An eight-year-old Cheshire girl with a rare degenerative disease has become one of the first to receive a pioneering new treatment that will attempt to save her eyesight.
Amelia Carroll, from Poynton near Stockport, was diagnosed with CLN2 Batten disease – an incurable and fatal condition that affects roughly around 30-50 children in the UK – when she was just two.
The disease is caused by a change in a gene that is responsible for making a specific enzyme in the nervous system, and it results in seizures, a gradual decline in a child’s ability to walk, speak and see, as well as progressive dementia.
Amelia’s 11-year-old brother Ollie also has the same condition, yet has sadly already lost his eyesight.
“We have watched our son Ollie go blind, and now the same is starting to happen to Amelia,” mother Lucy Carroll explained, which is why her and her husband Mike decided to contribute to a fundraising campaign together with families of other children affected by the disease alongside the Battens Disease Family Association (BDFA) to help fund a world-first trail of a groundbreaking new treatment at Great Ormond Street Hospital (GOSH).
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Over £200,000 was raised through the campaign, and clinicians and researchers at GOSH and ICH say the treatment programme has the potential to transform the quality of life for children living with CLN2 Batten disease.
Amelia became one of the first children in the world to receive the treatment, and is said to already be responding to it “perfectly”, CheshireLive reports.
For the past two years, enzyme replacement therapy has been used to help prevent neurological deterioration in children with CLN2 disease. The drug, called Brineura, was first approved for use on the NHS in 2019 and is administered directly into the brain by a regular infusion.
It has shown to restore enzyme activity and slow the onset of disability.
However, this infusion into the brain does not prevent children going blind, as the enzyme cannot cross the blood-retina barrier, and this means that the nerves in the eye cannot function and vision is lost, which is why clinicians and researchers at GOSH and ICH are trying a the treatment to prevent the onset of blindness instead.
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To do this, GOSH said they are using the tiny amount of the drug leftover from the brain infusion and injecting it directly into the back of the eye.
The 18-month treatment trail – which is said to be being given on a compassionate use basis – hopes to prevent eyesight deterioration in children by providing the enzyme that the nerves in the back of the eye need to function.
Ms Carroll said “to save a child’s sight would be incredible”, and if it does indeed work, it would “make such a big difference to Amelia’s quality of life”.
“We just pray it works,” she added.
Speaking on the groundbreaking treatment trail, Professor Paul Gissen, from GOSH and the UCL GOS Institute of Child Health, said: “If successful, we hope our work can pave the way to saving the sight of more children with this disease to preserve their quality of life for as long as possible.”
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Amelia’s father, Mike Carroll, said the “amazing” nurses and doctors treating his daughter were “incredible”.
NHS to begin offering new one-minute jab to women with ‘aggressive’ form of cancer
Emily Sergeant
The NHS is set to begin offering new immunotherapy for hundreds of women with aggressive cervical cancer across the country.
Pembrolizumab – which experts have described as being able to ‘take the handbrake off’ the body’s immune system to target cancer – will now be presented as a new treatment option for women in England with locally-advanced cervical cancer, which means the cancer has grown beyond the cervix to regions such as the pelvic wall, but not yet spread further around the body.
Trials found that adding pembrolizumab to standard chemoradiotherapy helped keep cancer ‘at bay’ for longer, and improved survival rates overall.
Two years after starting the treatment, nearly seven in 10 patients (68%) were still living without their cancer progressing, compared with 57% for those receiving chemoradiotherapy alone, according to NHS figures.
The trial also found that 82.6% of patients were still alive three years after treatment with pembrolizumab and chemoradiotherapy, compared with 74.8% with chemoradiotherapy alone.
Hundreds of women with aggressive cervical cancer are to be offered a new immunotherapy treatment.
It marks one of the biggest improvements in treatment for the disease in years, and could help more women survive and stay cancer-free in the long term.
The drug is either given every three or siz weeks via an infusion, or as a ‘one-minute’ injection, alongside chemoradiotherapy.
The NHS estimates around 550 patients in England will be eligible for the treatment – which has been approved this week by the National Institute for Health and Care Excellence (NICE) – over the next two years.
Patients will now receive fast-tracked access, funded by NHS England’s Cancer Drugs Fund.
“This is great news for women facing a diagnosis of aggressive cervical cancer, and represents one of the biggest improvements in treatment for this disease in recent years,” commented Professor Peter Johnson, who is the NHS National Clinical Director for Cancer.
“Combining this immunotherapy with existing treatment has had very positive effect for patients in trials, helping the body’s immune system to target cancer more effectively.
“We’re delighted it will be available for patients on the NHS as it could help hundreds more women survive and stay cancer-free in the long-term.”
Featured Image – NappyStudio (via Unsplash)
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Andy Burnham wants to bring the ‘Greater Manchester way’ to Westminster
Emily Sergeant
Andy Burnham has set out his vision for the country if he is to become Prime Minister.
In case you need brining up to speed first, after it was announced earlier this month that Andy Burnham had clinched the victory in the crucial Makerfield by-election, winning 24,927 votes (54.8% vote share) and a majority of 9,231, he then went onto announce his intention to run for Labour Party leader, and therefore Prime Minister, after Keir Starmer confirmed he would be stepping down.
And this week, Mr Burnham has now delivered his first speech as part of his ongoing campaign, addressing how he plans to give the country a ‘new direction’.
Burnham says that he wants to bring the ‘Greater Manchester way’ to Westminster.
A lot has been discussed and reported on when it comes to Burnham’s intentions to create a so-called Number 10 North here in Manchester, but what exactly does it mean to take the ‘Greater Manchester way’ to the capital?
“The Greater Manchester way is based on strong partnership between all sectors: public, private, community, voluntary, academic, faith, and our trade unions,” Burnham said in his speech.
He continued: “When I started as Mayor in 2017, we set about building a new approach, a new politics based on the exact opposite of the Westminster approach.
“Place-first, not party-first. Problem-solving, not point-scoring. Long-term, not short-term.
“A decade on, it’s incredible how much we’ve been able to achieve by working together instead of fighting against one another.”
Burnham said he feels the truth is that the country spends ‘too much time arguing and not enough time doing’ and that for Britain to get back where it ‘should be’, his Government would ask everyone to ‘face the same way’ and then ‘pull in that same direction together’.
He declared that No 10 North will be the ‘nerve centre’ for a rewired Britain.
“It will be the conduit through which we redistribute power and resources across the UK,” he concluded. “It will coordinate all parts of Government, at national and local level, to agree a long-term economic strategy and help all places set new growth ambitions.”
Emily Sergeant -
14th January 2022 
