July 5, 2017

expert reaction to the Charlie Gard situation

Courts have ruled against continuing life-preserving treatment for 11 month old Charlie Gard. Since this ruling, offers of support and experimental treatment have come from both the US and Italy.

Prof. Dominic Wilkinson, Consultant neonatologist and Professor of Medical Ethics at the University of Oxford, said:

“The decision, today, to invite a US medical expert to review Charlie Gard is a sensible, ethical solution to the divided opinions about his rare illness. To date, all of the experts who have examined him and have had access to his detailed medical records have felt that continued life support and the proposed experimental treatment would be futile. That includes the independent medical expert from the UK invited by Charlie’s parents to assess him. There have been different views expressed by some experts and non-experts particularly from overseas, but it isn’t clear whether those represent different reasonable assessments of the benefits and burdens of treatment, or a lack of appreciation of Charlie’s actual clinical situation.

“Faced with diverging expert opinion, the judge has sought a rational and ethical compromise. If the US expert visits and examines Charlie and remains convinced that the treatment has a real chance of significant improvement, it would be ethical for Charlie to receive the treatment that his family so ardently desires.”

Prof. Jonathan Montgomery, Professor of Health Care Law at University College London, said:

“This case is about Charlie and about what the evidence tells us. The parents stated in April that his life was not worth sustaining unless treatment is available. They believe that Charlie’s condition has now improved and the previous finding of brain damage was incorrect, and they say that they have been given new evidence that suggests the chance of Charlie living as a normal boy that may be as much as 10%.

“First, the factual basis of these claims must be established. The chances of success are then a matter of expert judgment on what can reasonably be inferred from the facts. In March, the US doctor who was prepared to offer the treatment said that he could not predict the outcome. It is not clear what has changed. If the evidence does show a 10% chance of recovery for Charlie it will clearly be in his interests to take that chance, but just hoping it’s true doesn’t make it true. The duty of the court is to examine the evidence.

“Unlike the USA, English law is focussed on the protection of children’s rights. The USA is the only country in the world that is not party to the UN Convention on the Rights of the Child; it does not recognise that children have rights independent of their parents. It is right to take the views of Charlie’s parents very seriously and to look hard at the evidence, including their observations of Charlie’s condition,  to see whether it supports their hope. But this case is about Charlie’s rights and what the evidence tells us that they require. That will be the only consideration of the judge at the hearing.”

Dr Dan Hawcutt, Senior Lecturer in Paediatric Pharmacology at the University of Liverpool, said:

“In some ways very little has changed since last week.  This remains a horrible situation for the child, family, and staff, and my every sympathy goes out to all involved. As Charlie has a right to privacy, GOSH staff are not able to talk to the public about his condition, and so it is still only the family, staff and courts that have all the information.  The possibility of success of a new treatment is therefore impossible for the rest of us to comment on in detail.  But the decision of the court will be all about Charlie’s best interests and will take pain, suffering, symptoms, chance of success, risks and more into account.

“Having said all this, and even though the courts have previously ruled that ongoing treatment is not in his best interest, he continues to receive 24/7 care from dedicated medical and nursing teams at GOSH. This involves reviewing him regularly, including: managing his symptoms related to his underlying disease; ensuring his breathing via the ventilator is at the right level and keeping the airway open; caring for his skin and washing him; measuring his temperature, pulse, respiratory rate, and other vital signs.

“If during these medical and nursing reviews he had demonstrated any new symptoms or signs (positive or negative) then these would also be used to guide his day to day management, and fed back to the court to help them to decide on his best interest. It is not the case that the medical and nursing teams stopped caring for him after the last court ruling; and their focus remains steadfastly on what they believe is best for him.”

Prof. Uta Frith, Emeritus Professor of Cognitive Development at UCL, said:

Research on social echo chambers suggests that there is particularly rapid transmission for emotional-moral information. This leads to escalation and polarisation because of an automatic bias to assume that if the same message is heard thousands of times it comes from thousands of independent people. In fact the voices are not independent. They are repeating the message that they have heard from just one person. That is why it is so heartening to hear the judge say he won’t be swayed by tweets.

