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Clinical trial for rare blood disease

An international team has reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease (X-CGD). 

Six of those patients are now in remission and have stopped other treatments. Until now, people with X-CGD – which causes recurrent infections, prolonged hospitalisations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.

Donald Kohn, a senior author of the paper, said: “With this gene therapy, you can use a patient’s own stem cells instead of donor cells for a transplant.

“This means the cells are perfectly matched to the patient and it should be a much safer transplant, without the risks of rejection.”

The researchers removed haematopoietic stem cells from X-CGD patients and modified the cells in the laboratory to correct the genetic mutation. Then, the patients’ own genetically modified stem cells – now healthy and able to produce white blood cells that can make the immune-boosting burst of chemicals – were transplanted back into their bodies.

Two people in the new study died within three months of receiving the treatment due to severe infections that they had already been battling before gene therapy. The seven surviving patients were followed for 12 to 36 months after receiving the stem cell gene therapy. All remained free of new CGD-related infections, and six of the seven have been able to discontinue their usual preventive antibiotics.  

go.nature.com/3bzpPRz

Image credit | Alamy

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