Thomas Carroll and Bob Dalgleish from Fanconi Hope attended a lecture on Feb 10th by Dr Juan Bueren, Hematopoiesis and Gene Therapy Division, CIEMAT, Madrid

Dr Juan Bueren and Prof Adrian Thrasher

on “Towards the efficient gene therapy of Fanconi anaemia patients”.
The meeting was hosted by Adrian J. Thrasher, PhD, MD, BS, FRCP, MRCPCH, FMedSci, Professor of Paediatric Immunology and a Wellcome Trust Senior Clinical Fellow in the Molecular Immunology Unit at the UCL Institute of Child Health (ICH).

Following the lecture Fanconi Hope had the opportunity to discuss progress in gene therapy in more detail with Dr Bueren and Prof Thrasher. The following is a brief report  from the lecture and subsequent discussion from a layman’s perspective ( Bob Dalgleish).

Progress to date:
Testing to date has been carried out on mice and have indicated that relatively high numbers of BM cells need to be transplanted after genetic correction. Results have shown that 6 months after BMT, sensitivity to MMC is significantly reduced. (MMC is a chromosomal breakage agent used to test for FA).

It was found that adding TNF alpha inhibitors significantly increased the size and numbers of cell colonies. (FA is highly sensitive to TNF alpha). As a

Prof Adrian Thrasher and Mr Thomas Carroll

result, Etanercept, (a TNF Alpha inhibitor) is used in all trials to prevent oxidative damage.

The conclusion for human gene therapy is that stem cells should be harvested as soon as possible to prevent the collection of pre-leukaemic hematopoietic (blood producing) stem cells.
How Close to Clinical trials?
The expectation is that the production of the ’vectors’ need to introduce the genes into FA patients will take around a year. However given that protocols have to be agreed and safety issues fully addressed it is likely to be around 2 years before trials will start in earnest.
Discussion is currently under way with European colleagues to achieve a consensus on the nature of the trials and the vector to be introduced as there are several ways of proceeding.

Trials Funding
Previous trials were funded under the European research framework (Framework Programme 6). However the rules of FP7, the next tranche of funding appear to discount work which simply follows on from FP6. As the nature of the call for FP7 is close to being completed, there is a short time available to attempt to influence this in favour of gene therapy trials for haematological conditions. (See post from Thomas Carroll on website).
UK based gene therapy trials, if not funded by FP7 will be funded from a number of charitable sources.

Fanconi Hope’s Position
Fanconi Hope will do everything possible to support and expedite gene therapy trials

This entry was posted on Friday, February 13th, 2009 at 8:16 am