Thursday, 13 December 2012

CF Gene Therapy: Small Bet/High Payout


The Cystic Fibrosis Foundation in the US does not provide any funding for the Cystic Fibrosis gene therapy programme based here in the UK (run by the UK Cystic Fibrosis Gene Therapy Consortium “UKCFGTC”).

The UKCFGTC is an innovative form of collaboration. Established in 2001 by Rosie Barnes, then CEO of the CF Trust, it brings together over 50 scientists from Edinburgh University (Centre for Molecular Medicine and Roslin Institute), Oxford University (Gene Medicine Group) and Imperial College London (Department of Gene Therapy, National Heart & Lung Institute).

The single-dose trial of the first generation (lipid-based) product was completed last year. It demonstrated safety and showed very promising results with CFTR protein expression better than VX-770 at the same stage (according to Prof. Eric Alton of Imperial College). 

Multi-dose clinical trials of the first generation product are now under way at the Royal Brompton Hospital in London and at the Western General Hospital and Royal Hospital for Sick Children in Edinburgh. These are due to complete in mid 2014. 

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.

Hopefully when the results of the multi-dose clinical trial are available, the results will justify the next stage of work and the UKCFGTC will be able to raise the capital necessary to get there. The programme continues to be supported by the CF Trust and Rosie Barnes, through her new charity, Just Gene Therapy. The problem is neither of these groups has sufficient resources to fully fund the UKCFGTC. It is too early-stage for Big Pharma and so it is going to need a well-funded new partner to bridge the gap. This could be the CFF (or another "venture philanthropist") or a biotech company with a strategic interest and infrastructure that the UKCFGTC can leverage to get the product to market faster than it could do on its own. Ideally a new partner would be a combination of CFF/venture philanthropist and biotech.

The science in this programme is cutting-edge and brings together the work of specialist teams in a number of global centres as well as the three principal UK teams. The consortium is not competing against other groups; all the leading scientists in the world are working together and it is therefore the only horse in this particular race. The IP situation is complicated but it has been well thought through and does not represent a barrier to third party investment.

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.

Unlike the mutation-specific small molecule drugs being developed by Vertex, Pfizer and others with the backing of the CFF - representing a total funding commitment by the CFF of $133m to date - the CF gene therapy treatment being trialled by the UKCFGTC substitutes correct DNA for faulty DNA, whatever the mutation. So it is a single product with the potential to treat the whole CF population as compared to the small molecule candidates which treat sub-populations and therefore have limited (sometimes “ultra-orphan”) markets.

It has taken much longer to reach this point than it would have done with a more appropriate level of funding. But through the sheer talent and tenacity of the people involved, the UKCFGTC has now got to a point where it has demonstrated the exciting potential of the science. A relatively small financial bet, at this point, would materially accelerate the programme offering any new partner the potential of a high return in regular financial terms and also in terms of world-changing impact with a fat moral dividend attached. There are no guarantees of course; it may or may not prove successful in terms of translation into a new medicine but, if not, at least we will be able to eliminate it quickly and focus on other areas. It does look highly promising though.

It may have been a better use of CFF resources to put at least some of the $58m it gave to Pfizer into other projects such as the gene therapy programme where its money would certainly not be displacing that of Big Pharma and where it would have  a transformational impact on the project.     

You can read all about the UKCFGTC here:

Click here: UK Cystic Fibrosis Gene Therapy Consortium




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