New advance in the treatment of sickle cell anaemia
Alfuzosin, a drug commonly prescribed for men with chronic prostatitis, a painful disorder of the prostate and surrounding pelvic area, failed to significantly reduce symptoms in recently diagnosed men who had not been previously treated with this drug, according to a clinical trial sponsored by the National Institute of Diabetes and Digestive and Kidney Diseases
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Sickle cell anaemia is a genetic disease characterised by the sickling of red blood cells in patients suffering from hypoxia (around 100,000 cases in Europe and North America).
The red blood cells then adopt a sickle shape, obstruct the blood vessels and cause repeated and very painful crises.
Current treatment involves frequent and substantial exchange transfusions to prevent or treat sickle cell crises.
“These red blood cell transfusions are difficult to manage, however, and sometimes require 10 to 12 bags of packed red blood cells per patient per session. And these patients often have blood groups that are rare in Europe, making the collection of blood bags a complex task. Also, in the long term, they cause highly problematic poly-immunisation phenomena. Faced with this public health issue, ERYtech Pharma’s technology could meet our as yet unmet need to significantly reduce the frequency and number of bags being transfused”, explains Dr Dominique Rigal, Scientific Director of the Etablissement Français du Sang (French Blood Service) of Lyon and Rh?Alpes.
In an in vitro model, ERYtech Pharma researchers have shown a very significant reduction in sickling when the red blood cells of sickle cell patients are mixed with GR-ARA1. This programme is awarded by the Agence Nationale de la Recherche (French research agency) 2008 competition. “We are very happy to receive this recognition which validates our approach both in scientific, medical and economic terms. This grant wouldn’t have been possible without the help of the teams of Dr Cyril Martin from the Université Claude Bernard in Lyon (CNRS E647) and Professor Pierre-Louis Tharaux from the Lariboisière Research Centre in Paris (INSERM 689)” says Dr Yann Godfrin, CEO and the company’s Scientific Director.
“Sickle cell anaemia is the first application for GR-ARA1, but we are in the process of developing it for use in other pathologies. This project, which is in its acceleration phase, is sparking the interest of manufacturers and numerous international transfusion centres. We are entering an active co-development phase”, says Pierre-Olivier Goineau, COO in charge of Business Development.
The Oxygen’ERY System technology consists of encapsulating an allosteric effecter of haemoglobin into erythrocytes to increase their ability to release oxygen in the presence of hypoxic cells.
The GR-ARA1 product has been developed with the aim of increasing tissue oxygenation and treating chronic genetic diseases characterised by hypoxia or severe anaemia such as sickle cell anaemia or thalassemia.
GR-ARA1 was also developed as an adjuvant treatment to the radiotherapy of resistant hypoxic tumours such as glioblastoma or tumours of the head and neck.
Screening with an ultrasound machine has proved highly successful in preventing stroke among children with sickle cell disease, by identifying children who are then preventively treated with blood transfusions. Over an eight-year period at The Children’s Hospital of Philadelphia, researchers found that the technique, transcranial Doppler ultrasonography (TCD), along with regular transfusions for children found
Full Post: Ultrasound helps prevent stroke in children with sickle cell disease
Researchers have identified a gene that directly affects the production of a form of hemoglobin that is instrumental in modifying the severity of the inherited blood disorders sickle cell disease and thalassemia. The discovery could lead to breakthrough therapies for sickle cell disease and thalassemia, which could potentially eliminate the devastating and life-threatening complications of
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A unique approach to bone marrow transplantation pioneered in part by a Children’s Hospital of Pittsburgh of UPMC physician has proven to be the only safe and effective cure for sickle cell disease, according to a new study. Lakshmanan Krishnamurti, MD, a pediatric hematologist/oncologist at Children’s Hospital, helped pioneer a form of bone marrow transplantation
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Itamar Medical, an international medical technology company developing noninvasive diagnostic technology using the Peripheral Arterial Tone (PAT) Signal, announced today the use of the company’s patented Endo-PAT2000 in BioMarin Pharmaceutical Inc.’s phase 2 study of patients with Sickle Cell Disease (SCD). The Endo-PAT2000 was used to measure endothelial function in a study designed to evaluate
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New data, generated in mice, by Pierre Sonveaux and colleagues, at University catholique de Louvain, Belgium, have identified a potential new target for anticancer therapeutics. Not all cells in a tumor are equal, for example, some are in regions rich in oxygen, whereas others are in regions deprived of oxygen (hypoxic regions). It had been
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