Breakthrough for bladder cancer patients as tablet treatment rolled out on NHS
A groundbreaking targeted treatment for the most common type of bladder cancer has been approved for use by the NHS, offering new hope to hundreds of patients.
The National Institute of Health and Care Excellence (Nice) has recommended erdafitinib, also known as Balversa and manufactured by Johnson & Johnson, for some adult patients in England. This decision is expected to benefit about 420 individuals.
Erdafitinib is an oral medication taken once daily. It works by blocking certain proteins to slow down or stop the growth of cancer cells and has been recommended to adults with metastatic or unresectable FGFR-altered urothelial cancer who have previously received at least one line of immunotherapy that blocks the interaction between certain proteins.
According to the NHS, about 10,000 people are diagnosed with bladder cancer every year.
Urothelial cancer develops in the cells that form the inner lining of the bladder, urethra, ureter, or renal pelvis, the hollow part in the middle of each kidney.
It accounts for about 90 per cent of all bladder cancers, according to Nice.
Helen Knight, director of medicines evaluation at Nice, said she was 'pleased we have been able to recommend this effective, targeted treatment option which will make a significant difference to people's lives'.
'There are limited treatment options for this devastating and debilitating disease, which in many cases responds poorly to immunotherapies, so I'm sure today's guidance will be welcomed by patients.
'One of the benefits of this drug is it can be taken in the comfort of the patients' own home rather than having to travel to hospital to receive the treatment.'
Jeannie Rigby, chief executive of the charity Action Bladder Cancer UK, welcomed the Nice decision.
'There are very few treatments available for bladder cancer in general, and particularly for people with advanced disease,' she said.
'It's time that bladder cancer patients had some kind of new treatment available – we hope this is just the start of new hope for those with bladder cancer and their families.'
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Leader Live
29 minutes ago
- Leader Live
Parents whose sons are losing ability to walk face ‘nightmare' battle with NHS
The medicine givinostat can slow progress of Duchenne muscular dystrophy – keeping children on their feet for longer – and is being offered for free by a drug firm while it goes through the process of NHS approval. However, only some NHS trusts across the UK, and few in England, are giving the drug despite it being part of an early access programme since November. Families are now facing a 'cruel' postcode lottery over who gets the medicine and are in a race against time, according to the charity Duchenne UK. Some NHS trusts are refusing to supply the drug while others delay it by drawing up lists of who they think should get it first. NHS trusts say they need to pay for monitoring the drug – around £1,900 per year per patient – out of their own budget. In desperation, parents have met with Health Secretary Wes Streeting to try and speed up access. According to the Government, it is up to individual NHS trusts to decide whether to provide the drug. Duchenne UK is supporting families in their fight for givinostat, including Rosie and Pete Day, an investment manager, and their 10-year-old son Jasper, from Horsham, West Sussex. The Day family have twins Jasper and Arabella, aged 10, and two older daughters. Both Jasper and Arabella have Duchenne, with Arabella a carrier of the genetic disorder and Jasper the most affected, as the condition mostly affects boys. The drug, developed by ITF Pharma UK, is only being given under the early access programme to children who are still able to walk. Mrs Day told the PA news agency: 'We try and live in the moment and, in this moment, there's an opportunity to make a massive change to Jasper. 'We're not going to cure it, but this drug gives him the best chance of having the early life that he wants to have and what every parent wants to give their child. 'It's so close – and yet we're stuck. 'We're stuck in this process where there's something in reaching distance that could make a huge difference to keep him walking, keep him active, keep him with his siblings in the garden, keep him walking up the stairs, all of those things, and it just feels that we can't quite get there. 