Latest news with #Exa-cel
Bahrain News Gazette
17-02-2025
- Health
- Bahrain News Gazette
Crown Prince of Bahrain Visits First Successful Sickle Cell Treatment Patient Outside US
Manama: His Royal Highness Prince Salman bin Hamad Al Khalifa, the Crown Prince and Prime Minister of Bahrain, visited the world's first sickle cell disease patient outside the United States to successfully complete treatment with Casgevy, a CRISPR-based gene-editing therapy. The treatment was administered at the Bone Marrow and Stem Cell Transplant Unit at the Royal Medical Services – Bahrain Oncology Center. According to Bahrain News Agency, HRH Prince Salman bin Hamad emphasized the Kingdom's dedication to integrating scientific advancements into treatment and rehabilitation services. This commitment aligns with the broader vision of His Majesty King Hamad bin Isa Al Khalifa to enhance citizens' health and wellbeing and strengthen the Kingdom's health sector. HRH Prince Salman bin Hamad reiterated Bahrain's commitment to advancing its health sector by delivering world-class healthcare and medical treatments. He highlighted the significance of forging partnerships with international health institutions to ensure access to cutting-edge medical technologies. During the visit, His Royal Highness congratulated the patient on the successful completion of the treatment and inquired about his health and well-being. He was accompanied by several senior officials. Under the directives of HRH the Crown Prince and Prime Minister, the National Health Regulatory Authority approved the treatment 'CASGEVY (Exa-cel)' for patients with sickle cell anaemia and beta-thalassemia. This approval followed a thorough evaluation of clinical trials and submitted data, positioning Bahrain as the first country in the region and the second globally to authorize the use of this treatment. The Royal Medical Services – Bahrain Oncology Center has achieved a historic milestone by completing the treatment of a sickle cell disease patient using Casgevy for bone marrow transplantation. This accomplishment marks the first successful treatment of its kind outside the United States, strengthening Bahrain's status as a leader in precision medicine and innovative healthcare solutions.
Daily Tribune
16-02-2025
- Health
- Daily Tribune
Bahrain Achieves Historic Medical Milestone in Bone Marrow Transplantation
In alignment with the directives of His Majesty the King and under the guidance of the Crown Prince and Prime Minister, Bahrain has prioritized the advancement of treatment options for patients with chronic and hereditary blood disorders. In a significant step forward, the National Health Regulatory Authority (NHRA) has approved the use of "CASGEVY (Exa-cel)" for the treatment of sickle cell anemia and beta-thalassemia. This approval follows a rigorous evaluation process, including an extensive review of clinical trials and manufacturer-submitted data, positioning Bahrain as the first country in the region and the second worldwide to approve this innovative therapy. Furthermore, the Royal Medical Services - Bahrain Oncology Center has achieved a historic medical breakthrough by successfully completing the treatment of a sickle cell anemia patient through bone marrow transplantation utilizing the CRISPR gene-editing technique. This accomplishment establishes Bahrain as a global leader in advanced medical treatments, demonstrating its commitment to pioneering healthcare solutions and enhancing the quality of life for its citizens.
Yahoo
31-01-2025
- Health
- Yahoo
A £1.65m hope for blood disorder patients is now on offer in Manchester
A £1.65 million treatment has been approved for use for some NHS patients, offering hope of a cure to those with an inherited blood disorder. Campaigners have shared their joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder. It is estimated that 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for certain patients with the genetic condition. NHS officials estimate that around 50 people a year will receive treatment. Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its inventors the Nobel Prize for chemistry in 2020. It works by editing the faulty gene in a patient's own stem cells. READ MORE: Suspect named and set to face court following alleged M6 pursuit READ MORE: Knifeman left friend fighting for his life following unexplained attack Cells are taken from a patient to a laboratory where the Crispr technology is used. The edited cells are then infused back into the patient, which prompts the body to produce healthy red blood cells. The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell transplant. But it is not given to many patients due to the risks involved. Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found. In March last year, Nice rejected Casgevy for people with severe sickle cell disease. Nice said it required more information on the drug's effectiveness and a commercial agreement, with its £1.65 million list price. Now, a confidential deal has now been struck with its creators, Vertex, on how much the NHS will pay for the treatment under a "managed access scheme". Nice has endorsed the treatment for some people aged 12 and over who suffer from "recurrent vaso-occlusive crises" and have a specific genotype. Dr Samantha Roberts, Nice's chief executive, said: "Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing Nice's aim of reducing health inequalities associated with the condition and getting the best care to patients fast." Ludovic Fenaux, senior vice president at Vertex International, celebrated the announcement, saying: "Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease." In the UK, approximately 17,500 individuals live with sickle cell disease, which is particularly common among those with African or Caribbean heritage. The disease can lead to intense pain, life-threatening infections, and anaemia. Mehmet Tunc Onur Sanli, 42, from London, who has been battling sickle cell disease since he was diagnosed at age 11, explained: "Because of my illness, I often experience pain in my chest, bones, and muscles. "I had surgery on my spleen when I was six and a hip replacement at 22 – I will probably need another hip replacement in the next few months or years. Speaking about the frequency and severity of his sickle cell crises, he said: "I also suffer from regular sickle cell crises – last year, I had to go to the hospital at midnight after waking up in severe pain, and overall, I had to visit the hospital five or six times due to crises." "The pain is the worst I have ever felt in my life – it's hard to put into words." 'Not having to go to hospital for regular transfusions or taking medicine anymore would be a dream to me – gene therapy could offer that – but there's still a lot to consider in terms of the side effects that could come with this treatment and whether it would be the right choice for me.' In clinical trials, all patients who received exa-cel also avoided hospital admission for a year following treatment – and almost 98% had still avoided being admitted to hospital around 3.5 years later. The treatment will be offered at specialist NHS centres in London, Manchester and Birmingham. NHS England chief executive Amanda Pritchard said: 'This is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition. 'This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them. 'We are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.' John James, chief executive of the Sickle Cell Society, said: 'We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. 'The significance of this milestone for the sickle cell community cannot be understated – today's result will give hope to many and is the result of determined campaigning.' The Medicines and Healthcare products Regulatory Agency (MHRA) approved the treatment in November 2023 for patients aged 12 and over after a 'rigorous assessment of its safety, quality and effectiveness'. It was the first regulator in the world to approve the treatment. Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said: 'This groundbreaking decision to fund the UK's first ever Crispr-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition. 'Previously, only individuals fortunate enough to have a stem cell donor match could access stem cell transplants as a potential cure. 'With Casgevy, we now have a treatment that offers hope to more patients, the majority of whom are from African and African-Caribbean backgrounds and have experienced years of feeling ignored. 'This treatment has the potential to transform lives and offers a glimpse into the exciting possibilities of gene therapy.' Nice approved the treatment for certain patients with a severe form of the blood disorder thalassemia in August last year. The NHS spending watchdog said that it will collect more data while patients receive the treatment on the NHS before it evaluates the medicine again. It said that collecting more data through a managed access agreement may resolve some uncertainty in the evidence, particularly about how long the benefits of treatment with exa-cel last. Health minister Andrew Gwynne said: 'By offering a treatment that could allow patients to live a life free of debilitating illness, we will give people with conditions like sickle cell disease more freedom and independence, all whilst protecting vital NHS emergency services.' Professor Bob Klaber, director of strategy, research and innovation at Imperial College Healthcare NHS Trust – which led the UK arm of the clinical trials for exa-cel, said: 'Together with patients and industry partners, we are proud to be part of the ground-breaking research and international academic collaboration that has made this treatment possible. 'The treatment is an example of true medical innovation and will provide patients with no other options a potential cure for the painful, debilitating symptoms of their diseases. 'It also offers promising research avenues for other genetic diseases.'
