The boy’s parents are convinced that an experimental therapy, developed by a neurologist in the United States, may help their son recover some functions — even though it has never been tested on someone with Charlie’s particularly severe form of the disease, known as mitochondrial DNA depletion syndrome.
The hospital said on Friday that it had not changed its view that Charlie had experienced “catastrophic and irreversible brain damage” and that the experimental treatment, known as nucleoside therapy, “would be futile and would prolong Charlie’s suffering.”
Nonetheless, the hospital said, it would ask the High Court — which on April 11 ruled in the hospital’s favor — to look at the case again “in light of the claimed new evidence.” The hospital added, “This is not an issue about money or resources, but absolutely about what is right for Charlie.”
The neurologist in the United States has played a central and at times confusing role in the courts’ deliberations — veering from optimism to pessimism and back again.
Courts have ordered journalists not to identify the neurologist, whom court papers refer to only as Dr. I.
Dr. I got involved in late December, after the family of a Baltimore boy reached out to him on the British family’s behalf. That American boy has a different, and less severe, form of Charlie’s disease.
Dr. I’s nucleoside therapy has been tested on mice and on 18 people with the TK2 mutation — including the Baltimore boy. But it has never been tried on someone with Charlie’s form of the syndrome, caused by a mutation in a gene called RRM2B.
When Charlie’s doctors asked for evidence that the therapy might help, Dr. I replied, “There is no direct evidence, but there is a theoretical scientific basis for saying it could.”
The hospital was open to the idea, but after Charlie suffered a series of epileptic seizures, doctors changed their minds, concluding on Jan. 13 that treatment would be futile.
Nonetheless, the parents began a campaign to raise money to take their son to the United States for the treatment. Soon after, both the hospital and the parents went to court, where Dr. I’s views were sought again.
In a March 23 letter, the neurologist said that he “cannot predict the outcome,” but that there was a “scientific rationale that the treatment could potentially ameliorate RRM2B deficiency.”
However, after reviewing new brain-scan data a week later, Dr. I appeared to back down, telling the court that it “is very unlikely that he will improve with that therapy.” He acknowledged that the brain damage was most likely irreversible, but he declined to categorically rule out the possibility of some recovery.
The parents persisted, producing a new letter from the neurologist, dated May 22, in which he once again took an optimistic approach: “In the best-case scenario, Charlie’s condition would stabilize, improve partially or continue to improve with long-term therapy, as we have seen in patients with TK2. Ideally, the treatment will ameliorate the seizures and allow more normal brain functions.”
An appellate court was unconvinced.
Desperate parents “may lose their objectivity and be willing to ‘try anything,’ even if, when viewed objectively, their preferred option is not in a child’s best interests,” the court found on May 23.
Britain’s highest court agreed, and last week, so did the European Court of Human Rights. But the pope and Mr. Trump weighed in with expressions of support this week, focusing a spotlight on the case.
The London hospital did not identify the two international institutions that have offered to help, but one of them appears to be NewYork-Presbyterian Hospital/Columbia University Medical Center. On Thursday, that hospital offered to treat the child, either as an inpatient in New York or by shipping the unapproved drug for the experimental treatment to the hospital in London.
“There is potential for him to be a completely normal boy, but we don’t know, as you just don’t know until you try,” Connie Yates, Charlie’s mother, said, adding, “There’s around a 10 percent chance of this working for Charlie.” She did not explain how she had arrived at that estimate.
Claire Fenton-Glynn, a legal scholar at the University of Cambridge, said that the last-ditch offers of help were not legally relevant.
“The central issue in this case is not the availability of treatment — there has always been a U.S. hospital willing to treat him — but, rather, that the courts have determined it is not in Charlie’s best interest,” she said.
But Kenneth Prager, a professor of medicine and director of clinical ethics at Columbia University, who is not involved in the case, argued that the parents’ wishes should not be brushed aside. The evidence on pain is unclear, he said. “Unless the parents are abusive, I think it is dangerous for society to arrogate to itself the power to override parental wishes and have the child die when they are clearly loving parents willing to expend time and resources to help their child,” he said.
That is also the view of Ms. Yates, who told ITV’s “Good Morning Britain”: “Euthanasia is illegal. Suicide is illegal. How is this legal?”
Ms. Yates said that she had been preparing last week to say goodbye to Charlie, until the White House got involved.
She had started preserving her son’s strands of hair and nail clippings.
“He was starting to look like a nutty professor, so I thought I’ve got to cut the sides — I wasn’t allowed to cut the top bit,” she said. “I got to keep his hair, as well. Just those little things I keep. It sounds ridiculous, but when I cut his nails I keep the clippings, in case it is the last time I cut them.”
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