'We're on the finishing line to get something that will make a difference and we can't quite get there because of the system we're in. 'The drug is not going to cure it, but it will give him the childhood you would wish to give your child – to keep them playing football, coming on dog walks, being able to walk up the stairs, playing the French horn, all of those things that you dream your child is going to get to do. 'We can't give it because it we're stuck, even though it's free and it's there – we can't give it. 'It feels like a nightmare, because you know that the doctors want to give it, but when it comes to getting the drug in his mouth and seeing what it would do, we're stuck. 'It's a lottery, basically, depending on where you are in the country … and within NHS trusts themselves.' Jasper is under the care of the Evelina London Children's Hospital, which is only now starting to contact patients and is drawing up a priority list for who gets the drug first. 'Every day that passes by is a day that we can see Jasper decline and see him struggling with his muscles,' Mrs Day said. 'Two weeks ago, he asked me, 'When will I get the drug, mummy?' 'We're stuck in a situation over who's going to make those decisions regarding who gets the drug – of whether Jasper gets it or another boy. 'For every boy it's heartbreaking, because someone ultimately is not going to get the drug at that moment based on the current criteria.' She added: 'If Jasper stops walking, we're not going to get that back. 'Every day matters. Six months ago, Jasper could walk up and down the stairs easily. He can't do that now and Pete has to push him up the stairs every night. 'Yet we can't give him something that is as simple as two spoons full of medicine and a blood test, and a little bit of maybe resource looking at the blood test results – that is what is standing in our way.' Mrs Day said it was unclear how the Evelina is 'going to make a fair choice' because knowing who will lose mobility is not an exact science. Mr Day said the process is 'hugely subjective' because there is no linear decline in the condition and the decisions made by NHS trusts could end up being 'completely wrong'. According to Duchene UK, boys whose condition is getting worse risk falling off the list entirely while waiting for the drug. Around 500 boys in the UK are eligible for givinostat, which can be taken at home like Calpol, with hospitals then doing follow-up blood tests of around eight in the first year, followed by twice a year thereafter. Children may also need an ECG, though these are already part of routine Duchenne monitoring. Emily Reuben and Alex Johnson, founders of Duchenne UK, said: 'As time ticks by, more boys are losing out on their chance to access givinostat. 'This is a simple treatment, which can be easily managed at home, and requires uncomplicated blood tests to monitor. It's free to the NHS and could offer real hope for patients and their families. 'The delays are cruel and the postcode lottery is unjustifiable. We are calling on the NHS to urgently make this available to both ambulant (walking) and non-ambulant boys.' To date, all health boards in Scotland are rolling out the drug, alongside those in Swansea and Cardiff. Leicester Royal Infirmary was the first trust in England to give the drug, but the Evelina has not yet, and neither have trusts in Manchester, Liverpool and Newcastle. Great Ormond Street Hospital is working to supply the drug. A spokesman for the Evelina said: 'We have begun contacting families of all existing Evelina London patients who may be eligible for givinostat and are working to set up appointments in the next few months. 'Our clinical team are working through our patient lists and are currently prioritising children who need the treatment most urgently, or are at a higher risk of losing movement (ambulation) soon.' An NHS spokesman said: 'The first National Institute for Health and Care Excellence (Nice) committee meeting to consider this treatment is scheduled to be held in July 2025, and if manufacturer ITF Pharma can offer a cost-effective price to enable Nice to recommend its use, the NHS will be ready to work with the company to explore fast-tracking access for patients. 'NHS England has published guidance on manufacturer-led early access schemes, which require trusts to cover substantial costs and find additional clinical resources to administer new treatments, and we understand a number of trusts across the country are preparing to offer givinostat via such a scheme.'
