Is experimental treatment for Charlie Gard worth it?
Charlie’s parents had been seeking experimental treatment that might improve his rare genetic condition. They had raised funds for him to travel to the US, where a medical specialist had offered to provide treatment. Many observers could not understand why the doctors at London’s Great Ormond Street Hospital opposed the requested treatment.
What can we learn from Charlie Gard’s case?
Jul 20, 2017 · The experimental therapy that has been suggested for Charlie would be an oral medicine that aims to deliver the correct type of nucleoside to improve the function of his mitochondrial DNA. The medication has been tested on mice and a small number of people with a different mitochondrial condition, some of whom have shown measurable improvement.
When did the hospital agree to admit Charlie Gard?
Jul 17, 2017 · the treatment he is developing, called “nucleoside bypass therapy,” is so experimental that it is not being tested in any clinical trial in the u.s., has never been tried in anyone with charlie’s...
What is Charlie Gard's condition?
Jul 14, 2017 · When the Great Ormond Street Hospital for Children NHS Foundation Trust, the hospital where Charlie was being cared for, refused to provide additional treatment, the Gard’s filed a lawsuit to allow their son to be taken to the United States for an experimental procedure called nucleoside therapy.
What gene mutation did Charlie Gard have?
How does nucleoside bypass therapy work?
What happened with Charlie Gard?
Is Charlie Gard still alive?
Why was Charlie Gard denied treatment?
What Happened to Baby Charly?
Is tafida raqeeb still alive?
Who started Charlies law?
What disease did Charlie Gard have?
Did Baby Charlie pass away?
What is Charlie's experimental therapy?
The experimental therapy that has been suggested for Charlie would be an oral medicine that aims to deliver the correct type of nucleoside to improve the function of his mitochondrial DNA.
What is Charlie's condition?
Charlie suffers from a form of mitochondrial depletion syndrome, a rare genetic disease that causes progressively increasing muscle weakness that leads to organ failure and becomes life-threatening within a few years. Though he is less than a year old, the baby has been on life support for several months.
Why is Charlie Gard on life support?
Michio Hirano, a neurologist at Columbia University, will evaluate 11-month old Charlie Gard, the British boy who is on life support because of a rare, lethal disease called mitochondrial depletion syndrome. Charlie is at the center of a fraught legal case in the U.K. concerning whether his parents can take him abroad ...
What is Charlie's mutation?
Charlie has a mutation in a gene called RRM2B, which codes for the production of an enzyme that helps produce the four “letters” (nucleotides) that constitute DNA in mitochondria.
Can Charlie's parents take him abroad?
Charlie is at the center of a fraught legal case in the U.K. concerning whether his parents can take him abroad to receive experimental treatment. Doctors at the hospital treating Charlie have said that every medical treatment option has already been explored, and have advised against transferring him abroad.
Can Charlie be treated in New York City?
Under a “compassionate use” exemption from the U.S. Food and Drug Administration, a program that allows patients who are otherwise out of options to receive experimental drugs that have not been approved by the FDA, he could receive the therapy in New York City. (Columbia said on July 6 that it is willing to treat Charlie.) It doesn’t involve any red tape: The FDA typically OKs such use in a single phone call with the scientist.
What is Charlie Gard's case?
The Charlie Gard case was a best interests case in 2017 involving Charles Matthew William "Charlie" Gard (4 August 2016 – 28 July 2017), an infant boy from London, born with mitochondrial DNA depletion syndrome (MDDS), a rare genetic disorder that causes progressive brain damage and muscle failure. MDDS has no treatment and usually causes death in infancy. The case became controversial because the medical team and parents disagreed about whether experimental treatment was in the best interests of the child. The case has been classified by legal academics as a 'stigmata case', cases that "are part of the meditation of a culture upon itself."
Why was Charlie put on a ventilator?
On 11 October, Charlie was taken to Great Ormond Street Hospital (GOSH) and put on a mechanical ventilator, because his breathing had become shallow. By November, the doctors suspected that he had mitochondrial DNA depletion syndrome (MDDS), a set of rare diseases caused by mutations in genes essential for mitochondria to function. This diagnosis was confirmed by a genetic test in mid-November, which found that he had two mutated versions of the gene coding for the RRM2B protein.
What did Gosh discuss with Charlie's parents?
