The Charlie Gard case

[Pea-n-Me]

In all the references I've seen, I thought the Wiki article in the OP captured the complexities of the case best, in a way that's understandable. (I have seen more complex articles as well.)

I added this to the OP, What is Nucleosides Therapy for MDDS, or Mitochondrial DNA Depletion Syndrome

https://mitochondrialdiseasenews.co...ned-why-doctors-debate-treating-charlie-gard/

 

[pryncess527]

I found evidence that Dr. Michio Hirano of Columbia University Medical Center believes there is a chance for improvement. Could you point to links for the other six? I tried, but didn't find anything.

Has there been a statement from Dr. Hirano or the boy's parents in the last few days? I know Dr. Hirano was in London on Monday & Tuesday to examine the boy, and at the time it was reported that if Dr. Hirano thought the treatment would be successful, he'd be filing a request with the FDA -- but I have not found any indication that such a request was filed. And Charlie's parents seem to be staying quiet about the content of their meeting with Dr. Hirano. It made me wonder what the doctor's opinion was now that he's actually seen the patient and had full access to the medical records

 

[wilkeliza]

I found evidence that Dr. Michio Hirano of Columbia University Medical Center believes there is a chance for improvement. Could you point to links for the other six? I tried, but didn't find anything.

To add to that I think some of the lawmakers who have gotten involved on our side aren't realizing that their miracle children are not the same as Charlie's case. The one I read about the little girl who survived with Potters' Syndrome and thus her parents believe Charlie can survive the same way aren't putting it into perspective. A kidney can be transplanted if one is available a brain as well as several other damaged organs can not.

As another poster pointed out I think the part people struggle with is we don't like to believe terrible awful things happen to the littlest of children and that there is nothing we can do about it. Coming to terms with that is very hard.

 

[piccolopat]

I'm not convinced that this new treatment will help Charlie but I am sure he will die if nothing else is done. There are plenty of times where a patient has to endure ongoing pain during treatment but they do so knowing there is a chance of recovering, no matter how small. Think of all the people with terminal cancer that undergo surgery, chemo and radiation multiple times. Do we deny them the slim possibility of beating the disease? Do we deny Charlie a chance at life just because no one has survived long with his condition? Isn't that what experimental treatments are trying to change?

I'm also not sure I understand why he has to travel for this new treatment. As far as I read, it is done orally so why can't the medical team be contracted to treat him where he is now?

I do believe that the parents should have the right to decide what is best for their son in this case. I also believe the UK health care system can opt to not cover the experimental treatment but it sounds like money has been raised to take care of this.

 

[Kingdome8]

It reminds me a bit of the Jahi McMath case. Parents, struggling to come to terms with the worst possible news abotu a child, clutching at false hpoe provided by a couple of random doctors in spite of multiple, more involved doctors all reaching the same conclusions.

I cannot imagine the horrible agony of knowing you will lose a child and nothing can really be done for them. My heart breaks for the parents and the medical providers who are trying to do right by the child in this.

I find it odd and overreach that our government is involved in any way--and particularly to provide permanent residency to teh child and his family with apparently little to no vetting, etc. Do the parents still get that if/when the child dies? Would we do this for a dying refugee child from a mid east nation? I am uncomfortable with that overreach.

Since the parents have the funds to pay why not give it a shot. Why should govt have control over who lives or dies.

And middle easterners govt pay for patients to come here for treatment at Hopkins. Which is awesome. And the USA gives them temp visas to do so.

Everyone deserves a chance to live especially if they are paying them selves.

 

[pryncess527]

I'm not convinced that this new treatment will help Charlie but I am sure he will die if nothing else is done. There are plenty of times where a patient has to endure ongoing pain during treatment but they do so knowing there is a chance of recovering, no matter how small. Think of all the people with terminal cancer that undergo surgery, chemo and radiation multiple times. Do we deny them the slim possibility of beating the disease? Do we deny Charlie a chance at life just because no one has survived long with his condition? Isn't that what experimental treatments are trying to change?