Powys County Times
29 minutes ago
- Powys County Times
Parents whose sons are losing ability to walk face ‘nightmare' battle with NHS
Parents whose sons are losing the ability to walk have described the 'nightmare' battle they face with the NHS to access a free drug. The medicine givinostat can slow progress of Duchenne muscular dystrophy – keeping children on their feet for longer – and is being offered for free by a drug firm while it goes through the process of NHS approval. However, only some NHS trusts across the UK, and few in England, are giving the drug despite it being part of an early access programme since November. Families are now facing a 'cruel' postcode lottery over who gets the medicine and are in a race against time, according to the charity Duchenne UK. Some NHS trusts are refusing to supply the drug while others delay it by drawing up lists of who they think should get it first. NHS trusts say they need to pay for monitoring the drug – around £1,900 per year per patient – out of their own budget. In desperation, parents have met with Health Secretary Wes Streeting to try and speed up access. According to the Government, it is up to individual NHS trusts to decide whether to provide the drug. Duchenne UK is supporting families in their fight for givinostat, including Rosie and Pete Day, an investment manager, and their 10-year-old son Jasper, from Horsham, West Sussex. The Day family have twins Jasper and Arabella, aged 10, and two older daughters. Both Jasper and Arabella have Duchenne, with Arabella a carrier of the genetic disorder and Jasper the most affected, as the condition mostly affects boys. The drug, developed by ITF Pharma UK, is only being given under the early access programme to children who are still able to walk. Mrs Day told the PA news agency: 'We try and live in the moment and, in this moment, there's an opportunity to make a massive change to Jasper. 'We're not going to cure it, but this drug gives him the best chance of having the early life that he wants to have and what every parent wants to give their child. 'It's so close – and yet we're stuck. 'We're stuck in this process where there's something in reaching distance that could make a huge difference to keep him walking, keep him active, keep him with his siblings in the garden, keep him walking up the stairs, all of those things, and it just feels that we can't quite get there. 'We're on the finishing line to get something that will make a difference and we can't quite get there because of the system we're in. 'The drug is not going to cure it, but it will give him the childhood you would wish to give your child – to keep them playing football, coming on dog walks, being able to walk up the stairs, playing the French horn, all of those things that you dream your child is going to get to do. 'We can't give it because it we're stuck, even though it's free and it's there – we can't give it. 'It feels like a nightmare, because you know that the doctors want to give it, but when it comes to getting the drug in his mouth and seeing what it would do, we're stuck. 'It's a lottery, basically, depending on where you are in the country … and within NHS trusts themselves.' Jasper is under the care of the Evelina London Children's Hospital, which is only now starting to contact patients and is drawing up a priority list for who gets the drug first. 'Every day that passes by is a day that we can see Jasper decline and see him struggling with his muscles,' Mrs Day said. 'Two weeks ago, he asked me, 'When will I get the drug, mummy?' 'We're stuck in a situation over who's going to make those decisions regarding who gets the drug – of whether Jasper gets it or another boy. 'For every boy it's heartbreaking, because someone ultimately is not going to get the drug at that moment based on the current criteria.' She added: 'If Jasper stops walking, we're not going to get that back. 'Every day matters. Six months ago, Jasper could walk up and down the stairs easily. He can't do that now and Pete has to push him up the stairs every night. 'Yet we can't give him something that is as simple as two spoons full of medicine and a blood test, and a little bit of maybe resource looking at the blood test results – that is what is standing in our way.' Mrs Day said it was unclear how the Evelina is 'going to make a fair choice' because knowing who will lose mobility is not an exact science. Mr Day said the process is 'hugely subjective' because there is no linear decline in the condition and the decisions made by NHS trusts could end up being 'completely wrong'. According to Duchene UK, boys whose condition is getting worse risk falling off the list entirely while waiting for the drug. Around 500 boys in the UK are eligible for givinostat, which can be taken at home like Calpol, with hospitals then doing follow-up blood tests of around eight in the first year, followed by twice a year thereafter. Children may also need an ECG, though these are already part of routine Duchenne monitoring. Emily Reuben and Alex Johnson, founders of Duchenne UK, said: 'As time ticks by, more boys are losing out on their chance to access givinostat. 'This is a simple treatment, which can be easily managed at home, and requires uncomplicated blood tests to monitor. It's free to the NHS and could offer real hope for patients and their families. 'The delays are cruel and the postcode lottery is unjustifiable. We are calling on the NHS to urgently make this available to both ambulant (walking) and non-ambulant boys.' To date, all health boards in Scotland are rolling out the drug, alongside those in Swansea and Cardiff. Leicester Royal Infirmary was the first trust in England to give the drug, but the Evelina has not yet, and neither have trusts in Manchester, Liverpool and Newcastle. Great Ormond Street Hospital is working to supply the drug. A spokesman for the Evelina said: 'We have begun contacting families of all existing Evelina London patients who may be eligible for givinostat and are working to set up appointments in the next few months. 'Our clinical team are working through our patient lists and are currently prioritising children who need the treatment most urgently, or are at a higher risk of losing movement (ambulation) soon.' An NHS spokesman said: 'The first National Institute for Health and Care Excellence (Nice) committee meeting to consider this treatment is scheduled to be held in July 2025, and if manufacturer ITF Pharma can offer a cost-effective price to enable Nice to recommend its use, the NHS will be ready to work with the company to explore fast-tracking access for patients. 'NHS England has published guidance on manufacturer-led early access schemes, which require trusts to cover substantial costs and find additional clinical resources to administer new treatments, and we understand a number of trusts across the country are preparing to offer givinostat via such a scheme.'