GOSH began discussing with the parents the ending of life support and the provision of palliative care. By this time, relations between the parents and the doctors had deteriorated. One GOSH doctor emailed another: "Parents are spanner in the works. Recent deterioration with worsening seizures means trial is not in his best interests." The email emerged in the subsequent court case; the judge said that he understood the distress it had caused the parents, but that it was important to view the email in the context of notes passed between consultants about Charlie's best interests. The parents disagreed with the doctors at GOSH; they wanted to take him to New York to receive the nucleoside treatment.
What did medical experts say about Hirano?
Medical experts criticised interventions by Hirano and others for raising the parents’ hopes and for causing delays to the process. Genetics expert Robert Winston said "interferences from the Vatican and from Donald Trump" were "extremely unhelpful and very cruel". Winston added: "This child has been dealt with at a hospital which has huge expertise in mitochondrial disease and is being offered a break in a hospital that has never published anything on this disease, as far as I'm aware." Some commentators in the United States argued that Charlie's plight was the result of the UK having a state-run national health service and that the decision to withdraw Charlie's life support was driven by cost. The High Court judge described these comments as "nonsensical", adding that "it was one of the pitfalls of social media that the watching world felt it right to have opinions without knowing the facts of the case". The chairman of GOSH made a statement condemning "thousands of abusive messages", including death threats received by staff at the hospital and harassment of other families in the hospital over the preceding weeks. GOSH asked the Metropolitan Police Service to investigate the abuse. The parents issued a statement condemning harassment of GOSH staff and said they had also received abusive messages. GOSH released a statement criticising Hirano for offering testimony without having physically examined Charlie and without review of the medical records; they also said Hirano had disclosed that he had a financial interest in the treatment very late in the process. Hirano made a statement in response saying that he had relinquished his financial rights in the treatment.
What is the likelihood of a positive effect and benefits to Charlie of the proposed nucleoside therapy?
the likelihood of a positive effect and benefits to Charlie of the proposed nucleoside therapy to be markedly improved compared to the views expressed in court; the likelihood that the proposed nucleoside therapy will cross the blood brain barrier to be significantly enhanced.
When was Charlie scheduled for a tracheostomy?
A committee meeting was scheduled for 13 January, and Charlie was provisionally scheduled for a tracheostomy on 16 January. GOSH invited Hirano to examine him in January, but he did not examine Charlie until July.
Why is the Moral Maze case not relevant?
Gillon, in the Moral Maze programme accepted the proposition that there could have been a resources argument against the use of the proposed experimental treatment but said that this was not a relevant issue in Charlie's case because the parents had raised sufficient funds to pay for the treatment. This was accepted by Wilkinson in the same programme. But Robert D. Truog, director, Harvard Center for Bioethics, Frances Glessner Lee Professor of Legal Medicine, Professor of Anaesthesia (Pediatrics) argued that the notion that Charlie's parents are paying is misleading. Tertiary care medical centres able to do research and provide care depend on communal investment made by society over decades. Society always has a financial stake in how communal services are used and has a legitimate claim in insisting that these resources be used wisely and for the benefit of all. "No one can demand non-beneficial treatments simply by claiming that they are paying out-of-pocket." It has been argued that the case had a negative impact on distributive justice, as Great Ormond Street Hospital incurred legal costs of £205,000, including VAT (money diverted from medical care to lawyers), and that distributive justice could be harmed further if proposed reforms known as 'Charlie's Law' (which would replace the best interests test with a significant harm test) are enacted.
Agreement and disagreement about experimental treatment. The Charlie Gard Appeal
Tomorrow, the UK Court of Appeal will review the controversial case of a British infant, Charlie Gard. Charlie’s parents are appealing a recent High Court decision that gave doctors permission to withdraw his life support. They have raised money for Charlie to travel to the US for an experimental medical treatment.
Best Interests
The legal decision for Charlie will be based upon an assessment of his best interests. He has a rare genetic disorder affecting his muscles and his brain. He has been on life support since last October and has been progressively deteriorating.
Disagreement
One approach to difficult decisions is to seek agreement or consensus. For example, doctors in an intensive care unit might hold a multi-disciplinary meeting to discuss a patient’s treatment, and whether it is worth continuing. If they all agree that treatment isn’t helping, they may then seek to withdraw life support.
Back to Resources
One final point is worth making about the resource implications of these decisions. Agreement or disagreement about benefits and risks is not the only consideration. Even if there are professionals prepared to provide a treatment, sometimes it cannot be provided because it is not affordable within the public health system.
What is the case of Charlie Gard?