I'm also not sure I understand why he has to travel for this new treatment. As far as I read, it is done orally so why can't the medical team be contracted to treat him where he is now?

I do believe that the parents should have the right to decide what is best for their son in this case. I also believe the UK health care system can opt to not cover the experimental treatment but it sounds like money has been raised to take care of this.

No, but those people have a voice, they get to choose to go through that. Many make the choice not to go through that.
Charlie can't make his own choice, and the options from here on out subject him to intense pain. That's why the courts got involved

NO ONE is saying he can be cured. Only that the pain/suffering can be mitigated through treatment, and even that seems to be highly questionable.

 

[Pea-n-Me]

This case isn't the same as the resuscitation of a functional adult. Not even close. (Although as a cardiac nurse I fully appreciate where a pp was coming from with that, i.e. low odds to overcome.)

This is a baby born without the capability at the cellular level of sustaining life. He is, at present, being kept alive artificially. If he were to be taken off life support, he would die, as his body cannot sustain life. Additionally, his seizures have pointed to his brain function deteriorating from what it was when he was born. Brain scans have been done, likely when he was born, before and after the seizures, and now, etc. Doctors at GOSH in England have been willing to try experiemental therapies which would possibly help his cells function, but there were lots of unknowns because the type of MDDS Charlie has is exceedingly rare, with only 16 cases known, and the experimental treatment shown to have some promise was only used on children with a different type of MDDS than Charlie has. So the experimental treatment is questionable, at best. Additionally, the seizures Charlie had (?has) highlights the fact that even if the experimental therapy helps, which is a long shot, his neurological status is still ominous. Doctors' arguments have not been financial, they've been humanistic; they feel Charlie is suffering, as is. Now the parents and some in the U.S. are fighting to have him medically transported over to the U.S. where his care will be taken over by Dr. Hirano and others. Doctors and England say that even if the experiemental treatment helps, and that's a big unknown, say, as to what degree, if any, they believe that Charlie will always be ventilator dependent and require round the clock care, for life.

It does beg the question of what quality of life Charlie will have, but the difference between England and the U.S. is that in England, courts can decide, and in the U.S., parents get to make the decisions.

 

[pryncess527]

Doctors' arguments have not been financial, they've been humanistic; they feel Charlie is suffering, as is..

This bears repeating. This is not about money. This is about the government stepping in to protect a minor child who can not make his own medical choices, from decisions that, in the unanimous opinion of doctors at the hospital treating him believe are harmful/painful

I understand why people disagree, but I think many are missing the crux of the issue here.

I'm very interested to know what Dr. Hirano's opinion is now that he's actually been able to examine Charlie.

 

[CarolAnn856]

Has there been a statement from Dr. Hirano or the boy's parents in the last few days? I know Dr. Hirano was in London on Monday & Tuesday to examine the boy, and at the time it was reported that if Dr. Hirano thought the treatment would be successful, he'd be filing a request with the FDA -- but I have not found any indication that such a request was filed. And Charlie's parents seem to be staying quiet about the content of their meeting with Dr. Hirano. It made me wonder what the doctor's opinion was now that he's actually seen the patient and had full access to the medical records

I haven't seen anything at all coming out of his meeting and examination in the UK. That is one reason I questioned the poster who claimed that 7 specialists saw a chance at improvement.

 

[Magpie]

Since the parents have the funds to pay why not give it a shot. Why should govt have control over who lives or dies.

And middle easterners govt pay for patients to come here for treatment at Hopkins. Which is awesome. And the USA gives them temp visas to do so.

Everyone deserves a chance to live especially if they are paying them selves.

You know, initially I felt this way, too.

But, upon reading further, I am not okay with putting an innocent non-consenting person though needless agony, just to keep "hope" alive for the parents. There is something truly horrifying about keeping a person alive on machines, unconscious, with deteriorating organs (including their brain), suffering seizures, and unable to communicate any desires or opinions about their quality of life.

Charlie cannot speak for himself. His doctors do not feel that his parents have his best interests in mind. Their only desire is to spare him further suffering. I'll be very interested to see what comes of the Friday court hearing, when all the tests are finally in.