Scientific information strives to stay clear of moral-emotional content and always rests on probabilistic reasoning. We can never be 100% certain about our facts and theories. Emotion, which is utterly certain, wins in comparison. However, scientific reasoning cannot be completely stripped of emotion. Perhaps this is an asset we need to cultivate more in science communication. Reasoned evidence needs champions to engage the hearts of people.

Prof. Jonathan Montgomery, Professor of Health Care Law at University College London, said:

“The court will quite properly want to consider whether there is new evidence that suggests there might be a significant chance of successful treatment for Charlie.

“However, it is not clear whether this evidence is new or whether the letter only offers a more optimistic assessment of previously available evidence. At least one signatory has indicated that his evidence was available to the family at the time of the trial. The judge may conclude that there is nothing to justify a reconsideration.

“Second, the court will be concerned that the prospect of benefit may be excessively and cruelly inflated. It is unclear how an estimate of a 10% chance could be made in relation to an untried treatment for a condition that has been said to affect only 16 people, and in relation to which no research has been undertaken, even in mice.

“Third, the court will need to consider whether the parents’ expectations are realistic. Charlie’s mother has expressed the hope that her son could live as a ‘completely normal boy’. From what had been made public, there does not seem to be any basis for this in the letter that has been received. Unless there is solid new evidence suggesting improved chances, it seems likely that the existing assessment – that prolonging Charlie’s life causes him such distress that it is not in his interest to continue treatment – will remain unchanged.”

Prof. Dominic Wilkinson, Director of Medical Ethics at the Oxford Uehiro Centre for Practical Ethics, University of Oxford, said:

“Any court decision is based on the evidence that is presented. The High Court made an ethically sound decision that Charlie Gard should not receive requested experimental treatment and that life support should be withdrawn. This was on the basis of convincing expert testimony that such treatment had no realistic prospect of helping him.

“However, given the claims of new evidence about the possible effectiveness of this treatment it is appropriate, indeed vital, that the decision is reviewed. The court needs to urgently assess whether there is, in fact, a realistic chance of helping Charlie with this treatment. If a review of this new evidence means that this treatment would be in Charlie’s best interests it should be provided, but that is by no means certain.”

Dr Dan Hawcutt, Senior Lecturer in Paediatric Pharmacology at the University of Liverpool, said:

“This is clearly a horrendous situation, and my every sympathy goes out to the young boy, his family, and the medical and nursing teams looking after him.

“I am a paediatric pharmacologist (so specialise in children’s drugs and treatments). I do not work in GOS, am not an expert in mitochondrial illnesses, and do NOT know any more about this patient than is in the press (summarised nicely in the GOS FAQ webpage).

“Apart from the doctors treating him, the parents, and the courts who have had access to his medical data, no one knows how severely affected by his condition this young boy is. The condition he has can cause irreversible damage to his brain (and other organs) – by which I mean are the cells there now dead, and no therapy can help. In order for the courts to make a decision, they will have had all this evidence presented to them. On each occasion it has been presented to a court, they have considered what is in his ‘best interests’ – and after all the evidence, decided it is not something that will help him.

“The team who signed the letter, while having great expertise in mitochondrial illness, do not have any additional information about the patient than any other member of the public. As such, speculating about the potential impact of this treatment on him is problematic. It is very positive that GOS have once again looked to get the courts to help support such a difficult decision.

“It would seem that this could be easily solved by giving out more information about his condition, but the clinical team cannot, and rightly have not done this. This would strip him of his confidentiality. It is therefore important to remember that this is not a PR battle. I cannot contribute to the debate, beyond repeating these generalities and commenting on public domain documents. No-one can.”

Prof. Alastair Sutcliffe, Professor of General Paediatrics at UCL, said:

“Poor Charlie has a very rare disease in which the organelle which provides energy source used in daily cell life, called the mitochondrion, has a gene defect. The reason mitochondrial diseases are rare is because they are usually fatal. They show up in babies or young children more often than in later life.  If not fatal they are progressive and cause serious neurological illnesses which cannot be cured.

“Charlie has one of those most severe of mitochondrial diseases and is untreatable. Medicine is advancing at a wonderful speed but some illnesses are still fatal.