In 2017, the court case over medical treatment of UK infant, Charlie Gard, reached global attention. In this article, I will analyze one of the more distinctive elements of the case. The UK courts concluded that treatment of Charlie Gard was not in his best interests and that it would be permissible to withdraw life-sustaining treatment. However, in addition, the court ruled that Charlie should not be transferred overseas for the treatment that his parents sought, even though specialists in Italy and the US were willing to provide that treatment. Is it ethical to prevent parents from pursuing life-prolonging treatment overseas for their children? If so, when is it ethical to do this? I will outline arguments in defense of obstructing transfer in some situations. I will argue, however, that this is only justified if there is good reason to think that the proposed treatment would cause harm.
What is Charlie's parents' treatment?
In early 2017, Charlie’s parents identified a potential experimental treatment . In animal models, supplementation with deoxypryrimidine nucleosides in the myopathic form of MDDS apparently led to reduction in the biochemical defect and severity of clinical phenotype. 22 A US physician involved in the nucleoside research offered to provide treatment, and Charlie’s parents raised funds for Charlie to travel to the United States.
Why did parents seek tracheostomy in Italy?
31 One reason for parents seeking treatment in Italy was because they felt that the ethical values of the Italian doctors aligned with their own values. The Italian doctors would apparently continue intensive care as long as Tafida was not brain dead. In contrast, the UK doctors regarded continued treatment as futile. 31
When was Charlie Gard born?
Charlie Gard was born in August 2016. He developed early signs of muscle weakness at a few weeks of age and by 2 months of age was admitted to hospital with poor feeding, failure to thrive, and respiratory failure. He was ventilated and admitted to intensive care, where investigations led to diagnosis of infantile onset encephalomyopathic mitochondrial DNA depletion syndrome (MDDS).
Is it justified to obstruct transfer of children?
It is justified for health systems to obstruct transfer of children for treatment that is believed would cause harm to the child. Of course, that then leads back to the contested question of whether the treatment requested by parents would be harmful. Doctors and nurses who provide treatment to seriously ill patients are only too aware that such treatment is sometimes extremely unpleasant and can do more harm than good. Where there is good reason to think that a child would experience pain but have no realistic prospect of improvement, further life-prolonging treatment may cause harm.
Did Charlie's parents want to withdraw life support?
However, Charlie’s doctors were not happy with him being transferred overseas for treatment. They applied to the Family Division of the High Court on February 28 for permission to withdraw life support and to provide palliative care. Charlie’s parents opposed this plan. On April 11, Justice Francis ruled in favor of the hospital. 23 Charlie’s family appealed, and the decision was reviewed (and upheld) in the Court of Appeal (May 23), Supreme Court (June 8), and European Court of Human Rights (June 20). 24
What is Charlie's treatment called?
Charlie's parents have said that they want their son to have an experimental treatment called nucleoside therapy , an unproven treatment aimed at the DNA materials that his cells can't produce. This treatment has previously been used on patients with a less severe form of mitochondrial DNA depletion, known as a TK2 mutation, according to the Times. However, the therapy has never been used on someone with RRM2B mutations. And even the doctor who initially agreed to help the Gards seek this treatment later acknowledged that the therapy was unlikely to help Charlie, because the baby was in the late stages of the illness.
What causes Charlie Gard's rare condition?
Charlie Gard Controversy: What Causes Infant's Rare Condition? Charlie Gard, an infant in Britain, has a rare condition that affects the cell's mitochondria, which generate energy. He cannot breathe on his own, and is on life support.
Why did Charlie's parents postpone the withdrawal?
However, his parents have since said that the hospital has postponed the withdrawal in order to give them more time to say goodbye to their child , according to The Guardian. Charlie's condition is called encephalomyopathic mitochondrial DNA depletion syndrome.
How old is Charlie from Charlie's disease?
4, 2016, and has been hospitalized at Great Ormond Street Hospital in London since October, according to The New York Times. The 11-month-old cannot breathe on his own, has seizures, and is blind and deaf, the Times reported.
What are the effects of Charlie's condition?
It can cause muscle weakness, microcephaly (a smaller-than-normal head size), kidney problems, seizures and hearing loss. Weakness in the muscles used for breathing can lead to serious breathing problems and, in Charlie's case, required that he be placed on a ventilator.
Did Great Ormond Street Hospital move Gard?
