Go tell it on the mountain

Before this site went ‘live,’ I asked a few friends and colleagues to give me feedback on the ‘beta’ version. The response was very generous  (thank you, by the way), though one senior member of the Clinical Studies Group (CSG) did comment that saying young people with cancer get a ‘raw deal’ was something of a negative statement. A fair point. So I suppose I had better explain myself.

Great strides have, of course, been made in the care of children with cancer over the past half century. In the 1960s most children with leukaemia had no hope of survival; now nine out of ten survive the most common form of childhood cancer, acute lymphoblastic leukaemia (1). This progress is, in no small part, due to the work of groups like the Children’s Cancer and Leukaemia CSG. Since 2001, when the group was set up, the number of patients participating in clinical trials and other research studies has increased fivefold.

Survival rates for all childhood cancers now average at about 80%, no small achievement, but even so cancer remains the most common form of non-accidental death in young people throughout Europe. Moreover, as we have entered the 21^st century (2), a kind of plateau has been reached and for many of the more deadly illnesses, there has been no improvement for two or three decades. Brain tumours, neuroblastoma, sarcomas and acute myeloid leukaemia are all examples of illnesses whose disappointing survival rates seem stubbornly intractable (3). Together these form a cluster of rare conditions which make up a significant proportion of the childhood cancer population.

When my daughter, Bethan, was diagnosed with Ewing’s, one of the bone sarcomas, we were told the survival rates were around 70 / 30, which, putting a brave face on it, did not such seem bad odds. However, despite the best the NHS and one of Europe’s leading cancer centres could offer, she fell into the 30% bracket and, three years later, I found myself requesting a review of her case a few months after her death. The review was sensitively handled, but we had to look some hard facts in the face. Nobody knew why she relapsed, nobody expected it and, when it happened, nobody could do anything about it, apart from make her as comfortable as possible for the months she had left. I remember chatting to the Chair after the meeting and asking how this could possibly be in one of the richest countries on earth. She explained that many childhood cancers are too rare to form a viable market for drug company products and we need to get the industry to invest in research and development. ‘But that,’ she said, ‘is a mountain to climb.’

I wish I had 50p for every time I have heard that story since. I was at a training course on cancer biology recently and both the trainer and a trainee from one of the big Pharma companies came up with the same explanation for the lack of research for children. Does this analysis hold water? Well, to give a referenced source, here is what the main network of European Oncologists has to say:

‘Access to innovative therapies developed by pharmaceutical companies has so far been extremely limited for children in Europe, one reason being that paediatric oncology does not represent a large and hence financially attractive area for drug marketing.’ (4)

Indeed the explanation ITCC gives of the progress that was made in the latter half of the 20^th Century is that this was all done without the help of Pharma, thank you very much.

 ‘Cure rates increased from less than 20% in the 1950s to over 75% in the 1990s and this level was achieved without any input from pharmaceutical companies because of limited commercial interest.’ (4)

But, if we are to move up from the plateau the old chemotherapy drugs have hauled us to, then we will need to develop new, smart ‘targeted’ therapies and:

‘more than 90% of anticancer drugs that should be evaluated in children within the next 10 years are expected to come from big pharmaceutical companies or small “start up" companies.’ (4)

Progress can only be achieved through entering the murky world of commerce and reaching some form of settlement whereby big capitalists and small enterprises are harnessed towards social goals. This is something legislators on both sides of the Atlantic have grappled with for decades now.  One such initiative has been the Paediatric Medicines Regulation, a piece of recent European law that so far has not lived up to the great promise it originally appeared to hold. 

A small band of patient advocates will be journeying to Vienna next month to take part in a review of the PMR and to help formulate a plan to make it more effective. Will this make any difference?

Watch this space.