New York-Presbyterian Hospital/Columbia University Medical Center on Thursday said it is willing to treat an 11-month old British infant with an extremely rare genetic disease whose care has drawn worldwide attention.
The infant, Charlie Gard, has a condition called mitochondrial DNA depletion syndrome, which prevents cells from producing energy needed to sustain organs. Charlie is deaf, experiences seizures, and appears to have brain damage. The syndrome affects only about a dozen children worldwide, according to the New York Times.
Gard has been treated by Great Ormond Street Hospital for Children in London since Oct. 11, when his parents observed that he couldn't lift his head and didn't appear to be growing. Gard currently is on life support.
Gard's parents had sought to bring him to the United States for treatment. However, a succession of European courts have sided with specialists at Ormond Street who say the proposed therapy won't help Gard and would cause further harm. On Tuesday, the European Court of Human Rights sided with previous courts, ruling that it was in Gard's best interest to be removed from life support.
New York-Presbyterian Hospital/Columbia University Medical Center offers care
In a statement released Thursday, Columbia said it would admit and evaluate Charlie, STAT News reports.
According to the New York Times, the offer comes after Gard's parents reached out to the father of an American child with a similar but less severe mitochondrial disease who is receiving an experimental treatment, called nucleoside therapy, at Columbia. The American child's father, Art Estopinan, said he then contacted researchers at Columbia.
The treatment does not have FDA approval, but the agency sometimes grants exceptions for compassionate use, according to the Times. According to Estopinan, his child has grown stronger with treatment. While the child, age 6, cannot walk, he can move his hands and his feet. He breathes with assistance from a ventilator and needs round-the-clock care, the Times reports.
In the statement, Columbia said it would be willing to admit Gard "provided that arrangements are made to safely transfer [Gard] to our facility, legal hurdles are cleared, and we receive emergency approval from the FDA for an experimental treatment as appropriate."
Columbia added that if Gard cannot travel to New York, Columbia—provided FDA OKs the plan—"will arrange shipment of the experimental drug to Great Ormond Street Hospital and advise their medical staff on administering it if they are willing to do so" (Begley, STAT News, 7/6; Rabin, New York Times, 7/6; People/Boston Herald, 7/7; BBC News, 7/7).
The journey to personalized medicine
From risk assessment to shared decision-making to self-management, learn the nine steps your organization can take on the path toward personalized medicine.