“Gene therapy is in its infancy and is a promising field of human endeavour.  But there are 6000 inheritable recessive conditions in humans – the prospect of them being cured is some time off.

“When a decision to withdraw life support is made for a baby this is not taken lightly and there are often tears in the medical and nursing staff looking after such a baby. But ultimately there is not a cure for many rare diseases. 40% of all rare diseases are in children under age 5 years, and of those most are fatal.  The combined burden of such conditions in the UK is such that 1 in 300 people have a rare disease.

“Scientists and medical technology companies are making strides to find cures or treatments for many conditions. But alas, in the case of poor little Charlie, there are simply limits to medicine as we know it.”

Prof. Julian Savulescu, Uehiro Chair in Practical Ethics at the University of Oxford, said:

“Charlie Gard should have been allowed to go to the US for experimental treatment back in April (or better January when it was first considered), not because he would have been cured but just because we couldn’t then be confident his life would have been “intolerable”, or not worth living. The rational strategy was to give a trial of treatment, say 3 months, and agree with the family to withdraw ventilation if there was no improvement. If this had been done we would now have some information on whether there is any prospect of improvement.

“The critical quote in the judgment of Justice Francis in the High Court back in April is from the independent US expert in nucleoside replacement therapy, Dr I: “He said that he thought that the treatment, if administered, was unlikely to be of any benefit to Charlie’s brain. He described the probability as low, but not zero.” Dr I said that if Charlie were his patient, he would push for a trial of treatment.

“Is 3 months of suffering associated with intensive care worth taking for a 1/10 000 chance of improvement? This is a value judgement about which there is reasonable disagreement.

“The state should not have to pay for expensive experimental treatment with low prospect of success but Charlie’s parents have raised the funds. Charlie should have been allowed to go straight away (and saved hundreds of thousands of pounds of scarce British taxpayer funds which have been used to provide months of intensive care) provided a reasonable physician would treat him in the US. Dr I appeared to be a reasonable and responsible physician.

“This is not a religious or right to life argument, or an argument based on compassion. It’s a secular ethical argument about the extreme complexity of judging someone’s life to be not worth living, or the prospects of having a life worth living not worth taking. The courts have deferred to one group of doctors who are experts in the facts but they are not experts in the ethics.

“More than six months have passed since experimental therapy was first considered. We don’t know how bad Charlie’s brain damage is now. Whether experimental therapy is still warranted depends on whether there remains any prospect of any meaningful life.  Perhaps the moment has passed.”

Prof. Jonathan Montgomery, Professor of Health Care Law at University College London, said:

“Charlie’s parents made it clear in the court proceedings that his present life is not worth sustaining unless treatment is available. There has now been a full judicial review of the evidence about the proposed treatment that has shown that it would be likely to cause him harm and has no reasonable prospect of doing good. It would be wrong to ignore that evidence.

“This is not a case where Charlie’s parents have not been listened to. It is a case where their hopes for improvement are not justified by the evidence that they and others have put before the courts. The law has great respect for the views of parents and it is strongly committed to the protection of life. However, it also recognises that this is a case about Charlie’s interests and that they must ultimately prevail. Parents are not entitled to override those interests, but they are entitled to a full hearing to ensure that nothing has been overlooked. That has happened and we now need to respect the outcome as determined by the courts.

“The case is tragic, but we owe it to Charlie to take decisions based on evidence. Hope requires some foundation if it is to justify subjecting him to harm.”

Prof. Penney Lewis, Professor of Law and Co-Director of the Centre of Medical Law and Ethics at King’s College London, said:

“The European Court of Human Rights has confirmed the decisions of the English courts that continued treatment is not in Charlie’s best interests, after weighing the burdens of continued treatment against any possible benefits. The courts found that it is likely that Charlie experiences pain, suffering and distress, and that the nucleoside treatment offered in the US has no prospects of success, would offer no benefit, and would prolong his suffering. The theoretical possibility of benefit from the nucleoside treatment had disappeared after Charlie suffered brain damage from seizures earlier this year. These findings were based on extensive evidence from all of those involved in caring for Charlie, and from a number of independent experts including one chosen by Charlie’s parents, and the doctor based in the US who is offering to treat Charlie.”

