Last week I went to the Greatest Teashop That Can Be Imagined: the Abbey Tearoom in Dorchester on Thames. In a low beamed twelfth century room, with late summer sun dappling in through the trees outside, two tables are laid with cakes (and scones and buttered teabreads). Around this cake-shrine the tea takers worship. At the teashop I met a number of other devotees, all in sombre mood: it was the last Sunday in September, the last day of summer before the local volunteer cake-bakers who run the teashop close up business for the winter.
The previous day I had seen Tom crawling across soft matting to the ball pit at an activity centre where he used to play. Tom is lucky: he is nearly thirteen and, while weak, he is still walking. For the last two years and nine months he has been on the Translarna trial. Translarna has slowed down the loss of muscle and helped to maintain Tom's upper body strength and stamina.
When Tom loses ambulation, he will no longer be eligible to take Translarna. It only has approval for people who can walk. This is because the primary clinical trial test criterion was a six minute walk test. Not because the drug won't continue to support upper body strength and function. The research did collect data on this as secondary clinical endpoints and anecdotal evidence also points to the drug's effectiveness.
This is no cream cake, this is no tearoom. It's not acceptable that new drugs simply close up shop for those who can't walk. Like everyone who is currently on a clinical trial which relies primarily on the six minute walk test, Tom faces not only the loss of ambulation but the actual withdrawal of a drug for Duchenne. The drug we were waiting and hoping for for five years. The only drug that currently exists for Tom.
Everyone affected by Duchenne has their own story of the devastating impact of the lack of clinical trials for non-ambulant people. This is Tom's story. Carl 'Action' Tilson campaigned to his dying day to be included in clinical trials. And I remember Stuart Wickison's words: 'It's too late for me'.
Thanks to Nick Catlin for his recent article on the limitations of the six minute walk test http://nickcatlin.org/2016/09/21/goodbye-to-the-6mwt. Thanks to Jack and Aaron Ebanks for their campaigning trip from Bristol to Birmingham this summer to highlight the need for clinical trials for non-ambulant people. Thanks to all the charities and clinicians for their current work to develop and roll out standardised upper body strength and functionality tests and the use of MRI as primary measures of clinical outcomes in future clinical trials.
