Page URL:

Very small beta-thalassemia gene therapy trial gets investors' blood up

23 June 2014
Appeared in BioNews 759

Two patients with the serious inherited blood disorder beta-thalassemia have been able to stop blood transfusions 12 days after receiving experimental gene therapy.

The LentiGlobin treatment, developed by Bluebird bio, builds on a previous prototype which allowed one patient to stop receiving blood transfusions after 12 months.

This positive clinical data, presented at the 19th Annual Congress of the European Hematology Association in Milan, Italy, offers hope to the 288,000 patients with beta-thalassemia worldwide.

'Thalessemia major patients are very sick', explains Professor Marina Cavazzana-Calvo of the Necker-Enfants Malades Hospital, Paris, who was the principle investigator of the study. 'They have to receive transfusions regularly for all of their life. Because of this huge number of transfusions they suffer from a huge iron overload. To set up a treatment that can make them free of any transfusion treatment should be a great step toward curative treatment'.

Beta-thalassemia is caused by a missing or defective gene which prevents oxygen-carrying hemoglobin from functioning properly. Bluebird’s LentiGlobin treats the disease by inserting a fully functional human beta-globin gene into the patient’s blood-forming hematopoietic stem cells using a modified HIV virus, often referred to as a lentivirus.

In 2010 a beta-thalassemia patient treated with a previous version of the therapy was able to go without blood transfusions for six years (reported in BioNews 576). However, in this instance the patient still required transfusions for 12 months after receiving the therapy. Since then, Bluebird, whose aim is to develop gene therapy products to treat patients with severe genetic and orphan diseases, has improved both the ability of the virus to insert the corrected gene and the process by which hematopoietic stem cells are extracted, and it is these improvements which are responsible for the positive clinical findings.

'Following the [gene] transplant, we're seeing near normal levels of hemoglobin,' said Bluebird's chief medical officer David Davidson. 'These early results far exceeded our expectations'.

Bluebird's shares jumped 70 percent on the interim results from the phase I/II study. The surprisingly strong findings have excited investors and fuelled hope that Bluebird could file with the US Food and Drug Administration sooner than expected.

31 October 2016 - by Sarah Gregory 
Researchers have developed a novel genome-editing technology to correct the defective gene that causes the blood disorder beta-thalassemia...
27 April 2015 - by Isobel Steer 
Six boys with the inherited immune disorder Wiskott-Aldrich Syndrome (WAS) have been successfully treated with a gene-therapy technique that harnesses a 'tamed' HIV virus...
30 June 2014 - by Emma Hughes 
In Wales, the subject of access to medicines has recently been in the spotlight. We are calling on the National Assembly to review the use of the 'exceptionality' rule in determining whether a patient can access a treatment through the IPFR process...
30 June 2014 - by Ayesha Ahmad 
An Australian fertility company has seen the price of its shares rise on the first day of its public listing on the stock market....
19 December 2011 - by Dr Zara Mahmoud 
Scientists have successfully used gene therapy to alleviate the symptoms of the blood disease haemophilia B in six human volunteers, raising hope for a potential cure. The study has been hailed as a landmark trial for gene therapy...
13 December 2010 - by Julianna Photopoulos 
Scientists have scanned the entire DNA of an unborn child from the mother's blood sample for the first time to safely check for genetic disorders...
20 September 2010 - by Dr Lux Fatimathas 
A patient with the genetic blood disorder beta-thalassaemia has been successfully treated using genetically-altered stem cells, according to US and French researchers...
8 December 2008 - by Evelyn Harvey 
By Evelyn Harvey: A new method for early detection of genetic diseases in unborn babies using a simple blood test can detect the inherited condition beta-thalassemia, according to a study published in Proceedings of the National Academy of Sciences. Although the technique, which analyses cell free fetal DNA (cffDNA) present...
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.