Death is always sad, but having just lost my beloved mother-in-law to cancer, I can definitely say that there comes a time when you just need to stop fighting and embrace the inevitable. My mother-in-law knew that, and fortunately was able to tell us so, deciding for herself that she'd reached the end of chasing after different treatments. I don't want my family to force me to linger like Charlie Gard. And I'm so very glad we didn't do that to our mum. Instead of fear and confusion and hospitalization, she spent the last few days of her life in her bed, hallucinating that several long dead loved ones had returned to her. It was... oddly comforting for us all. I do like to think that she died, somehow, in the arms of her beloved husband.

 

[Pea-n-Me]

This bears repeating. This is not about money. This is about the government stepping in to protect a minor child who can not make his own medical choices, from decisions that, in the unanimous opinion of doctors at the hospital treating him believe are harmful/painful

I understand why people disagree, but I think many are missing the crux of the issue here.

I'm very interested to know what Dr. Hirano's opinion is now that he's actually been able to examine Charlie.

Yes, it's interesting. Dr. Hirano is in a very difficult position. He is the doctor who developed this experimental treatment (for which I added a link above, and in the OP, if anyone wants to read about it). He went over to England to examine Charlie and look through his actual medical data, talk to the parents and the caregivers, review all the scans and other data, etc. There is always the chance that he'll say that he feels he can't help Charlie, then they're back to square one. Or, if he thinks he can, they'll go forward (if the English courts allow), and all eyes will be on the situation for years to come. What if the treatment is ineffective? With the world watching? Will that harm Dr. Hirano's ability to continue research, thereby hindering his helping many others besides this one patient? Will research money dwindle? (Or maybe it will increase, idk.) Will he feel he let the parents down (and the Pope, the President, Congress, all those hopeful for a "cure")? It's a lot of pressure on him. My guess would be that if he thinks the situation is futile, he might just end it there by saying he wishes he could, but in good faith, he can't, although I'm sure it would be a very difficult thing for him to do, especially with the whole world watching. I wouldn't want to be him right now. (And saying prayers for all involved, including Dr. Hirano, that he will have the wisdom and clarity to make the right decision, whatever he thinks that is.)

 

[Pea-n-Me]

Just came across this excellent article by a UK pediatrician re some of the complexities involved in this case:

http://www.independent.co.uk/Voices...-life-support-pope-donald-trump-a7827276.html

 

[wilkeliza]

Yes, it's interesting. Dr. Hirano is in a very difficult position. He is the doctor who developed this experimental treatment (for which I added a link above, and in the OP, if anyone wants to read about it). He went over to England to examine Charlie and look through his actual medical data, talk to the parents and the caregivers, review all the scans and other data, etc. There is always the chance that he'll say that he feels he can't help Charlie, then they're back to square one. Or, if he thinks he can, they'll go forward (if the English courts allow), and all eyes will be on the situation for years to come. What if the treatment is ineffective? With the world watching? Will that harm Dr. Hirano's ability to continue research, thereby hindering his helping many others besides this one patient? Will research money dwindle? (Or maybe it will increase, idk.) Will he feel he let the parents down (and the Pope, the President, Congress, all those hopeful for a "cure"? It's a lot of pressure on him. My guess would be that if he thinks the situation is futile, he might just end it there by saying he wishes he could, but in good faith, he can't, although I'm sure it would be a very difficult thing for him to do, especially with the whole world watching. I wouldn't want to be him right now. (And saying prayers for all involved, including Dr. Hirano, that he will have the wisdom and clarity to make the right decision, whatever he thinks that is.)

