Real Progress towards Gene Therapy Trials through the International FA Gene Therapy Working Group
Tuesday, October 25th, 2011
The 2nd Meeting of the International FA Gene Therapy Working Group was held at the FARF 23rd Annual Scientific Symposium in Barcelona on 20th October, 2011 with the meeting co-sponsored by Fanconi Hope, FARF, and the Spanish group, the Centre for Biomedical Network Research on Rare Diseases (CIBERER). The meeting was again ably chaired by Prof Jakub Tolar of the University of Minnesota.
The following is a synopsis of Professor Tolar’s summary of the proceedings of the meeting as presented at the conclusion of the Working Group Meeting and in the full FARF Research Symposium the following day. (Text approved by Prof Tolar).
The progress made through the activities of the International FA Gene Therapy Working Group has caused an acceleration in the movement from the concept to the reality of world scale collaborative clinical gene therapy trials for Fanconi Anaemia.
In the first meeting the best intellectuals & experts in FA were brought to the table to seek common ground in efforts to accelerate the transition from gene therapy research into clinical trials on FA patients.
Without collaborative efforts there will be flashes of brilliance with nothing practical coming out of them. People have realised that nobody is smart enough to do this alone and there is significant, even exponential value in combining forces.
Through the agreements reached within the Working Group, all the links in the chain and all the building blocks were put in place and care was taken to ensure that this would produce interpretable readout to be shared amongst the community.
In this second meeting, the group were able to move this process forwards, demonstrating that the first coordinated GT trials will be starting in the very near future.
The group is now looking to the second generation of FA GT trials, ensuring that the necessary funding mechanisms are identified and in place.
For affected families, for whom gene therapy has held promise for the last 20 to 25 years but failed to deliver, we can now say that the gene therapy community is close to answering this with solid action, with a system that is not going to fail.
The collaborative activities of the International FA Gene Therapy Working Group have now moved us into a new era and the challenge to all is to move to something genuinely useful to the patient.
Dr Patel, who is recognised as a world expert in molecular research related to Fanconi Anaemia, is being funded in this research programme by the US Fanconi Anaemia Research Fund (FARF). Fanconi Hope’s contribution to this funding represents a significant proportion of the total required over the next year and demonstrates our commitment to support key FA researchers in the UK.
