Gene therapy fulfilling its promise

Dear ESGCT members and gene/cell therapy stakeholders,

We wanted to draw your attention to a recent publication in N. Engl. J. Med. that once again highlights the progress in the gene therapy field. The article by Aiuti et al. (http://content.nejm.org/cgi/content/short/360/5/447) showed that gene therapy is a safe and effective treatment for SCID in patients with ADA deficiency and can restore immune function. Of the 10 patients treated, 8 had excellent and persistent immune reconstitution in the absence of enzyme replacement therapy, obviating the need for a protective environment to prevent infection. The enclosed insigthful commentary on the same issue by D. Kohn and F. Candotti (http://content.nejm.org/cgi/content/short/360/5/518) nicely overviews the road map to this successful gene therapy story and strikes an upbeat note. Though the field of gene therapy had faced some early setbacks, there is now steady progress on multiple fronts. The overall progress has also been highlighted in a recent commentary by D. Williams , editor-in-chief of Mol. Ther. (http://www.nature.com/mt/journal/v17/n1/abs/mt2008277a.html) based on the recent ESGCT conference that was organized in Brugge (Belgium) last November including advances in gene therapy for congenital blindness, adrenoleukodystrophy, cancer, Parkinson's disease and other disorders.

Though there will still be challenges ahead, the momentum is clearly building up thanks to the commitment of many scientists, clinicians and regulators to this promising field. As gene therapy stakeholders, we feel privileged to be sitting in the front seat during this exciting journey and to witness first-hand the continued progress on the clinical front and in preclinical studies.

On behalf of the ESGCT Board,

Sincerely,

Thierry VandenDriessche, PhD

President European Society of Gene & Cell Therapy

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