Charlie Gard: a life worth a legacy…

Charlie Gard will probably die in the next few days. His death I hope will be private, peaceful and dignified and my sincere sympathy to his parents Connie Yates and Chris Gard and I truly hope they can move on with their lives after the torture of the last few months.

Charlie’s short, tragic life has been lived too much in the glare of publicity, and as a result many will know of his case and the legal wranglings surrounding it, but I wonder what impact, if any, his tiny life will have on our attitudes to medical miracles.

What have we learnt from this unedifying mess where the Health Service was pitched against battling and desperate parents? The excellent medical staff of Great Ormond Street Hospital, who deals daily with the most difficult of childhood diseases, will not easily forget Charlie given that beyond their exceptional professionalism, they too, like his parents, are only human with human emotions and this must have been a particularly difficult case.

Charlie was born in August 2016 with a rare genetic condition; mitochondrial depletion syndrome which affected many of his organs including his brain and from the start it was clear, to the doctors at least, he would not survive. His loving parents strongly disagreed and the hospital, having done what they could, felt compelled to seek a court order that would allow them to switch off his life support and allow him to die. They won in the Family Division of the High Court, an appeal by the parents was dismissed, the Supreme Court agreed with the doctors as did the European Court of Human Rights. For the parents this is now the end of the legal process.

His parents’ fight was to keep Charlie alive so they could bring him to America for treatment. On the one hand this is completely understandable, commendable even. This is what parents do. Yet on the other hand their fight might be seen as reckless given the potential pain and discomfort Charlie suffered as they clutched at straws. The straw they clutched at was an unknown and untried treatment with nucleosides in America.

America spends vast sums on worthless and sometimes dangerous medicines and medical interventions which reflects its global position half way down healthy nations list. Spent properly they would be the healthiest nation on earth. Sadly American culture supports a cavalier, risky approach to treatment which is more to do with the commercial acumen of physicians rather than the clinical needs of patients. Where they offer a glimmer of hope they make a tidy profit. This is surprisingly seductive to despairing families. The Gard family raised £1.2 million towards Charlie treatment money which will now be given to I hope a UK based medical charity.

A Tyrone family is hugely indebt by keeping their son in America so that he can receive cannabis oil treatment for uncontrolled epilepsy, Wee-Oscar, the exceptionally brave and charismatic Oscar Knox, became a media sensation as he battled neuroblastoma in 2012 and got the all clear after family and friends raised over £250,000 to send him to America for an advanced experimental form of immunotherapy. He died of the disease in 2014.

Our natural instinct is to hope and this makes us believe all too easily in miracles. Modern medical practice does so many amazing things but it does not do miracles. We all need to become more dispassionate and less naive and be clear that, due to the medical culture in America, some American physicians are merely sophisticated purveyors of snake oil and their commitment to profit blinds them to the real needs of their patients. That should be the legacy of Charlie Gard.

,

  • Granni Trixie

    In recent years abortion has crossed over from the moral sphere into political sphere. Looks like end of life decisions could similarly do so.

    As I had a son with muscular dystrophy, I am well placed to have empathy with parents who would do anything, anything to prolong their child’s life. But looking at the decision regarding Charlie Gard more objectively, I think the decision based on his quality of life ( or lack of it) was probably the right one, however heartbreaking for all concerned.

  • SDLP supporter

    This is a heart-breaking case and this must be among the worst things that any family could suffer. I believe that Charlie’s parents have the right to fight to the very end and that a court of law is not necessarily the best place to resolve these awful ethical dilemmas.

  • Croiteir

    A very deeply disturbing case, if I have it correct. The London based medical profession say that they can do no more but in their arrogance they say that they will not allow anyone to do any more either. If I was the parent I would want to have the final say. I too would want to grasp any straw. I too would be willing to go into penury. How much of my money I would want to spend is my business, no one elses. If the state says it has to draw the line that is fine, we all realise that the state must concern itself with the greater good and has these awful decisions to make, even if the medicine can alleviate or cure, but simply is unaffordable. But as a private person I must be allowed to spend my money as I see fit. Even if I am vulnerable. So the money argument in the piece is irrelevant.

    This is no more than the pro abortion mentality now spilling over, logically, into the infancy period of development. Truly awful, completely logical.

  • Hawk

    By a long way and for some time America has been the number one country for drug innovation, in part because of the huge amount of money that companies can earn from creating a drug that actually works. Whilst there are plenty of snake oil salesmen to be sure, there are also plenty of companies producing drugs that are changing lives.

