Charlie Gard

[scrubchub]

A truly heartbreaking case with a tragic, though inevitable, outcome. Poor little kid. It seems strange to be saying it, but this is clearly the kindest outcome. Chris Gard's statement was very hard to watch, full credit to him for being composed enough to deliver it. I don't think I would be.

Such a shame that it has been hijacked and turned into such a dreadfully undignified spectacle by a particularly repugnant group of arrogant, over entitled, social media empowered idiots. "Charlie's Army" should be ashamed of themselves and the way that certain followers of their cause has behaved. Even today, there have been protestors outside court shouting "Shame on the Judge!" and "Shame on GOSH!". Absolutely despicable, and as it comes from such a wilful place of wallowing ignorance, absolutely unforgivable. Not that hubris of the level that these people have shown is ever followed by contrition in any case.

I looked through the Charlie's Army Facebook page, and one of the first comments states that this is inhuman and "Worse than Hitler." Another says that this "Takes us back to a pre Christian time for morals." Whatever that means.

The Professor's sudden interest after being so dis-interested earlier in the case (before certain high profile interventions) always smacked of snake oil salesman. It's hard to feel as though this hasn't confirmed that. The statement from GOSH is borderline incendiary. Will be interesting to see if or how he can reply to it.

My mother in law is a fully fledged member of Charlie's Army, and the other day made an off the cuff remark that America should bomb Britain if Charlie wasn't allowed to leave GOSH. She then asked my position. I merely said that I tend to defer to the experts on the ground as opposed to the ones on the internet.

One thing that I don't fully understand - Chris Gard has said now that if treament was given sooner, then Charlie's could have recoverd - in what sense is this wrong? Again, I defer to the experts (Wiccan we salute you) - is there any truth in this?

Genuinely upset watching Chris Gard read that statement. I can't personally be critical of the parents in this episode - I can't and don't want to imagine what they have gone through (and what they will go through in immediate future). Will go and give my boy - born a week before Charlie - an extra kiss on the forehead.

[andoverben]

This whole episode has been truly heartbreaking. There was never going to be any winners in it and while it is easy to point fingers, like any Parent I suspect that in a similar circumstance I would fight tooth and nail for what I felt was the best treatment for my children.

By the sounds of it they have been led on a bit by the Dr from the US who has given them hope which understandably they would want to cling to. That hope evaporated today when it became apparent he wasn't the expert they hoped he was.

Its all just desperately sad - and a shame that people want to hijack it for politics.

[FN2TypeR]

scrubchub said:

My mother in law is a fully fledged member of Charlie's Army, and the other day made an off the cuff remark that America should bomb Britain if Charlie wasn't allowed to leave GOSH.

[Wiccan of Darkness]

FN2TypeR said:

scrubchub said:

My mother in law is a fully fledged member of Charlie's Army, and the other day made an off the cuff remark that America should bomb Britain if Charlie wasn't allowed to leave GOSH.

You should show her this thread. Lets see if she argues with what I say.

In response to your question, the treatment would not have reversed any damage already done, it was given a 10% chance of 'success'. In this instance, success would have been a cessation in deterioration during the treatment. To be quite blunt, this treatment would have not been life saving. It would have been death delaying. I'm just watching 999 what's your emergency, ruminating on my evening meal before I post a response to the days events in court.

[KTF]

The Facebook page is all over the 'had he got the treatment earlier he would be normal' comment by the Dad. It's also the one being used by the media.

A real shame as it just isn't true.

[sugerbear]

"Further, GOSH was concerned to hear the Professor state, for the first time, whilst in the witness box, that he retains a financial interest in some of the NBT compounds he proposed prescribing for Charlie. Devastatingly, the information obtained since 13 July gives no cause for optimism. Rather, it confirms that whilst NBT may well assist others in the future, it cannot and could not have assisted Charlie."

[Vizsla]

scrubchub said:

My mother in law is a fully fledged member of Charlie's Army, and the other day made an off the cuff remark that America should bomb Britain if Charlie wasn't allowed to leave GOSH.

I'm sorry to have to point out to you that your MIL is a fking cretin (but then you probably already know that).

[llewop]

Wiccan of Darkness said:

the treatment would not have reversed any damage already done, it was given a 10% chance of 'success'. In this instance, success would have been a cessation in deterioration during the treatment. To be quite blunt, this treatment would have not been life saving. It would have been death delaying. I'm just watching 999 what's your emergency, ruminating on my evening meal before I post a response to the days events in court.

A question Wiccy, if you are able to comment; do you think it is possible this sorry, drawn out, torture for all involved could affect the research, treatment and perception on rare diseases?

It does bother me that it could make experimental treatments of rare conditions more challenging to progress. Clearly this case is at the very extreme of rarity, but others that are slightly less rare may struggle to develop and improve treatments, particularly those that are at best, as above, delaying progress, but not able to cure. (Vested interest- I have a rare, progressive condition, UK diagnosed population only a couple of hundred.)

[danllama]

The Professor sounds like a criminal.

[scrubchub]

Vizsla said:

I'm sorry to have to point out to you that your MIL is a fking cretin (but then you probably already know that).

She certainly has her moments.

Thanks for the clarification Wiccan.

What will the Professor do now? Will he come out guns blazing in his own defense or will he lay low and keep his mouth shut? He has willingly placed himself front and centre in an international media st storm. I imagine him sleeping rather fitfully tonight.

