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.

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ESGCT and BSGT Collaborative Congress 2011, Brighton UK

Fanconi Hope is contribution sponsorship of £1000 for this important gathering of the European Gene Therapy community in Brighton from 20th to 23rd October 2011. As well as having a Fanconi Hope stand in the exhibition area on the 20th we will be providing literature which will be distributed to all attendees at the conference in their delegate pack, explaining the reasons why FA is an ideal candidate for gene therapy research. With many hundreds of delegates and a significant attendance from the Press this is an important forum in which to raise awareness and get our message across.

The President of the British Society for Gene Therapy, Prof Adrian Thrasher, will also be participating in our International FA Gene Therapy Working Group Meeting in Barcelona the previous week.

“The theme for Brighton 2011 is “Letting the science do the talking” so you can expect a focus on cutting edge science, translational research and latest data from right across the world. With topics and extra events for students, young scientists, industry, clinicians and principal investigators at a quintessentially English seaside town, Brighton 2011 is set to be among the best ESGCT Congresses to date”  More information

A report on the first meeting by Prof Jakub Tolar published in Nature can be read in its entirety here

You can read more about the Working Group and the participants at the first meeting here

Our intention is to have an interview with Dr Patel shortly, in particular so that a description of the research programme can be provided in layman’s terms.

Conference Programme

Monday morning session on Fanconi Anaemia:

Congratulations to Simon Boulton, a researcher at Cancer Research UK’s London Research Institute and a member of our UK & Ireland FA Research Network, who has been awarded the 2011 European Molecular Biology Organisation (EMBO) Gold Medal.

Simon, who works at Cancer Research UK’s Clare Hall Laboratories in Hertfordshire and is one of the UK’s key researchers in Fanconi Anaemia, has been chosen to receive the award in recognition of his groundbreaking work on DNA repair mechanisms. more

Annual FARF Symposium Oct 20-23rd 2011, Barcelona.
Each year, the Fanconi Anemia Research Fund holds a Scientific Symposium for doctors, researchers and scientists who study Fanconi anemia to share information and learn more about the latest findings regarding this devastating disease.

Call for Abstracts for the 23rd Annual Fanconi Anemia Research Fund Scientific Symposium

Sponsored by the British Society for Gene Therapy

The UKNSCN conference will continue to showcase the latest and best developments in UK stem cell and regenerative medicine research, with a number of world-class speakers from overseas. more

A group led by KJ Patel from the LMB, together with collaborators at the Wellcome Trust Sanger Institute and CRUK Cambridge Research Institute (CRI), have developed the first model for the human genetic illness Fanconi Anaemia (FA). This genetic condition results in abnormal development, bone marrow failure and a huge lifetime risk of developing cancer. At present the only long-term treatment for FA is bone marrow transplantation. The mouse model will allow scientists to study further the molecular basis of this disease at the whole organism level and allow them to work on novel therapies for FA. more