LONDON – Time is running out for Charlie Gard, the 11-month-old from England has a rare disease that doctors say is untreatable and they plan to take him off life support soon.
His parents want to bring him to the United States, where they believe an experimental treatment has a chance, hopefully, to prolong their son's life. But three courts in Britain agree with the hospital, and say the child should be taken off life support, despite efforts to help by two of the most powerful men in the world: President Trump and the pope.
Charlie's rare genetic disorder is called mitochondrial DNA depletion syndrome. It will cause him to die at a very young age, but his parents refuse to give up.
"He's a little trooper and he's a soldier,” his father, Chris Gard, said. "He’d fight to the very end and he’s still fighting but we're not allowed to fight for him anymore.”
Dr. William Craigen, of Baylor College of Medicine in Houston, has not met Charlie but researches this genetic disorder and said that, in his entire career, he's seen fewer than 10 patients with this disease. None of them have survived.
So what exactly is happening inside these tiny children?
“An energy failure, because that's what mitochondria do, and that causes cell death,” Craigen said. “Typically (the symptoms are) neurologic, so loss of muscle tone, loss of breathing ability, poor swallowing.”
It's a difficult biology lessen to digest but, basically, mothers who carry this have a 1 in 4 chance of passing it on to their children and when they do, it’s devastating.
“It's such a tragedy for a family like this. It's outside of the normal experience and nobody wants their child to die before they do, so I think it's a very trying time and it's understandable why a family would be grasping at any possible therapies in this regard,” Craigen said.
Craigen and his department work to find treatments and early diagnosis but he said even small progress with experimental drugs that Charlie’s parents are pleading for, may not be the miracle they need.
Charlie's parents are putting a lot of hope into a tweet from President Trump that said, " If we can help little Charlie Gard as per our friends in the U.K. and the Pope, we would be delighted to do so."
Craigen said it could be possible to bring Charlie to the United States in special medical planes but typically the cost of that kind of transportation is not possible for individual families.
