RugbyRebels

A very sad story, but one that has evoked plenty of discussion, opinions and others sticking their oar in.

It's just about impossible to know what his parents are going through. I expect many parents in the same situation would try their best to keep hope alive by seeking out alternative treatments as well as taking legal action, but I do wonder if some of the advice they have received has done more harm than good, especially for Charlie?

Any thoughts?

I feel that the parents of this poor child have been the victims of gross manipulation by church, politicians and commercial interests. It was abundantly clear to anyone who could raise themselves above the perfectly human response that the sliver of hope they were offered was abysmally false. They cannot be blamed for seizing upon it and holding on to it.

It can only be hoped, because no one will ever be able to know, that their efforts did not cause the child to suffer unnecessarily.

What is equally appaling about this tragic episode is that good people who devote themselves to easing the suffering of desperately sick children have become the target for a deluge of abuse and threats.

What's the skinny there?

Baby is born with genetic wasting illness. Doctors determine that the condition cannot be treated, is terminal and that 11 month old baby is suffering. Doctors determine that 'best' outcome is to withdraw life support.

Parents take doctors to court. Court rules with the doctors.

Then US research offers experimental treatment that provides some 'evidence' of improving similar conditions. Trump offers to pay to bring the child to US and church leaders, including the Vatican, offer support to parents.

Parents take the case to the high court. High court asks to see the 'evidence'. There isn't any. Parents have given up their fight.

The welter of publicity has set tempers a-boil and thousands of abusive messages have been posted against Great Ormond Street Hospital, including death threats against staff.

It's not just been hospital staff receiving threats, the parents have had what would be considered threats sent their way too. Utterly barking mad to be sending them to either 'side'

I see. That little boy has an intractable condition. That is the reality of it. It looks as though sending him to the US for the experimental treatment would have been just that....experimental. Patient advocacy is a wonderful thing, and especially powerful when driven by families and I hope the parents can direct their energy to supporting future effort via research now. We are learning more and more about mitochondria every year and at some stage, this understanding will merge with technology and translate clinically. But not right now unfortunately. Threats against hospital staff is just absurd.

Digby wrote:It's not just been hospital staff receiving threats, the parents have had what would be considered threats sent their way too. Utterly barking mad to be sending them to either 'side'

Agreed. There were also some abysmal cunts targetting random parents and visitors outside GOSH. It is fecking staggering that these social neanderthals think that there is a place in society for such displaced rage.

Googleland - where everyone is an expert.

morepork wrote:Patient advocacy is a wonderful thing, and especially powerful when driven by families ...

It is when it is informed, but I can't help but feel that in this case the parents were acting out of the most basic of instincts and against all of the informed opinion. That is not to say that I attach any blame to them; I do not. I do blame those who sought to make political or commercial mileage out of their incomprehensible suffering and in the process gave them false hope and perhaps led to a prolongation of the suffering of their child.

SerjeantWildgoose wrote:

morepork wrote:Patient advocacy is a wonderful thing, and especially powerful when driven by families ...

It is when it is informed, but I can't help but feel that in this case the parents were acting out of the most basic of instincts and against all of the informed opinion. That is not to say that I attach any blame to them; I do not. I do blame those who sought to make political or commercial mileage out of their incoprehensible suffering.

Blame is a strong word in this instance and I'm not sure how else to word it but the parents have a duty of care for their child whether that's to offer him the best chance or end his suffering.

What was the nature of commercial meddling in this case?

My wife's just back from a week in Barcelona at some world congress for cardiac medicine. She's obviously of the opinion the the doctors at gosh know what they're doing and should be listened to in terms of whats best for the baby. But there is a flip side to the coin... medical advances are often made by doctors/teams just trying things and seeing what works (educated guesswork). Clearly noone wants to treat patients simply as guinnea pigs, but if you dont allow some experimentation to take place then you're putting up a barrier to advancement.

This isnt really part of the Charlie Gard debate, the doctors who have been treating him his whole life and who know him best say he's suffering and nothing can stop that; they should be listened to. But it's always worth keeping in mind the knock on effects of well meant decisions.