Dr Giles Birchley, Senior Research Associate in Surgical Innovation and Bioethics at the University of Bristol, said:

“Research shows that doctors are exceptionally committed to reaching a consensus with parents. Because of this only the most desperate cases reach the courts. I’d take this as an indication that if there was even the remotest chance any child could be helped, doctors would have agreed.

“Nobody in their right mind would not feel deeply upset by the plight of any terminally ill child. It is natural to reach out to that child’s poor parents, whose suffering is dreadful. But putting any terminally ill child through an experimental treatment which cannot make them better will not help either that child or their parents. It will only prolong that child’s hurt and suffering.”

Prof. Neena Modi, Professor of Neonatal Medicine at Imperial College London, said:

“The Royal College of Paediatrics and Child Health will not comment on individual cases, but recognises that situations like this are tragic and extremely difficult for everyone involved. The principles underpinning the RCPCH framework for practice in these kinds of situations are clear: the child’s interests are paramount; child rights must be respected; doctors must act within the law”.

John Harris FMedSci., Professor Emeritus, University of Manchester and Visiting Professor in Bioethics, King’s College London, said:

“When I read ‘it seems heartless’ not to do everything possible for Charlie and that ‘Charlie should be given every chance’, the agonising question – to which the answer can never be completely clear – is ‘every chance of what?’

“What is an appropriate and heartfelt response to Charlie’s predicament?  ECHR said the Court had determined that ‘it was most likely Charlie was being exposed to continued pain, suffering and distress and that undergoing experimental treatment with no prospects of success would offer no benefit, and continue to cause him significant harm’.  This is not a respectable objective.”

Prof. Sian Harding, Director of the BHF Cardiovascular Regenerative Medicine Centre at Imperial College, said:

“Mitochondrial diseases are cruel because they strike babies and young children, who rapidly deteriorate. It is because there is no cure that the scientific and medical community have concentrated on pre-conception mitochondrial therapy, and it has been an enormous advance that this is now licenced by the Government. It allows parents with these mutations to have healthy children, though sadly cannot help babies already born.”

Prof. Dominic Wilkinson, Director of Medical Ethics at the Oxford Uehiro Centre for Practical Ethics, University of Oxford, said:

“The sad and difficult case of Charlie Gard is the latest in a series of court cases in the UK when parents and doctors have disagreed about medical treatment for a child. It is, sadly, common for doctors and families to need to make decisions about life support for a critically ill child. In almost all cases, parents and doctors make decisions together about what would be best. Cases of disagreement are rare, and needing to go to the court is rare.

“The reason why these decisions come to the court at all is because parents do not have an absolute right to make medical decisions for their children. Parents will not always make the best choices, but for the most part the state will not interfere or intervene. However, where parents’ decisions run a significant risk of causing serious harm, their decisions should be challenged, if necessary in a court.

“These decisions about life-sustaining treatment for critically ill children are fraught and difficult for all involved. Charlie Gard’s parents have passionately fought for what they sincerely feel would be best for their child. Parents are, rightly, at the heart of the decisions that are made in intensive care. Their views about treatment are important, and their wishes are usually followed. However, there are limits.
“Sadly, reluctantly, doctors and judges are justified in concluding that continuing life support is not always helpful for a child and is in fact doing more harm than good. Providing comfort, avoiding painful and unhelpful medical treatments, supporting the child and family for their remaining time: sometimes that is the best that we can do, and the only ethical course.”

Declared interests

Prof. Montgomery: Prof. Montgomery is also Honorary Fellow, Royal College of Paediatric and Child Health; General Editor, Butterworths Family Law Service; Chair of the Health Research Authority; and a member of the European Group on the Ethics of Science and New Technologies. He was involved in the development of good practice guidelines for children in research in 1992 and on decisions about withdrawal of treatment from children in 1997.

Prof. Harding: Prof. Harding is on the Working Party of the Nuffield report “Novel
techniques for the prevention of mitochondrial DNA disorders: an ethical review”

None others declared.