal to improve the life of patients with thalassaemia major and sickle cell disease by complementing their red cells with fetal haemoglobin (HbF). HbF stops being synthesized in the normal adult but may re-appear under the influence of various drugs or other agents which act by various mechanisms and include the butyric acid and its derivatives, erythropoietin and hydroxyurea or other cytostatics. These substances are now under active evaluation.

Over the past years, the above evaluation took place only in the US. European Centres have just started being active in this field but they still lack coordination and provision for pooling and comparative evaluation of the results. The present Concerted Action aims to bridge this gap by supporting the members of the European Group for the treatment of the haemoglobinopathies (formed under the auspices of the European Association of Haematology) who signed this application. This Action is imposed by the fact that b-thalassaemia and sickle cell disease now constitute a severe Public Health problem not only in the Mediterranean countries, where they occur in high frequency, but also in several countries of Northern Europe, where the number of "immigrants" from the Mediterranean countries, India and the Black Africa has lately increased to an alarming degree. The socio-economic impact of these diseases is extremely severe because they are chronic and incurable (more than 10,000 ECU/year-patient by gross estimation). The above factors havegenerated a lot of pressure, support and scientific efforts towards some form of novel non-conventional therapies. Within this context the proposed CA foresees the design and development of multi-arm trials which will answer several crucial questions with regards to effi-cacy, cost and short- or long-term toxicity of the agents which will be studied. To achieve these goals, participants will be given the possibility to have regular meetings, exchange information, obtain standardised materials and reagents, and use common electronic data bases. A further objective is to devise, standardize and quality control methods which would allow quantification of the response, and to develop systems for in-vitro preclinical testing such as the erythroid cultures, in order (a) to study the drugs currently available and to test those which will be proposed in the near future, and (b) to identify the patients who may respond better than others, thus saving expenses and increasing safety.

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Université de Paris VII
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1,Avenue Claude Vellefaux 1
75475 Paris

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