As to the abuse directed at those involved in this case... a sorry indictment of modern times, just one more sign of the hysteria that threatens to envelop us.

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morepork wrote:What was the nature of commercial meddling in this case?

Theres some stuff detailed in the GOSH statement, here: http://www.gosh.nhs.uk/news/latest-pres ... -july-2017

It's pretty depressing stuff. The Prof from the US who said he could treat the patient, the guy whose testimony prolonged the appeals process for 6 months or so.... yeah so it turns out he hadn't met the patient, hadn't read the patient files and had financial incentives to offer the "treatment" he was offering which it turns out couldn't have worked. Paragraph 10 in the statement linked above. About as horrible as it gets.

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What I found a little distasteful today was the US doctor saying it was too late to use his treatment on Charlie. Is it me or is he implying he could have helped the boy had he been allowed to earlier? Is this yet another unhelpful comment aimed at the GOSH specialists who have already done the absolute best for the boy?

As I understood things, the nucleoside therapy has no data supporting it being effective with the type of mitochondrial DNA depletion syndrome that Charlie has. If that is the case, surely the doctor would've been better off just saying that he is unable to help the boy.

Donny osmond wrote:

morepork wrote:What was the nature of commercial meddling in this case?

Theres some stuff detailed in the GOSH statement, here: http://www.gosh.nhs.uk/news/latest-pres ... -july-2017

It's pretty depressing stuff. The Prof from the US who said he could treat the patient, the guy whose testimony prolonged the appeals process for 6 months or so.... yeah so it turns out he hadn't met the patient, hadn't read the patient files and had financial incentives to offer the "treatment" he was offering which it turns out couldn't have worked. Paragraph 10 in the statement linked above. About as horrible as it gets.

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He is one of the inventors of a patent for NBT, which was filed by Columbia University last year. I don't know who, if anyone, has the license for the technology, but he did not stand to directly benefit financially from the experimental treatment. That's not how it works. It does however look like he mouthed off far too quickly regarding the potential of the therapy for the patient in question.

As for the death threats, the parents certainly didn't help matters by accusing the hospital of lying and making other inflammatory statements; as if one of the leading children's hospitals in the world suddenly decided to kill their child.

WaspInWales wrote:What I found a little distasteful today was the US doctor saying it was too late to use his treatment on Charlie. Is it me or is he implying he could have helped the boy had he been allowed to earlier? Is this yet another unhelpful comment aimed at the GOSH specialists who have already done the absolute best for the boy?

As I understood things, the nucleoside therapy has no data supporting it being effective with the type of mitochondrial DNA depletion syndrome that Charlie has. If that is the case, surely the doctor would've been better off just saying that he is unable to help the boy.

Having read the position statement, if it was too late that was entirely down to him not accepting the invitation to visit from the hospital until 6 months after it had been given .

Eugene Wrayburn wrote:As for the death threats, the parents certainly didn't help matters by accusing the hospital of lying and making other inflammatory statements; as if one of the leading children's hospitals in the world suddenly decided to kill their child.

The tantrums in court haven't helped matters but have been perfect for setting an 'us V them' mentality that have stoked the flames on social media.

Then again, if the parents have been fed information from 'trusted' sources that there are viable alternatives, then their reaction is at least understandable to some extent.

SerjeantWildgoose wrote:

morepork wrote:Patient advocacy is a wonderful thing, and especially powerful when driven by families ...

It is when it is informed, but I can't help but feel that in this case the parents were acting out of the most basic of instincts and against all of the informed opinion. That is not to say that I attach any blame to them; I do not. I do blame those who sought to make political or commercial mileage out of their incomprehensible suffering and in the process gave them false hope and perhaps led to a prolongation of the suffering of their child.

My missus and I were talking about this issue earlier. We both agree, with our 14 month old son, that we would do anything to look after him and , heaven forbid, that we find ourselves in the same position as Charlie Gards parents, we would also be desperate for a solution.

