There is an awful lot that everyone involved in the heart-rending case of Charlie Gard agrees on. No one disputes that the 11-month-old boy, currently a patient in the intensive care unit at Great Ormond Street Hospital (GOSH), has an extremely rare genetic condition – mitochondrial DNA depletion syndrome (MDDS) – that causes death in early childhood.
The variant that Charlie inherited affects many organs, principally the brain and muscles, depleting those tissues of the energy needed for them to function. He remains alive because of artificial ventilation; he can’t move, he is deaf, he suffers from epilepsy, and his vision is not developing normally. Everyone accepts that there is no cure. And all agree that there is no currently recognised treatment to ameliorate the effects of the disease.
The case is commanding enormous public attention because of the crucial disagreements between Charlie’s parents and the medical experts in his case. These boil down to three areas: the degree of permanent brain damage already sustained; what Charlie’s quality of life is like (particularly whether he is experiencing pain); and whether an experimental treatment – nucleoside therapy – might improve his condition, however imperfectly and temporarily. As Charlie’s parents raised more than £1m through a crowdfunding campaign in order to take him to America for nucleoside therapy, GOSH went to the courts.
In April, Mr Justice Francis, sitting in the Family Division of the High Court, agreed with the hospital that this would not be in Charlie’s best interests, and ordered instead that Charlie’s life support be withdrawn in favour of palliative care and a “dignified” death. Over the intervening months, Charlie’s parents have appealed that decision all the way to the European Court, meeting at every stage with the same judgement.
What is really going on here? It is not, at its heart, a dilemma about administering modestly life-prolonging treatment to a child with a poor quality of life and an abysmal prognosis. That goes on up and down this country every single day: kids with incurable cancer being given toxic treatments in the hope of buying them just a little more time with their loved ones, perhaps to manage that holiday of a lifetime to Disney; severely disabled youngsters with catastrophic chromosomal abnormalities being prescribed antibiotics to cure yet another chest infection that might otherwise be the one that will eventually see them succumb.
The issue in the Charlie Gard case is the experimental and uncertain nature of nucleoside therapy. This has not previously been administered to a patient with Charlie’s specific condition, but there are indications from laboratory research – and the use of the therapy in related disorders – that it might just do him some partial and temporary good. Indeed, GOSH itself apparently applied for ethical approval to try the unproven therapy earlier in his illness, but abandoned the idea because of the marked deterioration in his condition during the time it was being deliberated.
I’m quite sure of this: were there to have been previous experience with nucleoside therapy that demonstrated a modest difference to patients like Charlie, then he would be receiving it even now. But because there isn’t, arguments about the degree of reversibility of brain damage that he’s already sustained, and disputes about his quality of life (his parents insist he experiences far more interaction and enjoyment than GOSH recognises, and they are certain that he is not living in pain) are being marshalled to support the don’t-go-there decision.
This is why the case is back at the High Court this week. Since the final failed appeal to the European Court, seven experts around the world have made known their belief that nucleoside therapy has a small but real chance of providing some palliation to Charlie. GOSH, under pressure from Charlie’s parents’ legal team, have gone back to Mr Justice Francis to get his ruling on these developments. At a preliminary hearing on Monday, the judge gave Charlie’s parents, Chris Gard and Connie Yates from south-west London, 48 hours to present any fresh information as to the potential for nucleoside therapy to make some difference. The hearing resumes on Thursday. The judge has said that he will require “dramatic new evidence” to reverse his previous decision.
No one is going to be able to give Mr Justice Francis that – there cannot be convincing evidence as to the role (or otherwise) for nucleoside therapy until it is tried on patients just like Charlie, who number a mere handful worldwide. The decision is still going to come down to extrapolated facts, and the attitudes and beliefs on the part of doctors and researchers. But Mr Justice Francis would do well to recognise what medicine already does for thousands of children and parents every year: gives them evanescent comfort amidst otherwise unremitting despair.