    You said that if the money was spent ‘properly’ the US would be a lot healthier. However if the money was spent ‘properly’ it might kill off much of the innovation for new drugs. The American government recognises as much, it is the reason why there are so few price controls. If price controls kill profits it will also kills the incentive to make a drug that might only ever help 1,000 people.

    The reality is that the America policy on drug pricing, as commercially orientated as it appears to be, and as immoral as it might appear in individual cases, is almost certainly a net benefit to society.

    If we remove all the drugs brought to the market by the massive innovation from America our collective health would be a lot worse. Stop the righteous condescension.

  • Ricardo Gomes

    So in this article are you judging America which granted allready the life of 18 children with similar conditions like Charlie, Just because you in UK want that amount of raised money for U.K. Charity which takes children from their parents (a lot Portuguese families) and let children die because there are no competencies available in U.K. To threat a lot of similar cases like Charlie (like we got know now from the media)…so for you is also all about the money… so people can add this 1.4 million to their pocket without changing anything… U.K. Kills children…that’s murder… the parents of Charlie should leave U.K. With that amount of money…no cent should be left there.

  • Korhomme

    As I understand it, and I have only media reports, Charlie has one of several mitochondrial deficiency syndromes. There isn’t any real treatment for this, beyond a liver transplant for those kids who are suitable.

    Court cases like this occur occasionally, and always make distressing reading. Here, it seems that the Courts had to decide between the ‘rights of the child’ and the ‘rights of the parents’, with I suspect some ‘duty of care’ as well.

    These syndromes hadn’t been recognised when I was a student; at that time, such kids would have ‘failed to thrive’ and then died.

    Medicine has become a victim of its own success in some ways. We are almost at the point where death is seen as a ‘failure of treatment’ rather than inevitable, and where we expect a cure — or at least effective treatment — for every condition.

    A possibility might have been problems with transport to where ever in the US. Charlie is on a ventilator and is tube fed. A medical team with ventilator would have had to accompany him. I don’t know how realistic this might be.

  • Linsey Tucker

    I agree with this statement. It’s a pity so many only see this case in black and white. All these people banging on about children being saved who had conditions that were similar to charlie. First off in genetics, even the slightest difference can have catastrophic effects. The syndrome charlie has is a rare thing to begin with, the type Charlie has being the rarest and most complex…..clinical research can only be carried out on eligible patients….Charlie was not one of them – there were no other trials done for this type of the syndrome because there are only a handful of people suspected to have had it in the entire world. The treatment would only have been able to be tried if the damage done was not so far advanced. There’s no way to reverse that amount of damage and these images of results ppl keep sharing proves nothing. They are reports of comparisons of 2 different scans – with details of any changes. People have took this and ran with the idea of there is no damage; it says so. Not only can i not see any statement on these reports saying that, even if it does there was no result of the original scans to show the original damage….a small change later on could look at lot more meaningful with those images and it’s not the right of anyone to be sharing this information anyway. Genetic syndromes are not like other disabilities or illnesses. They often vary from case to case and have a wide spectrum of varying severity and most have many different forms. I have loeys diets syndrome…..it’s not as rare as Charlie’s syndrome, and was newly discovered in the last 15 years or so….even now though most people won’t have a clue what it is and there have been many misconceptions from people and medical staff of what this syndrome is and how it should be managed. It’s a connective tissue disorder so has an impact on everything pretty much….there is no known cure. Fortunately my brain is not affected but it can be with the nature of this syndrome….along with a whole host of other life threatening situations. The reason I bring this up is because I was told by my doctor (he specialises in treating Loeys Dietz patients who have cardiology and vascular problems that arise from the syndrome. He probably deals with 100+ people some who will have the same type of LDS as I do I. E. The same genetic mutation in the same part of DNA, yet he told me that I am the singularly unique case of LDS patient that he’s got. Nobody with lds presents exactly the same but I am by far the most individually affected in both symptoms and severity especially of certain symptoms that he has come across. Now. Lds , although life threatening if unmanaged, its not anywhere near what charlie suffers. He cannot make a sound. He has no way to express pain he may be feeling. That’s why the treatment can not be tried. Even the doc in USA stated to the courts that he wasn’t aware of the severity of deterioration of Charlie’s brain activity at the time of offering to treat him. (I cannot entertain the idea of any childrens hospital deliberately falsifying health claims of a patient.) I have nothing against the parents appealing. As a parent I would have been doing the same. It’s natural for them to fight with every ounce of strength for their sons life, I can’t begin to imagine the pain they have endured and have every sympathy to their plight. I commend their brave and fierce strength and determination throughout. They cannot be expected to do anything but what their parental instincts tell them. It’s natural. But that is why we have these legal proceedings in the first place. For similar reasons why a medic can not treat a close relative or friend. Their logical judgment would be clouded by their instinct to save and protect their loved one which is what you’d expect. It’s been a difficult case from start to finish but when slamming the courts and the judges and the hospital staff, remember this: these people needed to make a call based on Charlie’s state of health. If these people followed what their own gut was telling them, they wouldn’t be able to deal with the complex situations they deal with every day. They were strong. They had to make that decision for Charlie because his parents would have had love and grief and everything else blocking their logic. Again that is what you’d expect. But it’s why these courts exist. Because it would have been wrong to expect the parents to go through with this decision on their own. Unfortunately sometimes logic and reason cannot be dealt with by the people at the centre, which is why we have these services available. I hope that they will manage to find a way to move on and live as normal and happy life as they can. I hope they find inner strength to find peace that their son won’t be suffering anymore once it’s done and I hope against hope that they can forgive the people who made these decisions as it would have hurt them too…to the parents of little charlie if you read this I say this: .I’m so sorry that you have had to suffer like this and I hope that you don’t see my words and feel any pain anger or hurt from what I say. I will be thinking of you both and little charlie and I dont want you to think that anything that I’ve said is criticism on the way you’ve gone about things. On the contrary, I would have been doing the exact same if it were my child, I have every respect for you both. I am so very very sorry xxx 💙 charlie gard 💙

