A New York hospital said on Thursday that it has agreed to admit and treat the British baby at the center of a life-and-death debate after European courts ruled that he could be removed from life support against the wishes of his parents.
New-York Presbyterian Hospital and Columbia University Irving Medical Center said in a statement that it would admit and evaluate Charlie Gard, 10 months old, who suffers from an extremely rare genetic disease called mitochondrial DNA depletion syndrome. A Columbia physician, Dr. Michio Hirano, is developing an experimental therapy that has been used on at least one American patient with a similar but less severe mitochondrial disease. In both cases, a mutation impairs the function of DNA in mitochondria, microscopic structures within cells that produce the energy they need to survive.
Charlie, who has irreversible brain damage, is currently on life support at Great Ormond Street Hospital for Children in London. Earlier this week a hospital in Rome affiliated with the Vatican offered to have him transferred to its care, but then told CNN that a transfer would not be possible for legal reasons.
The admission would occur, Columbia said, “provided that arrangements are made to safely transfer [Charlie] to our facility, legal hurdles are cleared, and we receive emergency approval from the FDA for an experimental treatment as appropriate.”
The experimental treatment that Hirano is developing is called nucleoside bypass therapy. It consists of molecules that are the building blocks of DNA, including the DNA in mitochondria. The idea is that molecules such as deoxythymidine monophosphate and deoxycytidine monophosphate would be converted to those required for the synthesis of mitochondrial DNA. Because the treatment is experimental, Columbia would need approval from the Food and Drug Administration to administer the compounds to Charlie under a “compassionate use” exemption.
If Charlie cannot travel to New York, Columbia said, and if the FDA approves, “we 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.”