Since it was created in 2001, the programme has been funded to the tune of £34.3m, of which the CF Trust has provided £30m. This may sound like a lot of money but it equates to £3m per annum and this is very cheap compared to most pharmaceutical R&D projects. The hard reality is the gene therapy progamme is run on a shoestring and it relies on the incredible commitment and determination of a team of very smart scientists. It very nearly collapsed at the beginning of this year but the government did step in at the eleventh hour and, through the MRC and NIHR, provided £3.0m needed to get to the next stage with the first generation product and a further £1.3m to progress work on the second generation (lentivirus-based) product. This money was provided as a grant.
The discoveries and tools coming out of the programme may well be applicable to other "autosomal recessive disorders" like Cystic Fibrosis. These are inherited disorders where an affected person usually has unaffected parents who each carry a single copy of the mutated gene (and are referred to as carriers). Two unaffected people who each carry one copy of the mutated gene have a 25% chance with each pregnancy of having a child affected by the disorder. Other examples of this type of disorder are Tay-Sachs disease, sickle-cell disease, Niemann-Pick disease, spinal muscular atrophy and Roberts syndrome.
Click here: UK Cystic Fibrosis Gene Therapy Consortium