A toddler with a rare fatal genetic condition has become the first child in the UK to receive a lifesaving gene therapy treatment on the NHS.
19-month-old Teddi has been treated with a revolutionary gene therapy known by its brand name Libmeldy – which has a list price of £2.8 million, and was the most expensive drug in the world when the NHS negotiated a “significant confidential discount” last year to make the treatment available to patients.
Despite this discount though, it still remains the most expensive drug licensed in Europe – with Teddi being the first child to be given the treatment.
The Northumberland toddler has a rare and fatal genetic disease called metachromatic leukodystrophy (MLD), and was sadly diagnosed with the condition along with her three-year-old older sister Nala in April last year.
MLD causes severe damage to the affected child’s nervous system and organs, and devastatingly results in a life expectancy of between just five and eight years.
Teddi was treated by a specialist service being delivered at Royal Manchester Children’s Hospital (RMCH) – which is collaboration with Manchester’s Centre for Genomic Medicine at Saint Mary’s Hospital.
The centre in Manchester is one of just five European sites administering the treatment – which works by removing the child’s stem cells and replacing the faulty gene that causes MLD, before re-injecting the treated cells into the patient – and is the only site in the UK.
NHS England explains that the most common form of MLD usually develops in babies younger than 30 months, and due to the development of a crucial enzyme that leads to a build-up of fats that then destroy the protective layers around the child’s nerves, it can lead to loss of sight, speech, and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death in childhood.
Teddi was the first person in the UK to receive the Libmeldy treatment outside of a clinical trial – which began when she was 12 months old.
Treatment first began with the removal of stem cells at the end of June, which were then treated before the transplant took place in August, and Teddi was discharged back to her home in Northumberland in October to become “a happy and healthy toddler” now showing no signs of the devastating disease she was born with.
Sadly though, Teddi’s older sister Nala was not eligible for the treatment, as the clinical guidance requires the gene treatment to be administered before the irreversible damage caused by the disease progresses too far, NHS England explained.
Teddi’s mum, Ally Shaw, has praised Manchester doctors for saving her little girl.
“In April last year, our world was turned upside down when not one, but both of our daughters were diagnosed with MLD,” Ally 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 heart-breaking 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 would like to say a huge thank you to our specialists, doctors and nurses and all the staff at Royal Manchester Children’s Hospital who have been fantastic in caring not just for Teddi, but us as a family.”
NHS England has called this “a huge moment of hope” for parents and their babies who are born with this devastating inherited disorder.
Speaking on the success of the treatment, NHS chief executive Amanda Pritchard said: “Thanks to advancements in gene therapies, and the commercial ability of the NHS to strike deals for cutting-edge drugs and then deliver them through our phenomenally skilled specialist staff, children born with this condition now have the opportunity to lead normal, healthy lives.”
Previous treatment options for MLD on the NHS were limited to managing symptoms and providing supportive care.
But now, the new Libmeldy treatment will be available to babies and young children with no clinical signs or symptoms, as well as those with early symptoms of the condition, so long as they can still walk independently and with no evidence of cognitive decline.
Featured Image – NHS England
85-year-old gran from Altrincham stars in new Adidas running advert
Everyone’s favourite gran, Barbara Thackray, is back in the headlines and this time she isn’t just jogging and raising money, she’s featuring in Adidas’ brand new running advert.
The Altrincham-born and bred grandmother, who has become a local legend and inspiration across the UK with her incredible fitness and fundraising feats in recent years, is no stranger to being interviewed following her amazing efforts, but now she’s popped up in the new Adidas running ad too.
Barbara stars alongside the likes of Liverpool footballer Mo Salah, Qatari hurdler Mariam Farid and Egyptian runner Khadija Hegazy in the new TV and YouTube ad spot.
In the genuinely moving minute-long commercial, Barbara’s lovely little face pops up right at the end. Always save your best till last.
Actually quite stirring and inspiring, right?
Maybe it’s just us going soft in our old age (we never used to well up and interviews, promise), or maybe it’s because we know all the truly wonderful things she’s been doing for charity and her local community of late.
The Alty nan, who turned 85 earlier this month — the same day she broke her own PB in the annual Trafford 10k — has raised over £20,000 for St Ann’s Hospice in just a few short years, having been a champion of the organisation for more than 10 years amongst her sister’s illness and eventual passing.
She only started running when she turned 77 but now she runs around 12 miles every week.
The organisation’s Fundraising Manager, Lucy Leeming, said: “The awareness Barbara has raised across Greater Manchester for the importance and vitality of St Ann’s Hospice has undeniably helped raise funds and awareness for our charity.
“Her passion and dedication to towards our charity shines through in everything she does for us, we’re so grateful to have her support.”
As for Barbara herself, she’s still encouraging people of all ages to get into running; her only advice is “to begin gently and listen to your body.” No wonder the global sports brand chose her: she embodies everything the campaign is about.
To show this Alty gran some love and help her continue her incredible efforts, you can donate HERE.
A restaurant in Ancoats is serving up plates of crispy fried squirrel
A Manchester restaurant is serving up plates of crispy fried squirrel – and their customers absolutely love it.
Taking game dishes to a whole other level, the wild crispy buttermilk fried squirrel at Ancoats restaurant Street Urchin comes served atop a creamy pulled ham hock cassoulet, with hazelnut bread croutons and nettle pesto and costs £22.50.
Sourced from their game supplier in Cumbria, it might sound nutty but owner Rachel Choudhary told The Manc that the dish has proven incredibly popular – and that the team has been ‘really surprised’ at how much of a hit it has become with customers since adding it to the menu.
She said: “We were looking for something new for the game options on the menu. Kev was speaking to our game supplier and randomly asked if he had any grey squirrels. Happily, he did.
“The whole team tried the dish the day it went on and the majority thought it was really good.
“We weren’t sure if it would sell, but have been really surprised. So many people have tried it and given good feedback. We’ve recommended that they eat it like chicken wings and pick it up, that way you get most of the meat.
“I’d never tried squirrel before and I absolutely loved it, it has great flavour, rich buttery texture, and it’s wild, free-range meat.”
The gray squirrel currently has an estimated population of 2.5 million in the UK according to the Game and Wildlife Conservation Trust and is considered a good sustainable alternative to factory-farmed meat.
It is legal to control grey squirrels by shooting or trapping them in the UK in a humane manner and even helps protect the UK’s endangered native red squirrel population.
Whilst some might find the idea of eating squirrels a tad unappealing, many chefs argue it is better to eat them when culling as it means the meat does not go to waste.
English market diner Street Urchin was first opened on Great Ancoats Street in 2019 by husband and wife team Rachel and Kevin Choudary.
Prior to opening in town, the couple ran The Victoria in Altrincham for eight years before deciding that it was time to move on.
Their Ancoats market diner has made its name on being one of the few city centre restaurants specialising in fresh fish, with everything from clams and mackerel to cured chalk stream trout, king scallops, and whole grilled red gurnard regularly available on the menu.