...
It seemed, at the outset of this hearing, that there might have been a lone voice in the USA that was offering what has been described in some reports as “pioneering treatment”. Understandably, Charlie’s parents have grasped that possibility, they have done all that they could possibly have done, they have very publicly raised funds. What parents would not do the same? But I have to say, having heard the evidence, that this case has never been about affordability, but about whether there is anything to be done for Charlie. At one stage GOSH got as far as deciding to apply for ethical permission to attempt nucleoside therapy, a treatment that has never been used on patients with this form of MDDS. But by the time that decision had been made, Charlie’s condition had greatly worsened and the view of all here was that his epileptic encephalopathy was such that his brain damage was severe and irreversible, that treatment was potentially painful but incapable of achieving anything positive for him.
I was aware that I was to hear evidence from the doctor in the USA who was,reportedly, offering what had been referred to as pioneering treatment. Before he gave evidence, I encouraged the treating consultant at GOSH to speak with him,which she was able and willing to do. I am truly grateful to these experts for the time that they have given to this case. The outcome of that discussion is illuminating and the doctor in the USA said the following:
“Seeing the documents this morning has been very helpful. I can understand the opinions that he is so severely affected by encelopathy that any attempt at therapy would be futile. I agree that it is very unlikely that he will improve with that therapy. It is unlikely.”
However, the US doctor made it clear that, were Charlie in the US, he would treat him if the parents so desired and could pay for it. As I have already said, funding in this case is not in issue. The US doctor also confirmed during this telephone conversation that he had never treated with nucleoside therapy anyone who had encephalopathy, therefore he was unable to indicate from any scientific basis whether a patient with encephalopathy would respond positively.
Charlie suffers from the RRM2B mutation of MDDS. No one in the world has ever treated this form of MDDS with nucleoside therapy, although patients with a different strain, TK2, have received nucleoside therapy, with some recorded benefit.In mouse models the benefit to TK2 patients was put at 4% of life expectancy. There is no evidence that nucleoside therapy can cross the blood/brain barrier, which it must do to treat RRM2B, although the US doctor expressed the hope that it might do so. There is unanimity among the experts from whom I have heard that nucleoside therapy cannot reverse structural brain damage. I dare say that medical science may benefit, objectively, from the experiment, but experimentation cannot be in Charlie’s best interests unless there is a prospect of benefit for him.
The GOSH team believe that Charlie can probably experience pain, but is unable to react to it in a meaningful way. Their evidence was that being ventilated, being suctioned, living as Charlie does, are all capable of causing pain. Transporting Charlie to the USA would be problematic, but possible. Subjecting him to nucleoside therapy is unknown territory, for it has never even been tested even on mouse models, but it may (or may not) subject the patient to pain, possibly even to mutations. But if Charlie’s damaged brain function cannot be improved, as all agree,then how can he be any better off than he is now, which is a condition that his parents believe should not be sustained?
It is with the heaviest of hearts, but with complete conviction for Charlie’s best interests, that I find it is in Charlie’s best interests that I accede to these applications and rule that GOSH may lawfully withdraw all treatment save for palliative care to permit Charlie to die with dignity. I want to thank the team of experts and carers at GOSH, and others who cannot be named, for the extraordinary care that they have provide to this family. Most importantly of all, I want to thank Charlie’s parents for their brave and dignified campaign on his behalf, but more than anything to pay tribute to their absolute dedication to their wonderful boy, from the day that he was born.
