Child suffering from rare disease saved by world’s most expensive drug
Baby Teddy Shaw has become the first British child to receive millions of dollars of NHS gene therapy for a rare, deadly genetic disease.
A 20-month-old child inherited life-limiting metachromatic leukodystrophy (MLD). It affects one in 40,000 babies and causes loss of nervous system and brain function, leading to a life expectancy of five to eight years.
Libmeldy’s revolutionary treatment, which has a list price of £2.8 million, is a one-time gene therapy and was the world’s most expensive drug when NHS England began negotiations for a massive discount.
It works by removing the baby’s stem cells and replacing the faulty MLD-causing gene before reinjecting the treated cells.
Now Teddy has become the first British child to have Libmeldy on the NHS. She began treatment last June and is now happy and healthy, recuperating at home in Northumberland.
Unfortunately, Teddy’s 3-year-old sister, Nala, also suffers from MLD, but is medically inappropriate for therapy.
The treatment is available on the NHS as a specialized service and in England it is provided at the Royal Manchester Children’s Hospital, one of only five facilities in Europe where treatment is provided.
Mum Ellie Shaw, 32, married to the girls’ father Jake, 29, said: “Teddy is feeling amazing! He walks, runs, talks, there are no signs of MLD yet.
“Last April, our world was turned upside down when not one but both of our daughters were diagnosed with MLD.
“When we were told that our first daughter, Nala, was beyond any treatment and would die very young, it was heartbreaking. However, in the midst of the pain, there was hope for Teddy.
“We are honored that Teddy is the first child to receive this in the NHS. Without it, we would have faced both of our children being taken away.”
Libmeldy, manufactured by the British firm Orchard Therapeutics, is a one-time treatment that addresses the underlying cause of MLD.
NHS Executive Director Amanda Pritchard said: “Thanks to advances in gene therapy and the commercial ability of the NHS to negotiate cutting-edge medicines, children born with the disease now have the opportunity to lead normal, healthy lives.”
Professor Rob Wynn of Manchester Royal Children’s Hospital said: “It’s great to be part of this breakthrough moment.”
Commentary by Stephen Powis, Medical Board, NHS England
MLD is a rare but devastating disease that has historically led to premature death.
About five babies fall ill every year in England.
But now, thanks to revolutionary advances in so-called gene therapy and a deal struck by NHS England, we can treat previously untreatable patients with a single course of therapy.
Libmeldy, which was the world’s most expensive drug when the NHS first made it available, is being introduced by a specialized service in Manchester.
It removes the baby’s stem cells and replaces the defective gene that causes MLD before they are reintroduced into the patient.
The disease itself is caused by a deficiency in an enzyme that allows fats to break down the protective layers around the nerves.
The therapy reprograms the cells to produce the missing enzyme.
This is yet another example of how the NHS is leading the way in delivering patient-friendly and tax-payer-friendly deals – we already have five drugs for every four people on the continent.
This is a momentous moment in how we treat genetic diseases and a moment of hope for children and their families to give toddlers like Teddy the chance to live happy and healthy lives.
The Sarpy County – Latest News:
Omaha Local News || Nebraska State News || Crime and Safety News || National news || Tech News || Lifestyle News