Recently, the Bambino Gesù children's hospital in Italy asked if Gard could be transferred to its facility for care, but Great Ormond Street Hospital declined to move Gard, citing legal reasons, according to The Washington Post. U.K. Foreign Secretary Boris Johnson also said that it is "right that decisions continue to be led by expert medical opinion, supported by the courts," in line with the child's best interests, according to Press Association, a U.K.-based news agency.
Is Charlie Gard terminally ill?
Charlie Gard, a terminally ill infant in Britain, is receiving international attention as his parents are seeking an experimental treatment they hope could help their son, whose rare type of "DNA depletion" condition usually results in death in the first few months of life.
What is Charlie Gard's condition?
For those unfamiliar with the case in question, Charlie Gard was born in the UK in 2016 with mitochondrial DNA depletion syndrome, a severe condition that left him struggling for his life. This past March doctors told his parents that there was nothing more they could do.
What is the impact of Charlie Gard's case?
The case of baby Charlie Gard has the gravest consequences for the rights of parents and the autonomy of the family. This assault against the family and life stretches beyond the borders of the UK, and we should all be very concerned.
What injustice does Charlie die in?
The injustice is that Charlie will die when the hospital administration wants, and where the hospital administration wants. His parents have been deprived of their right to supervise his case. They could not take him the U.S. for experimental treatment. They could not take him home, to die in peace.
How much money did Charlie's parents raise?
In all, the parents raised over 1.3 million British pounds (nearly $1.7 million U.S.) – more than enough to pay for the treatment. For the first time, there was a glimmer of hope for Charlie.
What does it mean to forego extraordinary or disproportionate means?
… To forego extraordinary or disproportionate means is not the equivalent of suicide or euthanasia; it rather expresses acceptance of the human condition in the face of death.
Where is Charlie being kept?
For the first time, there was a glimmer of hope for Charlie. Then, inexplicably, the hospital where Charlie is being kept – Great Ormond Street Hospital in London – refused to release Charlie. They said they had determined that the proposed treatment was unlikely to help the boy, and would only prolong his suffering.
Is Charlie still on life support?
A ferocious public debate ensued, with global figures including President Trump, Pope Francis and many others weighing in. Charlie remains on life support, but at any moment the hospital could decide to remove his ventilator. Sadly, confusion has plagued ...
Overview
Treatment
Charles Matthew William Gard was born on 4 August 2016, at full term and normal weight, to Christopher Gard, a postman, and Constance Yates, a carer for young people with learning difficulties, both of Bedfont, west London. He seemed to develop normally at first, but his parents noticed after a few weeks that he was less able to lift his head, and they took him to his GP. On 2 Octobe…
Charles Matthew William Gard was born on 4 August 2016, at full term and normal weight, to Christopher Gard, a postman, and Constance Yates, a carer for young people with learning difficulties, both of Bedfont, west London. He seemed to develop normally at first, but his parents noticed after a few weeks that he was less able to lift his head, and they took him to his GP. On 2 Octobe…
Reactions
On 30 January 2017, the parents launched an appeal on the crowdfunding website GoFundMe to finance experimental treatment in the United States. Donations had exceeded £1.3 million by the end of April. The publicity campaign was well under way before the legal process had started. At no time during the court process was any criticism levelled at the parents for this campaign and there was no suggestion that the court's powers be used to limit or control this publicity.
Legal proceedings
The court proceedings were under the inherent jurisdiction of the High Court (wardship), conducted within the principles and provisions of the Children Act 1989.
In wardship, the High Court assumes parental rights and responsibilities if an application is made and the court deems the application appropriate. Parents and public bodies have a responsibility to take actions in the best interestsof a child. Doctors treat children with the consent of parents; …
Outcome
On 27 July 2017 Charlie was transferred to a hospice and the next day his mother announced at 6:30 pm that he had died. The mechanical ventilator had been withdrawn; he was given morphine to relieve any pain beforehand, and died within minutes.
Issues
Medical experts criticised interventions by Hirano and others for raising the parents’ hopes and for causing delays to the process. Genetics expert Robert Winstonsaid "interferences from the Vatican and from Donald Trump" were "extremely unhelpful and very cruel". Winston added: "This child has been dealt with at a hospital which has huge expertise in mitochondrial disease and is being offered a break in a hospital that has never published anything on this disease, as far as I'…
External links
• Charlie's Fight, family website
• Smith-Squire, Alison (parents' media representative). Charlie Gard—a family's real life story in the spotlight, FeatureWorld.
• GOSH's position statement (pdf), hearing on 13 July 2017