Leader Live
29 minutes ago
- Leader Live
‘Trojan horse' treatment given green light for NHS use
The targeted therapy can stop myeloma from progressing by nearly three times as long as existing treatments, studies suggest. It is estimated that around 1,500 patients a year with multiple myeloma – an incurable cancer of the bone marrow – will benefit from the treatment. It comes after the National Institute for Health and Care Excellence (Nice) approved belantamab mafodotin, also known as Blenrep and made by GlaxoSmithKline, for NHS use. NHS England said that it is the first health system in the world to roll out the treatment. Officials said that the drug can now be offered to patients whose cancer has progressed or not responded to another first-line treatment. The treatment, which is given as an infusion every three weeks along with other cancer drugs, is a type of antibody drug which targets and attaches to cancer cells. It has been dubbed a 'Trojan horse' treatment because it works by being taken into a cancer cell, before releasing a high concentration of a lethal molecule to destroy the cell from inside. 'Myeloma is an aggressive type of blood cancer, but we have seen a steady improvement in the outlook for patients over recent years as we have introduced new targeted therapies,' Professor Peter Johnson, NHS England's national clinical director for cancer, said. 'I am delighted that patients in England will be the first to benefit from this new treatment, which has the potential to keep cancer at bay for years longer, giving people the chance of more precious time with friends and family. 'This treatment could be life-changing for many patients and their families, and that's why it is so important that the NHS continues to secure quick access to the latest, innovative treatments like this, at affordable prices to the taxpayer.' 🚨 Do you know the key symptoms of #myeloma? 🚨 Help us spread the word by sharing this post! Early diagnosis makes all the difference. If something feels off, don't wait – talk to your GP!#BloodCancerAwarenessMonth @MyelomaUK — DKMS UK | We Delete Blood Cancer (@DKMS_uk) September 6, 2024 Helen Knight, director of medicines evaluation at Nice, said: 'We're delighted that people in the UK will become among the first in the world to access belantamab mafodotin for this indication. 'This recommendation demonstrates our commitment to getting the best care to patients fast, while ensuring value for the taxpayer.' Trials have suggested that the treatment, in combination with bortezomib and dexamethasone, delayed progression of the disease by an average of three years, compared to just over a year for patients taking commonly-used drug daratumumab along with the other treatments. Patient Paul Silvester, 60, from Sheffield, was diagnosed with myeloma in July 2023 and received treatment at the Royal Hallamshire Hospital. The first treatment he was given failed to stop his cancer from progressing so he was given belantamab mafodotin through an early access programme. 'I feel like this treatment has brought the party balloons back in the house. It has been amazing – within the first two or three weeks, after the first dose, I was in remission,' he said. 'It gives me quite a lot of confidence in the drugs and it makes me more optimistic about the future. 'I've been feeling well and I'm still quite active – that's what's important in terms of your quality of life. 'One of my daughters is graduating from university in October and it's a goal for me to be there.' Shelagh McKinlay, director of research and advocacy at blood cancer charity Myeloma UK, said: 'It's fantastic to see the UK at the forefront of myeloma treatment. 'We have been working very hard for the last year to get this treatment approved and we know it will transform the lives of thousands of people with myeloma.' Health Minister Karin Smyth said: 'This groundbreaking therapy puts the NHS at the forefront of cancer innovation. 'By harnessing cutting-edge 'trojan horse' technology, we're offering new hope to blood cancer patients across the country.'
