Ther­apy at the cen­tre of the de­bate

Daily Mail - - News -

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.

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