Reading around on the internet at the stories and responses relating to this and it is a little worrying where it is going. There is a very strong rhetoric along the "if only it was done sooner then he would be cured" line. And very much painting the hospital and the courts as the bad guys. I worry that this morally reprehensible vilification of GOSH is going to get worse. The arrogance of the protestors is astounding, and they seem emboldened.

Hope it doesn't boil over in any way.

Edited by scrubchub on Monday 24th July 23:40

[Wiccan of Darkness]

GOSH has released several position statements which can be found on their media page (I did have the link but lost it). I have summarised the sorry state of affairs below. There's already information elsewhere in this thread, so I won't go in to too much detail but will briefly touch on the progress so far.

• Charlie had an incurable condition that caused catastrophic damage to his mitochondria.
• There was never any cure or active treatment. He was always going to die. Full explanation of the condition is in my previous posts
• Contrary to current medical guidelines, Charlies parents felt that they had the last word on treatment. This was not possible under the law as it stands. All hospitals, including GOSH have a primary duty of care towards their patient first and foremost. This is outlined in the GOSH position statement on 13th July.
• 3 independent opinions came to the same conclusion, that in Charlies case, nucleoside treatment was not in his best interests.
• In Charlies case particularly, nucleoside treatment would have had no meaningful benefit whatsoever, irrespective of when it commenced. Reports of other children suffering mitochondrial disease showing improvement with nucleoside therapy is correct but they suffered from a different form(TK2); one where nucleoside therapy was beneficial. Charlie has RRM2B
• Charlies parents instructed their own expert back in April. He too came to the same conclusion that Charlies prognosis was 'bleak'. The parents rejected his opinion and sought expert opinion that reinforced their false hopes.

• No tests have been conducted on mice with RRM2B form of the disease, as such the proposed 'therapy' was completely untried, untested and unlicensed. The law prohibits administration of untested therapies irrespective of informed or perceived consent; it simply cannot be done.
• Professor Hirano is one such expert in mitochondrial disorders. He was contacted by GOSH for an expert opinion back in december and invited over for a consultation. That offer was not taken up until July 18th, at the bequest of the court.
• Professor Hirano provided extensive written and oral testimony for the courts at the hearings. After the final judgement that nucleoside therapy was not in Charlies best interests, Professor Hirano continued to supply evidence, to the contrary of the view of the court.
• Hirano then claimed 'new evidence' on 6th July, and GOSH went back to court to hear the novel development. It transpired there was no new 'research'
• On july 13th, Hirano confirmed he had not seen Charlie nor seen or read his medical notes. Whilst various claims to the efficacy of the treatment can be professionally made after patient reviews, in this case the patient had not been reviewed by Hirano and therefore, the claim could not be made.
• All the other expert opinions in Charlies case had been made after lengthy study of both Charlie and his medical notes, including diagnostic reports. Hirano, the only person NOT to have made that effort, was the only person who was making the claim the treatment would be beneficial.
• Hirano then further compounded the conflict of interests by not only admitting he had failed to review the patient and his notes, but that he actually had a financial vested interest in the therapy being offered. One can only speculate as to what proportion of the £1.3 million fund Hirano stood to gain personally.
• As well as personally having a financial vested interest in providing the therapy, the use of unlicensed and untested therapies in the UK is prohibited; Hirano would have had to administer the treatment within the US. Whilst completely outlawed in the UK, the US has a clause known as the compassionate use exception, where an untested or unlicensed drug, therapy or treatment could be used as a last resort.
• Under the compassionate use exception clause, approval would have to be granted by the Federal Drug Administration authorising the first ever use of the nucleoside therapy.
• To date, Hirano has not made any FDA application. It is therefore unclear as to what therapy Hirano was in fact proposing, and under what jurisdiction.
• GOSH acted under the principle that novel therapies are best provided in the context of formal clinical trials. The hospital does not treat its most vulnerable children simply because it can and on no account does it treat them purely because novel treatment furthers GOSH’s research.
• The same cannot be said for Hirano, who having opined the novel nucleoside therapy in which he had a pecuniary interest, had then failed to make the relevant FDA application for an immediate start to the therapy, were he successful
• The final act of Hirano's self interest lay with the lack of a long term plan once the £1.3 million fund had been depleted. From the lack of evidence, one can only assume the therapy would only be provided whilst the parents were in funds. Furthermore, no plans for repatriation to the UK were apparent, once said funds were depleted.
• GOSH released in todays position statement the following.
  GOSH said:

  It is hoped that those who, like the Professor, have provided the opinions that have so sustained Charlie’s parents, their hopes and thus this protracted litigation with its many consequences, will also find much upon which to reflect.

Clearly, peddlers of snake oil and false promises whose vested interests are in the financial procurement of a £1.3 million fighting fund only serve to prolong the intense suffering of terminally ill patients.

It is therefore my belief that, considering all the evidence available, Professor Hirano is nothing more than a money grabbing bd who having not made any FDA applications, probably had no intention of ever providing the worthless treatment once the funds had been released to his own personal bank account. Furthermore, as Hirano had neglected to bother reviewing Charlies medical notes, visit the patient or even review any of the previous judgements, as well as make any FDA applications, it cannot be said that the FDA would even have approved the therapy, had an application been made.

This then creates the next question. What were Hirano's intentions, had the FDA turned down the application to provide the therapy for a living patient?