CF gene therapy: Why it's all bollocks

So today after trying on my hats and making some important parking spaces for my slippers I began researching CF gene therapy. Every now and then my mother will wheel spin her Clio into my street and inform me there's been an article in the Daily Mail about how a cure for Cystic Fibrosis is just round the corner arriving just in the nick of time before I wet myself from being knackered.

I feel bad each time I have to explain how she ought not to be reading the Daily Mail, that gene therapy will almost certainly never been developed by any pharmaceutical company with an interest in continuing as a going concern and that anyone with advanced lung disease (i.e. me) probably won't benefit from this therapy anyway as according to the UK CF gene therapy's own website:

"Patients who have quite advanced lung disease are not suitable because the comparatively advanced lung disease in these patients would make it very difficult to deliver the gene and, therefore, reduce the chance of seeing an improvement in lung disease severity. In addition, we are concerned that the treatment may be less well tolerated in patients with severe lung disease, because gene delivery may cause a transient drop in lung function."

None of this however appears on the homepage of the CF Trust's website alongside their request for donations to fund this programme. Which angers me. This seems very exploitative to me. CF patients themselves are usually fairly cognisant of where the bear sits with these treatments and concoctions from our chats and consultations with our CF clinic boffins and carers.

Parents on the other hand know only what they read in the Daily Mail or the begging letters they receive from charities. And parents buy it because it's obviously very difficult to accept that they have produced defective progeny who will be burdened with a grotty lung disease for ever more and they desperately what to see it cured.

The facts of the matter are if charities worked, which is to say, if they cured the diseases they seek to cure, there'd be no diseases by now. Certainly if campaigns and fun running and wearing coloured ribbons or lying in baths full of beans for a week to raise money were all that were needed to cure diseases we'd all be fighting fit. Obviously it isn't.

Most of the money raised by charities goes towards funding the charity itself or the financing of campaigns to raise money. A circular self-serving process if you will. The CF Trust for example raised £8m in 2010, but it had to invest £2.5m to raise that money. The head cheese at the CF Trust earns over £100,000 a year (total salaries £1,25m).

Charities exist so they can exist. If a charity cured the disease they were set up to cure, they'd cease to be necessary, they can't have that. So they want to be seen to be seeking the holy grail of a cure, while simultaneously knowing full well it's a fantasy.

The UK CF gene therapy consortium boffins will have done their job. Those boffins just care about the science of it all. They just want their names in the medical journals as the dudes who developed the therapy, which is fair enough, but they're indifferent to whether it is actually progressed by a pharmaceutical company and actually reaches patients.

In order for this therapy to actually cure anyone or at least have a therapeutic difference that beats the current treatments a major pharmaceutical company will be required to finance the phase III stage of the trials. Pharmaceutical companies have no incentive whatsoever to develop cures.

There's no money in cures. The money is in the medicine, the maintenance of the condition. They invest their money in treatments that will help you live with your condition via expensive repeat prescriptions. The patents they take out on the treatments afford them a monopoly and they whack a 500% mark up on any medicines that really work.

CF only affects 9,000 in this country. Even if Gene Therapy had to be delivered by the patient routinely it still would not offer enough of a reward on the initial investment to develop it. Also, the ironic thing about patents anyway, is that when a treatment works really well or is needed in an emergency for example like the swine flu vaccine tamiflu - the Government will usually lean on the pharmaceutical company to give up it's patent privileges in order to allow a generic copy of the drug to be manufactured or to significantly lower it's prices.

CF does not effect enough people for a cure in the true sense of the word ever to become a reality. The CF Trust however will continue to insist that it can.

Even if their current £6m target is missed and their "once in a lifetime" opportunity missed to realise their goals, they will continue this line and insist to yet another generation of hopeful CF families that their dreams will become a reality. Even if I could, I wouldn't hold my breath.