  • G W

    Unfortunately the child’s parents have become victims of the media circus, to the point of strangers who have no facts, except what is being fed by the media, campaigning to save a child who, sadly, has no chance of survival. His parents suggest he is stable, but as he cannot move or cry, what does ‘stable’ actually look like? As desperately sad this case is, this has become a battle of wills with the parents at the centre not really considering that their beloved child may actually prefer death to a life of stillness, brain damage and pain. I pray Charlie is out of his misery soon. This has gone on too long and no one should suffer just because of their parents neediness and inability to cope without them. Controversial? Yes. Do I have children? Yes. Would I listen to the advice of medical professionals? Absolutely.

  • Croiteir

    really – at that age – preferring to die? Would he have any concept of death?

  • G W

    I doubt it. I doubt he has concept of anything. It was meant an an analogy not a literal statement.

  • G W

    You miss the point. This is not about money. This is about life. Put yourself in that place, where you cannot see, hear, move, swallow, breathe unaided, not to mention the uncomfort, even pain felt. Could you cope with that as an adult? Why expect an infant with no concept or expectation to either? How many parents have to see their child deteriorate and die but act with dignity and don’t play to the media. The disgrace here is not the medical professionals or the courts. Its the parents who cannot see the woods for the trees.

  • lizmcneill

    If you develop a drug that only benefits 1000 people by making a common treatment too expensive for many thousands of people (eg $600 epipens that cost the NHS about a tenth of that), is it really a net benefit to humanity?

  • Starviking

    Do you have a reference for the 18 children whose lives have been saved by nucleoside treatments in the US?

  • George

    The incentive to make a drug to help 1,000 people is resulting in less investment on drugs that help a lot more. The profits are now in drugs for rare diseases.

    Almost a third of drugs for rare diseases now exceed US$1 billion in annual sales. The global orphan drugs market is expected to reach US$176 billion by 2020, and account for 19% of total branded prescription drug sales. Is that sustainable?

  • anglo-irish

    A bit strong to imply the parents are “a disgrace” – they seemed like good, decent, loving people in a terrible moral dilemma. I thought the courts made the correct and moral decision, though.

  • SeaanUiNeill

    I fully agree Granni. Anyone who has experienced such hard decisions will recognise just how difficult it is to decide the balance between the tremendous emotional pain and the pragmatic realities.

  • Granni Trixie

    I presume that investment in research into drugs treatments for rare conditions also generates knowledge applicable more broadly, a byproduct difficult to quantify.

  • lizmcneill

    Meanwhile the antibiotic resistance crisis is hurtling towards us but no new antibiotics have been developed for the last three decades because a drug patients take for a few weeks isn’t as profitable as a drug they take for the rest of their lives.

    Even if the return of fatal infections from minor wounds or childbirth doesn’t move Big Pharma, you’d think the collapse of the “market” for routine and elective surgery when any wound becomes Russian roulette with a fatal infection would.

  • Hawk

    Even if the price of drugs are $1 million per treatment it still ends up helping because drugs have to come off patent at some point.

    There are times when this is a choice between having an expensive drug where maybe only a few can afford it, with broader access when the patient ends, or having no drugs at all.

