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.
Parents thank Manchester doctors as toddler with fatal disease is saved by world’s most expensive drug / Credit: NHS England
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.
Ally and Jake have two beautiful girls, Nala and Teddi. In April 2022, both girls were diagnosed with metachromatic leukodystrophy (MLD), a genetic disease resulting in a life expectancy of five to eight years.
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 and older sister Nala were both diagnosed with MLD in April 2022 / Credit: NHS England
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.”
19-month-old Teddi has been treated with a revolutionary gene therapy / Credit: NHS England
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
Manchester
A long-standing Italian restaurant in Trafford has sadly announced its imminent closure
Danny Jones
Another day, another bit of unfortunate hospitality news, as long-standing Greater Manchester restaurant, Little Italy in Timperley, has announced it is closing permanently very soon.
The Trafford favourite, which has been serving locals for nearly 20 years, has informed regular diners that it will sadly be closing its doors for good, with Little Italy’s final service set for later this month.
Having earned plenty of praise in its time, including being one of the few spots in and around the Altrincham area to have won a TripAdvisor Certificate of Excellence in 2015, before the borough saw its foodie boom begin.
But fast forward a decade, and the Timperley institution is yet another culinary casualty to be confirmed of late, with Little Italy sharing a tear-jerking statement online.
Sharing the tragic news on social media this Friday (5 Sep), the restaurant wrote: “To all our lovely customers, it is with a heavy heart that we share some news with you all. After over 18 years of serving you all in Timperley, we will be closing our doors later this month.
“This has not been an easy decision. Unfortunately, rising costs and a significant increase in rental [costs] have made it impossible to continue in a sustainable way. As much as we love what we do, the reality is that it has become too difficult to maintain under these circumstances.
“Over the coming weeks, we will continue to cook with love and serve with the same passion as always. We would love to see you all and share one more meal, and raise a glass together.”
Safe to say that the post has been met with an outpouring of condolences and support from natives nearby and those who have travelled to enjoy their food.
Little Italy went on to add that they will detail more about their final week of business as soon as possible.
They can’t be accused of not keeping up with times and not trying to promote via social media either, as they small family-run team – headed up by Sam Cauchi, who bought the site back in 2008 – have always given things a go besides the day-to-day operation of the restaurant.
Being one of many saddened customers to respond underneath the announcement, we couldn’t possibly sum up things better than local Vanessa Nice, who said: “Look at all these amazing comments – you certainly made your mark as a home from home for the residents of Timperley.
“The end of an era will always be sad, but sometimes life and families have to move on.”
As for Cauchi and co., they signed off by simply adding: “From the bottom of our hearts, thank you for your support, kindness and friendship over the years. Timperley has been our home, and you have all been our family.
“Love from all the team at Little Italy.”
It goes without saying that we’re gutted for the gang and our thoughts are with everyone involved at this difficult time. For now, we can only say thank you to the countless Greater Manchester locations that continue to fall foul in the face of the rising pressures industry-wide. Case and point…
The bizarre explanation behind Manchester’s new mini ‘green space’ – that’s completely closed off
Danny Jones
One thing you always hear people who live in central Manchester calling out for is green space, but when a new patch of grass does arrive, you don’t exactly expect it to be completely inaccessible.
Just a quick FYI to those of you passionate about supporting what little nature there is left in our city centre: brace yourselves, as this one is rather annoying…
For anyone who’s walked down Whitworth Street West past the once lively Locks, or even got off at Deansgate–Castlefield tram stop and come down the glass stairs closest to the Albion Bridge, you may have noticed a rogue patch of grass surrounded by black fencing next to the City Road Inn pub.
It could be considered a nice little lawn were it not completely fenced off, but the question is, why has it popped up there? Well, local lad Curtis Rothwell has recently debunked the whole frustrating mess for us in a helpful video.
Yes, as explained by the Pre-Construction Manager who works for local utilities contractor Zenit Infrastructure Solutions Limited, the somewhat laughable but disheartening gated green space on Whitworth Street is essentially a feeble attempt at adhering to permission laws.
As detailed by Rothwell, the plot bought for a sizeable sum last year (only the original developers didn’t have the funding to actually pull off their initial plans) is now set to become student accommodation.
Currently, real estate firm Tri7 – which has now been fully folded into the parent group known as Fusion, as previous part-owners already – still has a hold on the land for now.
However, after being ordered by Manchester City Council to tidy up the area in the meantime, as they await planning permission to be fully approved, they decided to lay these new rolls of turf there.
Ok, so while the notion of the Council ordering a property company to make urban spaces look pretty and the idea of introducing more grassy zones into the city sound good on paper, as you can see, the real-life result doesn’t quite match up.
Not only does this feel like a bare minimum effort to appease the local authorities ahead of erecting yet another skyscraper, but more importantly, it feels like sheer nonchalant neglect and/or plain dismissal of those Manc residents crying out for more grass, nature and a break from the concrete jungle.
The fact that the rare green space is totally blocked by tall, almost authoritarian-feeling fences and sealed off by a big, black gate comes across like a casual two fingers to both the Council and those living in the city centre.
Speaking to The Manc, Curt commented: “It’s positive the council can take charge of how our empty spaces look after demolitions. In this case, maybe the demolition was carried out too early, which happens, but when it does, greenery is the best look they can give to local people.”
You can see the latest update on the planning permission HERE, and for now, if you’re looking for green spaces in Manchester, you’ll find them more frequently beyond the outskirts and heading into the boroughs; this one is strictly off limits. Find a whole bunch of ones that aren’t down below:
Emily Sergeant -
15th February 2023 