Yes, it's interesting. Dr. Hirano is in a very difficult position. He is the doctor who developed this experimental treatment (for which I added a link above, and in the OP, if anyone wants to read about it). He went over to England to examine Charlie and look through his actual medical data, talk to the parents and the caregivers, review all the scans and other data, etc. There is always the chance that he'll say that he feels he can't help Charlie, then they're back to square one. Or, if he thinks he can, they'll go forward (if the English courts allow), and all eyes will be on the situation for years to come. What if the treatment is ineffective? With the world watching? Will that harm Dr. Hirano's ability to continue research, thereby hindering his helping many others besides this one patient? Will research money dwindle? (Or maybe it will increase, idk.) Will he feel he let the parents down (and the Pope, the President, Congress, all those hopeful for a "cure")? It's a lot of pressure on him. My guess would be that if he thinks the situation is futile, he might just end it there by saying he wishes he could, but in good faith, he can't, although I'm sure it would be a very difficult thing for him to do, especially with the whole world watching. I wouldn't want to be him right now. (And saying prayers for all involved, including Dr. Hirano, that he will have the wisdom and clarity to make the right decision, whatever he thinks that is.)

I don't think it will affect the trial he is trying to get or his funding as all of that is for a different mutation that causes a mitochondrial disorder. In Charlie's case no studies not even in mice have been done for his mutation so this is a true hail mary. It is very promising in the most common type and is possible that if caught early enough it could stop a lot of the damage that happens but it has to be caught early.

 

[Pea-n-Me]

I don't think it will affect the trial he is trying to get or his funding as all of that is for a different mutation that causes a mitochondrial disorder. In Charlie's case no studies not even in mice have been done for his mutation so this is a true hail mary. It is very promising in the most common type and is possible that if caught early enough it could stop a lot of the damage that happens but it has to be caught early.

What I was trying to say, probably badly, was that it's possible this could all have a negative effect on his career, and research.

 

[wilkeliza]

What I was trying to say, probably badly, was that it's possible this could all have a negative effect on his career, and research.

That's what I took from your post I was just saying I don't think it will because the other research he has done is on a different mutation. Most scientific research doesn't fault someone for a bad study as long as it is performed properly. Now if he just gave Charlie the meds with out approval or seeing him then perhaps it would but he isn't doing that.

 

[Pea-n-Me]

That's what I took from your post I was just saying I don't think it will because the other research he has done is on a different mutation. Most scientific research doesn't fault someone for a bad study as long as it is performed properly. Now if he just gave Charlie the meds with out approval or seeing him then perhaps it would but he isn't doing that.

Oh, I get what you're saying, but I'm not talking about the specifics of his research, persay. My thought is just that this has become an international "event" and the whole world is watching to see what happens. If it goes badly, it could have a potentially negative impact on all of his research. JMO, and just one thing among many that I'm betting he's thinking about.

 

[larissawbb]

I haven't seen anything at all coming out of his meeting and examination in the UK. That is one reason I questioned the poster who claimed that 7 specialists saw a chance at improvement.

I don't offend that easily. I'm at work right now but did find a brief statement on BBC news. I have read it on several other websites too. When I get home , I will look it up. The statements that I read was the seven other specislists from the UK, Italy, Spain and the USA implored GOSH to consider new research. I just don't think anyone should draw conclusions until the medical reports come out next week. The reason we haven't heard anything is because the findings can't be commented upon by the parents or physicians since it's a judicial matter. http://www.bbc.com/news/uk-england-london-40535043

 

[DaisyJ]

How much is a life worth?

He doesn't have a life and never will.

 

[CarolAnn856]

What I was trying to say, probably badly, was that it's possible this could all have a negative effect on his career, and research.

I really doubt it. If they get the emergency, compassionate-care, approval from the FDA, it will be with everyone (except, perhaps, the parents) knowing full well that this is a long shot. If anything, I would think that even an emergency approval could stand to improve even further his standing in that field.

 

[Pea-n-Me]

I really doubt it. If they get the emergency, compassionate-care, approval from the FDA, it will be with everyone (except, perhaps, the parents) knowing full well that this is a long shot. If anything, I would think that even an emergency approval could stand to improve even further his standing in that field.

As I said. Yes, it could make his work more well known and perhaps this will be a good thing for him and his research. But there could also be a negative effect, as this has become an international incident as well. Not everyone is on board. We'll have to see how it plays out. I suspect Dr Hirano is the type of guy who is on the up and up, so hopefully his career will not suffer because of this. No doubt, he is trying to help, if he can.