A girl born with a rare genetic disorder has become the first person to be cured on the NHS with a revolutionary gene therapy.
Teddi Shaw suffers from suffers metachromatic leukodystrophy (MLD), a deadly inherited condition that causes serious damage to the nervous system and organs.
But the 19 month old has been given the chance of a normal life after NHS England struck a deal to buy Libmeldy – one of the world’s most expensive drugs.
The treatment had a list price of £2.8 million when it was released last year, but Orchard Therapeutics agreed to supply it at a discounted price.
Mother Ally Shaw, said:
“Teddi is doing absolutely brilliant.
“She is walking, running, a chatterbox – absolutely no signs so far of MLD. She is an absolute character and has everyone around her laughing all the time.”
NHS England chief executive, Amanda Pritchard, said:
“This is a huge moment of hope for parents and their babies who are born with this devastating inherited disorder, that can now be treated with a single round of revolutionary treatment at a specialist centre on the NHS.”
Libmeldy corrects the genetic cause of MLD by inserting functional copies of a faulty gene into the patient’s stem cells.
The stem cells come from the patient’s bone marrow or blood and are fed back into the body with the new genetic information.
Teddi received the treatment over several stages between June and October last year.
However, her three-year-old sister, Nala, who also has MLD, is too far advanced in her illness to be treated with the breakthrough therapy.
Ally Shaw said:
“Being told our first daughter, Nala, wasn’t eligible for any treatment, would continue to lose all functions and die extremely young was the most heartbreaking and hardest thing to come to terms with.
“However, amongst the pain was hope for our younger daughter, Teddi. We were told that a new gene therapy treatment had, luckily, recently been made available on the NHS.
“We are extremely privileged that Teddi is the first child to receive this on the NHS and grateful that she has the opportunity to lead a long and hopefully normal life.
“Without this treatment, we would be facing both our children being taken away.
“We can only hope that one day a treatment becomes available for all stages of MLD, and we feel strongly that it should be added to the newborn screening test to save more families from having to go through this heartache.”
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