In a gene therapy trial promoted by Genethon, teams from Necker children’s hospital working with teams from Inserm and Université Paris Cité, at Institut Imagine, have identified 51 biomarkers that could predict the success of this treatment.
The gene therapy trial for chronic septic granulomatosis, a severe, rare immune system disorder, involved taking blood stem cells from patients, genetically correcting them using lentiviral vectors, and then reinjecting them. Two of the four children treated showed an effective response to this gene therapy. Study of the patients’ genetically corrected cells identified 51 specific biomarkers for inflammation in the 2 patients for whom treatment was not effective.
The clinical trial promoted by Genethon is the result of work by Dr Anne Galy, as part of the European project Net4CGD, in collaboration with GOSH teams led by Prof. Adrian J. Thrasher, and the Clinical Investigation Centre for Biotherapy (CIC-BT) of the Institut Imagine led by Prof. Marina Cavazzana.