(MENAFN- Gulf Times) The potential power of gene therapy to transform the lives of people with sickle cell disease (SCD) has been proven through a world-first breakthrough in Paris, as reported in the New England Journal of Medicine last week. SCD is an inherited disease caused by a gene mutation that results in red blood cells losing their usual donut-like appearance and taking on a sickle or crescent moon shape.
SCD mainly affects people of African, Caribbean, Middle Eastern, Eastern Mediterranean and Asian origin and it is estimated to affect around 5mn worldwide. The patients often have anaemia and get tired easily, run a higher risk of infections and stroke, and experience bouts of severe body pain. Many need chronic blood transfusions, usually every three to four weeks - as a form of treatment for the condition.
A team from the AP-HP university hospital group in Paris, the Imagine Institute of Genetic Diseases and gene therapy company bluebird bio said they managed to get a French teenager off transfusions. The boy was the first person to be treated, in Paris in October 2014, for SCD in a clinical trial with gene therapy. Others have been tested since, but no official results published.
The team collected so-called haematopoietic stem cells, which give rise to red blood cells, from the bone marrow of the youngster, then aged 13. The immature cells were treated with a therapeutic gene, carried in a deactivated virus, which recoded their DNA to correct blood cell production.
The report on the child's health 15 months after treatment revealed he was still doing well after this point, but an official, updated status has yet to be published in a peer-reviewed journal. 'He is well, he no longer needs monthly (blood) transfusions, anti-pain medication, or hospitalisation, study leader Marina Cavazzana told AFP.
SCD is common in Africa, where up to 40% of a country's population can carry the mutated gene, though most never get sick. Early results from a trial in Togo, Mali and the Democratic Republic of Congo suggested the Sickle Scan was a faster, cheaper blood test than existing ones relying on lab equipment, its makers said. Rapid diagnosis is crucial to start SCD sufferers, especially young children, on potentially life-saving treatment.
Philippe Leboulch, a professor of medicine at the University of Paris, told the BBC News website that the same therapy has to be performed in many patients to feel confident that it is robust enough to propose it as a mainstream therapy.
Prof Leboulch is nervous about using the word ‘cure' as this is just the first patient to come through clinical trials. But the study does show the potential power of gene therapy to transform the lives of people with sickle cell.
However, the expensive procedure can only be carried out in cutting-edge hospitals and laboratories, while most sickle cell patients are in Africa. The next big challenge will be to transform this pioneering science into something that really can help millions of people.
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