  • Hawk

    Are we assuming that the incentive to make broad use drugs are somehow diminished by having another ‘market’ for rare diseases? Are we assuming that the money used to develop drugs for rare diseases is being diverted from those other diseases which affects more people? On what basis have you made that judgement given that even R&D spend continues to increase year on year on most fronts? I don’t think that is obvious at all.

    You’re assumption that money is being diverted rests on the idea that there a fixed pot of money that gets divided up. Is there any case where providing a NEW service that is profitable will NOT attract NEW capital to produce that service? Having the incentive to develop profitable drugs in a new market increases the capital thrown at that market. Nowadays is you have a drug product which you may think will be beneficial you IPO a company on the stock exchange and those people looking to make a buck will take a punt on some of your shares. That’s new money.

    This market is sustainable for as long as people are willing to pay for those drugs. If individuals don’t find those drugs to be of any benefit they will stop using them, that’s typically how this works. The overall spend of the market tells us very little about sustainability, the individuals who require treatment make that decision independently from anyone else. Yours or my opinion of whether this is sustainable to not relevant to the actual sustainability of the market.

    There is also a moral argument here, if you don’t provide an incentive for individual to develop drugs for rare diseases you are condemning people to die. The big diseases like cancer and antibiotic resistance are awash with R$D funding, I think they can stand on their own two feet, because the market is enormous, the profits are enormous and the incentive to keep developing is enormous.

  • Hawk

    That’s a load of nonsense, any company who patents a drug has a large say in what the eventual price point with a corresponding impact on profitability. You think a company that develops a new antibiotic is going to going to accept a price where they make pennies?

    Part of the problem with antibiotics is that all the low hanging fruit as been picked, that’s why few products have came to market recently.

    Another reason for the delay is that until the last decade or so antibiotic resistance wasn’t a major problem, why bother developing an antibiotic when there were others proven to work.

    Necessity is the mother of invention. Large amounts of money is now being thrown at this problem because drugs have stopped working.

  • Croiteir

    You may doubt it – but you will never really know, will you?

  • Croiteir

    I agree – it isn’t. I wonder why the opening post mentioned it then?

  • Croiteir

    How do you think the parents are playing to the media?

  • Ricardo Gomes

    How about looking in the web?? There are plenty of information out there https://uploads.disquscdn.com/images/9c74023f2f27216ebb69f3ff9012961a528de1b76ec96fc5da6bf90ffe923952.jpg

  • Starviking

    You have some information with which to start looking, even if it comes from the Sun. Here, from the article:

    “Arturito has a similar mitochondrial depletion syndrome to Charlie and was the first person in the United States to receive the experimental and pioneering nucleoside therapy.”

    Arturito’s case is similar, not the same.

    If you spend a minute searching you find out that Arturito was diagnosed when he was 1. A little more digging reveals that Charlie Gard was diagnosed at 8 weeks – so his case is obviously more severe.

    So, you have provided some information on one case, which I have filled out for you. How’s about the other 17?

  • Ricardo Gomes

    I think you are intelligent enough to look it by your self just by searching I found 5 similar cases with google and on Charlie’s army Facebook page you will find another 2 one from Italy… Doctors are not God..when I was 1 year old I also had a desease. Doctors told my parents that i would die in couple of months and I was suffering… I cannot remember to suffer, right now I am absolutely normal, I’m 33 I have a beautiful daughter I have 3 master degrees in Ethic, Economics, computer Science…that alone proves me that doctors don’t know everything… infact they know very less. They have a profession like me, they should Do their job, and if they don’t understand a thing then they should pass the case to someone with more compentencies. They should Stop playing God and let parents decide. I was allowed in 1894 to be threaded without Quimiotherapy in Germany my country did not wanted me dead like UK wants Charlie, Alfie and HarryThomas dead.

  • Starviking

    Ricardo, you mentioned 18 cases. It is your responsibility to expand on that information, not mine. You say you found 5 similar cases with Google, and 2 on Facebook, does that mean you do not have 18 cases, only 8?

  • Ricardo Gomes

    Oh man, how can stupidity come so far? Tell me, please…

  • Starviking

    Ricardo, you claim to have 3 Masters Degrees, which is odd to me – most people go Batchelors, normally one, maybe two Masters, maybe a Doctorate. Still, perhaps in your country that is the way to go.

    Still, given your academic experience, and the fact that you your self claimed:

    “So in this article are you judging America which granted allready the life of 18 children with similar conditions like Charlie”

    you are unable to provide information on 18 children.

    If there is stupidity here, it is not coming from my side.

  • lizmcneill

    But then you end up with things like pharma companies being able to put up the price of a drug that’s been around since the 1950s by 5000% because there is no generic version.

  • Ricardo Gomes

    OMG, your competencies are by far lower then I expected… search friends, get a dog.