New York hospital offers to treat British baby with rare disease

People gather in support of continued medical treatment for critically-ill 11-month old Charlie Gard in London.
People gather in support of continued medical treatment for critically-ill 11-month old Charlie Gard in London.PHOTO: AFP

NEW YORK (NYTIMES) - A leading academic medical centre in New York City has offered to treat Charlie Gard, an 11-month-old infant in Britain who was born with a rare and fatal genetic disease.

European courts have ruled that he should be taken off life support, as there are no effective treatments for his condition. His parents were denied permission to bring him to the United States for experimental therapy.

The court decisions captured the attention of Pope Francis and President Donald Trump, who tweeted on Monday that if the United States could help, "we would be delighted to do so".

A statement issued on Thursday by New York-Presbyterian Hospital/Columbia University Medical Centre said it has agreed to admit Charlie as an inpatient as long as he could be transferred safely, and there were no legal or regulatory barriers to treating him with an experimental medication.

The statement also said the medical centre would be willing to ship an experimental drug to the Great Ormond Street Hospital in London, where Charlie is being treated, if the Food and Drug Administration (FDA) approves.

U.S physicians would "advise their medical staff on administering it if they are willing to do so", the statement said.

Charlie was diagnosed with an extremely rare form of a disease called mitochondrial DNA depletion syndrome, believed to affect just over a dozen children worldwide. The syndrome prevents cells from producing the energy needed to sustain organs.

The baby was taken to the London hospital on Oct 11, when his parents, Connie Yates and Chris Gard, both in their 30s, noticed he was not growing and could not lift his head. He has been there since, breathing with the help of a ventilator, and being fed through a tube.

Charlie is deaf and suffers from persistent seizures. He also appears to have suffered brain damage.

Researchers at Columbia University have provided an experimental treatment to a child in Baltimore, Art Estopian Jr, suffering from a similar but less severe form of the syndrome.

The child's father, Art Estopinan, said he had been contacted by Charlie's parents and, in turn, had asked the researchers at Columbia University if they could help the infant.

Art Jr was 18 months old in 2012 when doctors diagnosed a form of mitochondrial DNA depletion syndrome and said he had less than two months to live.

"Everyone told me the same thing: There is no medication, there is no cure," Mr Estopinan said.

The treatment, called nucleoside therapy, is not approved by the FDA but can be requested under exceptions for compassionate use.

Mr Estopinan said that, with treatment, his son had slowly but steadily become stronger. Now six years old, Art Jr cannot walk but can move his hands and feet.

He breathes with the help of a ventilator, is fed through a tube and needs round-the-clock care.

Mr Estopinan said he was speaking out because "my wife and I believe that little Charlie Gard should be given a chance, because we believe there is hope". Ms Yates and Mr Gard have raised about US$1.7 million (S$2.35 million) to pay for the experimental treatment and to travel to the United States to seek care for their child.