Gene therapy trial for patients with Fanconi anaemia to open at GOSH

A clinical trial is to open at Great Ormond Street Hospital (GOSH) that will test the use of gene therapy during transplantation for the treatment of young people with Fanconi anaemia. 

The trial will be led by Dr Claire Booth, Dr Phil Ancliff and Professor Adrian Thrasher – who is the Gene, stem and cellular therapies Theme Lead at the Great Ormond Street BRC.

Fanconi anaemia is a life-limiting, inherited haematological disorder which causes bone marrow failure in childhood as well as other serious complications. Children can be treated with a bone marrow transplant but the success of this procedure depends on finding a well matched donor for the patient.

The clinical trial due to open at GOSH in the coming months will assess whether gene therapy could offer hope to children lacking a suitable donor for transplant. 

Researchers will make use of the BRC-funded GMP facility to genetically modify the patient’s own stem cells during the trial.