Therapy at the centre of the debate
THE treatment Charlie Gard’s parents long to try is a drug called nucleosides therapy.
It replaces deoxynucleosides, which are naturally produced in healthy people, to repair DNA.
It has never been tested on anyone with the rare strain of mitochondrial disease Charlie suffers, but has had some success on patients with a similar strain.
Despite its experimental nature, Charlie’s parents believe it must be worth a try. But doctors said it would only cause him more pain, and the High Court agreed. Now the court has been asked to think again.
The debate involves two issues – if the disease itself can be treated, and whether there is any point in trying to do so if Charlie is brain-damaged.
Today’s High Court hearing is about whether the therapy has a chance of reaching Charlie’s brain by crossing the ‘bloodbrain barrier’, which separates the blood stream from the brain’s fluids.
The court had ruled there was ‘no evidence’ it could do so.
But seven international scientists wrote a letter last week declaring there was actually ‘substantial direct and indirect evidence clearly demonstrating’ that the drug can cross the blood-brain barrier.