Re: Charlie Gard case: an ethicist in the courtroom
Daniel Sokol (BMJ 2017 358: j3451) argues that the position taken by Great Ormond Street Hospital for Children (GOSH) in not allowing Charlie Gard to undergo experimental nucleoside treatment in the US was more in the spirit of the Hippocratic principles than that taken by his parents. This is because “he has no quality of life and no real prospect of any quality of life.”
However, we argue that if indeed there was a 55% chance of the experimental nucleoside treatment reducing the amount of time on a ventilator and a 10% chance of completely weaning him off, as claimed by Professor Hirano, this could have considerably improved his quality of life and allowed his parents to “have some quality time with their son” as Charlie’s mother expressed in court. Weaning him off the ventilator would have allowed him to come out of the intensive care unit, potentially to be discharged out of the hospital and spend the rest of his short life in the comfort of his parents at home.
The basis of this claim was rejected by the court as there was no published evidence to show benefit in Charlie’s particular mitochondrial DNA depletion syndrome yet Charlie is one of only 4 patients in the world with this condition. Charlie’s parents claimed he was only the 16th person ever to be diagnosed with this condition. It would be impossible to carry out large trials or develop animal models in time for such a rare condition. The proposed nucleoside treatment has been tried with some benefit in less severe forms of the condition. We suggest that it may be justifiable to try novel experimental treatments for the first time in very sick patients who have everything to gain and very little to lose.
We argue that once the experimental treatment had been identified, his case should have been referred to an independent clinical ethics committee with medical and ethical experts represented. We feel the family court is a last resort after all other measures have been exhausted as it adds further delay in the initiation of possible beneficial therapy. As past members of a Research Ethics Committee, we would have proposed a time-limited trial of nucleoside therapy at GOSH. The medication could have been brought over from the US and its administration supervised by a member of Professor Hirano’s team. Such a trial of therapy would have included a clear end-point (time off the ventilator) and agreement to stop treatment if significant side-effects occurred. At a later date, the case could have been referred back to the committee for continuation of therapy beyond the limit of the trial, if meaningful benefit had been demonstrated. Sadly, by the time that Professor Hirano finally came to London and assessed Charlie, it was too late.
We intend no criticism of the dedicated staff at GOSH. Our point relates to how we, as doctors, deal with such complex cases and keep within the spirit of the Hippocratic principles. The Hippocratic Oath demands that the doctor “will use treatment to help the sick according to my ability and judgment, but never with a view to injury and wrong-doing.” We suggest that acting with beneficence in an effort to improve this infant’s quality of life, even if likely to be ultimately unsuccessful, would certainly have been in keeping with the Oath. Furthermore a supervised trial as proposed, would have added to our knowledge and experience with this form of therapy, potentially benefiting others with this condition in the future.
Stephen Hyer, Consultant Endocrinologist, Carshalton
Hervey Wilcox, Consultant Chemical Pathologist, Carshalton
Competing interests:
No competing interests
03 August 2017
Stephen L Hyer
Consultant Endocrinologist
Hervey Wilcox, Consultant Chemical Pathologist
Epsom & St Helier University Hospitals NHS Trust
St Helier Hospital, Wrythe Lane, Carshalton, Surrey SM5 1AA
Rapid Response:
Re: Charlie Gard case: an ethicist in the courtroom
Daniel Sokol (BMJ 2017 358: j3451) argues that the position taken by Great Ormond Street Hospital for Children (GOSH) in not allowing Charlie Gard to undergo experimental nucleoside treatment in the US was more in the spirit of the Hippocratic principles than that taken by his parents. This is because “he has no quality of life and no real prospect of any quality of life.”
However, we argue that if indeed there was a 55% chance of the experimental nucleoside treatment reducing the amount of time on a ventilator and a 10% chance of completely weaning him off, as claimed by Professor Hirano, this could have considerably improved his quality of life and allowed his parents to “have some quality time with their son” as Charlie’s mother expressed in court. Weaning him off the ventilator would have allowed him to come out of the intensive care unit, potentially to be discharged out of the hospital and spend the rest of his short life in the comfort of his parents at home.
The basis of this claim was rejected by the court as there was no published evidence to show benefit in Charlie’s particular mitochondrial DNA depletion syndrome yet Charlie is one of only 4 patients in the world with this condition. Charlie’s parents claimed he was only the 16th person ever to be diagnosed with this condition. It would be impossible to carry out large trials or develop animal models in time for such a rare condition. The proposed nucleoside treatment has been tried with some benefit in less severe forms of the condition. We suggest that it may be justifiable to try novel experimental treatments for the first time in very sick patients who have everything to gain and very little to lose.
We argue that once the experimental treatment had been identified, his case should have been referred to an independent clinical ethics committee with medical and ethical experts represented. We feel the family court is a last resort after all other measures have been exhausted as it adds further delay in the initiation of possible beneficial therapy. As past members of a Research Ethics Committee, we would have proposed a time-limited trial of nucleoside therapy at GOSH. The medication could have been brought over from the US and its administration supervised by a member of Professor Hirano’s team. Such a trial of therapy would have included a clear end-point (time off the ventilator) and agreement to stop treatment if significant side-effects occurred. At a later date, the case could have been referred back to the committee for continuation of therapy beyond the limit of the trial, if meaningful benefit had been demonstrated. Sadly, by the time that Professor Hirano finally came to London and assessed Charlie, it was too late.
We intend no criticism of the dedicated staff at GOSH. Our point relates to how we, as doctors, deal with such complex cases and keep within the spirit of the Hippocratic principles. The Hippocratic Oath demands that the doctor “will use treatment to help the sick according to my ability and judgment, but never with a view to injury and wrong-doing.” We suggest that acting with beneficence in an effort to improve this infant’s quality of life, even if likely to be ultimately unsuccessful, would certainly have been in keeping with the Oath. Furthermore a supervised trial as proposed, would have added to our knowledge and experience with this form of therapy, potentially benefiting others with this condition in the future.
Stephen Hyer, Consultant Endocrinologist, Carshalton
Hervey Wilcox, Consultant Chemical Pathologist, Carshalton
Competing interests: No competing interests