Daily Mirror (Northern Ireland)
‘Up to 56% chance that Charlie may get better...’
CHARLIE Gard’s chance of life could be boosted by over 50% with trial treatment, a US expert claims.
The leading neurologist told the High Court his pioneering therapy was “worth trying” and could lead to a “small but significant” improvement for the desperately sick 11-month-old.
Charlie would be the first person given the therapy for the particular mutation of the wasting disease that is killing him.
But in trials on a different form of the disease, patients’ conditions improved and their muscles regained strength.
The US professor questioned claims by doctors at Great Ormond Street Hospital that Charlie has irreversible brain damage and that keeping him alive is only prolonging his suffering – claims also rejected by the baby boy’s parents.
Explaining how Charlie’s unresponsiveness could be due to his muscles shutting down, he said: “If you do not have the wheels, you cannot tell if the engine is working properly.”
The doctor, who cannot be named for legal reasons, has not yet seen Charlie for himself and admitted he was not “fully briefed” in all aspects of the case.
Appearing via video link from
New York, the medic, who previously gave evidence to the High
Court in April, said: “Since
April we’ve evaluated more data. Based on that information I’ve put the chance of clinically meaningful success at at least 10%.
“But that is somewhat conservative. Of the nine patients, five improved, so it is between 11% and 56%.
“This is the best scientific data we have.
“When dealing with ultrarare diseases you have to make the most of every example.”
He said he would now come to the UK to see Charlie for himself, after being invited by the court. He added: “We cannot fix or cure his disease. I’m confident we can improve cognitive function but don’t know how much.”
He said he had carried out tests on mice that slowed the progression of the RRM2B mutation of mitochondrial DNA depletion syndrome, the wasting disease that is killing Charlie.
But in human trials on a different type of MDDS he saw patients’ conditions improve and their muscles regain strength. They were measured by the amount of time they were reliant on a ventilator to breathe. One patient was completely weaned off the machine, while four others used it less.
But Katie Gollop QC, for Great Ormond Street Hospital, where Charlie is in intensive care, called the doctor’s claims “highly theoretical”.
She added: “The court is not helped by evidence from those who have never ever met Charlie Gard.”
Charlie’s mum Connie Yates and dad Chris Gard want their son to be given the
If you do not have the wheels, you cannot tell if the engine is working properly US DOCTOR REJECTING CLAIM CHARLIE HAS IRREVERSIBLE BRAIN DAMAGE The court is not helped by evidence from those who’ve never met Charlie KATIE GOLLOP QC ACTING FOR GREAT ORMOND STREET HOSPITAL
non-invasive oral treatment and have been locked in a battle with his doctors at GOSH in Central London where Charlie is on a ventilator.
The High Court ruled in April that life support should be removed. Charlie’s parents launched unsuccessful challenges in the Court of Appeal and Supreme Court, and have also failed to persuade European Court of Human Rights judges to intervene.
But the case was referred back to the High Court amid claims of new evidence from abroad.
Mr Justice Francis said he had a duty to deal with the case “swiftly but fairly”.
He added: “It is now more than three months and either every day is a wasted one because he could be having the treatment or, according to others, every day is increasing the turmoil for Charlie.
“If there is important evidence which suggests that I should change my decision then I will change it.”
In emotional scenes in court yesterday the parents stormed out after arguing with the judge. Chris shouted: “I thought this was supposed to be independent,” as they went.
When they returned, Connie, 31, gave the US expert a thumbs-up as he presented his evidence while Chris, 32, held Charlie’s toy monkey to his face.
After an exhausting afternoon, Connie left the court sobbing and was comforted by family and friends. In a statement before the hearing began, the couple from Bedfont, West London, said they loved their son “more than life itself ”, adding: “If he’s still fighting, we’re still fighting.”
They have raised over £1.2million to take him to the US for treatment.
Their battle has touched hearts across the world, with the Pope and President Donald Trump both backing the family as hospitals in Rome and the US offered to treat Charlie.
Mr Justice Francis delivered a warning to internet trolls who have hurled abuse and threats against GOSH and its staff as the campaign to keep Charlie alive has gathered pace.
Warning they will face the harshest penalties possible, the judge said: “Anyone who threatens doctors doing their very best for a young baby... how they think that can help the parents’ case is beyond me. It is grossly unfair.”
GOSH paid tribute to the parents’ untiring fight for Charlie, but questioned the idea that only they should have the right to decide on Charlie’s treatment. The hospital said it “owes a duty of care” to each individual.
The hearing continues today.