The problem with experimental cures is that they often aren't widely known or have low likelihood of success. Sometimes the kindest thing to do is let nature take its course rather than prolong life artificially, and anyone who tries to manipulate the parent switch false hope is despicable. I do recall a case from a year or two ago where parent s took their child abroad to get treatment denied to them in the NHS and managed to get their son cured. In that case, the authorities looked high handed and just plain wrong, but that perception can't overshadow every case.

A horrible position to be in and a difficult one also for doctors and the judges involved.

As for abuse via social media, it's a total disgrace. I've just been reading a report about MPs getting death threats via social media and it's something that needs to be dealt with. We wouldn't tolerate a face to face death threat, the pathetic cowards who do so via social media deserve punishment.

WaspInWales wrote:What I found a little distasteful today was the US doctor saying it was too late to use his treatment on Charlie. Is it me or is he implying he could have helped the boy had he been allowed to earlier? Is this yet another unhelpful comment aimed at the GOSH specialists who have already done the absolute best for the boy?

As I understood things, the nucleoside therapy has no data supporting it being effective with the type of mitochondrial DNA depletion syndrome that Charlie has. If that is the case, surely the doctor would've been better off just saying that he is unable to help the boy.

Thatbwas the implication I picked up as well. Really helpful.

With really rare diseases such as this, there is (or should be) a systematic recruitment process for experimental therapy. When I say experimental, I mean the early, formal stages of therapeutic development as part of a clinical trial. This means a comprehensive patient data base, and easy access to information regarding recruitment criteria for patient families. With a disease as rare as this, it is unlikely that an industry entity will pick it up and run with it (although they often do if the technology has broader potential applicability) but there will be government funding to support the applicability of these sorts of therapies. More specifically there is a "compassionate use" mechanism that can be fast tracked for individual patients that circumvents FDA approval (very high risk/benefit ratio). Safety is the primary and paramount initial criteria. In the US, the NIH has specific funding mechanisms available for situations like this that will support clinical trials for orphan (rare) diseases, and Dr. Big Trousers should have been part of an effort that has a formal conduit for diagnosis, recruitment criteria (what forms of disease are actually amenable to treatment) and definition of expected outcome measures. Administration of the therapy is oral, and the active agent is non-biologic (not a virus, antibody) and should have a very well defined safety profile as it consists simply of analogues of DNA that are used by mitochondria to repair/synthesise their genome. When looking up this stuff, all hits were from the Sun, Fox News and other bastions of informed science. What these parents should have been exposed to is networks for patient recruitment that were not anecdotal, if they in fact exist. They have been completely fucked around by social media misinformation, and it is the obligation of physicians and scientists to direct these people away from that bullshit.

For anyone interested, this is the venerable 'murrican neurologists "new insight" into the treatment. Note, firstly, the availability of a fairly limited model system of disease, but secondly, how simple administration of the treatment is. I ask Professor wotshisname, what the fuck are doing to get this into the clinic properly sir?

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4154130/

I really hope this ends soon. The current legal debate on where the poor lad is going to die is not helping anyone. It seems like the parents are trying to keep their son alive for as long as possible. I understand that completely; but on the other hand reality needs to be faced soon.

Having all this played out in the courts and the media is helping no one.

Sandydragon wrote:
Having all this played out in the courts and the media is helping no one.

It's what we have in the event a hospital staff and family can't agree. And no it's not helpful, especially given some other child might well be able to make use of a bed in the outstanding GOSH

Digby wrote:outstanding GOSH

This. They saved my brothers life.

I can't help but think their time and energy would be now best spent with their son rather than in court. By 'now' I mean quite a few days ago.

Mellsblue wrote:

Digby wrote:outstanding GOSH

This. They saved my brothers life.

I can't help but think their time and energy would be now best spent with their son rather than in court. By 'now' I mean quite a few days ago.

Indeed. Once it became apparent that there was no way to save his life, I'd want to spend every second with my child, not sat in court.

At the risk of sounding harsh, looking at the amount of support they require to take their son home, would those resources not be better used in a hospital treating a number of kids?