Euronews
31-01-2025
- Health
- Euronews
‘Much-needed hope' as CRISPR gene editing therapy recommended for sickle cell disease
Some with severe sickle cell disease in England will now be able to access landmark CRISPR gene editing therapy, according to new health guidelines which were hailed as marking a 'significant shift' in treatment for the blood disorder. The UK's National Institute for Health and Care Excellence (NICE) said on Friday that it had approved the use of the gene editing therapy exagamglogene autotemcel (exa-cel) by England's National Health Service (NHS). NICE had previously rejected the therapy's use on the NHS to treat some people with sickle cell disease in draft guidance released in March. It approved the use of exa-cel for the rare blood disorder beta thalassemia later in the year. The therapy, which the UK's medicines regulator cleared for use in November 2023, has a list price of £1.6 million (€1.9 million) per course. Sickle cell disease is a group of inherited blood disorders that affects the shape of red blood cells. People with the condition can experience severe pain, infections, anaemia, and other problems, according to the NHS. The disease is more common in people from African, Caribbean, Middle Eastern, or South Asian family backgrounds. There are currently few treatments for symptoms of the disease and the treatments that exist have 'intolerable side effects,' NICE said. The gene editing therapy will be available to some people aged 12 and older with severe complications from the condition and where a stem cell transplant is 'suitable' but a donor cannot be found, according to the watchdog. 'Much-needed hope' "The approval of exa-cel today marks a significant shift in the treatment landscape of sickle cell disease in the UK,' Funmi Dasaolu, who has sickle cell disease and spoke to the NICE committee, said in a statement. 'It is the beginning of re-addressing the inequalities in care experienced by so many with the condition,' she said, adding that it provides 'much-needed hope'. The discovery of the CRISPR/Cas9 gene editing tool won two researchers, Emmanuelle Charpentier and Jennifer A. Doudna, the Nobel Prize in Chemistry in 2020. Exa-cel, also called Casgevy, involves taking a person's blood stem cells, using CRISPR to edit them in a lab, and replacing them in the patient. 'The approval of Exa-cel for NHS use in England is a very exciting moment, not only because this marks the first approval of a CRISPR-based gene therapy for SCD [sickle cell disease] in the NHS, but also because it offers a potentially curative treatment for eligible patients,' said Felicity Gavins, a professor of pharmacology at Brunel University of London. 'However, while Exa-cel is a breakthrough, it is not a cure for all SCD patients, and uncertainties remain about its long-term effectiveness, safety, and accessibility,' Gavins said in a statement. 'It is critical to continue funding research to develop treatment that benefit the broader SCD population and address remaining challenges in care'.
The Independent
31-01-2025
- Health
- The Independent
£1.65m gene-editing therapy offers hope of cure for some blood disorder patients
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder hope of a cure. Campaigners reacted with joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder. It is thought that 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for certain patients with the genetic condition. NHS officials estimate that around 50 people a year will receive treatment. Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its inventors the Nobel Prize for chemistry in 2020. Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications Dr Samantha Roberts, Nice It works by editing the faulty gene in a patient's own stem cells. Cells are taken from a patient to a laboratory where the Crispr technology is used. The edited cells are then infused back into the patient, which prompts the body to produce healthy red blood cells. The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell transplant. But it is not given to many patients due to the risks involved. Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found. In March last year, Nice rejected Casgevy for people with severe sickle cell disease. At the time Nice said that it needed further detail about the effectiveness of the treatment, also known as exagamglogene autotemcel (exa-cel) and made by Vertex. It also needed a commercial agreement for the drug, which has a list price of £1.65 million. Now health officials have reached a confidential agreement with Vertex on how much the NHS will pay for the treatment. Nice said that the treatment can be offered under a 'managed access scheme' for treating severe sickle cell disease in some people 12 years and over with 'recurrent vaso-occlusive crises' who have a certain genotype. 'Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing Nice's aim of reducing health inequalities associated with the condition and getting the best care to patients fast,' said Nice chief executive Dr Samantha Roberts. Ludovic Fenaux, senior vice president at Vertex International, added: 'Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease.' There are around 17,500 people with sickle cell disease in the UK. It is particularly common in people with an African or Caribbean family background. Symptoms can include very severe pain, serious and life-threatening infections and anaemia. Mehmet Tunc Onur Sanli, 42, from London, who was diagnosed with sickle cell disease aged 11, said: 'Because of my illness, I often experience pain in my chest, bones, and muscles. 'I had surgery on my spleen when I was six and a hip replacement at 22 – I will probably need another hip replacement in the next few months or years. 'I also suffer from regular sickle cell crises – last year, I had to go to the hospital at midnight after waking up in severe pain, and overall, I had to visit the hospital five or six times due to crises. 'The pain is the worst I have ever felt in my life – it's hard to put into words. 'Not having to go to hospital for regular transfusions or taking medicine anymore would be a dream to me – gene therapy could offer that – but there's still a lot to consider in terms of the side effects that could come with this treatment and whether it would be the right choice for me.' In clinical trials, all patients who received exa-cel also avoided hospital admission for a year following treatment – and almost 98% had still avoided being admitted to hospital around 3.5 years later. The treatment will be offered at specialist NHS centres in London, Manchester and Birmingham. NHS England chief executive Amanda Pritchard said: 'This is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition. 'This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them. 'We are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.' John James, chief executive of the Sickle Cell Society, said: 'We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. 'The significance of this milestone for the sickle cell community cannot be understated – today's result will give hope to many and is the result of determined campaigning.' The Medicines and Healthcare products Regulatory Agency (MHRA) approved the treatment in November 2023 for patients aged 12 and over after a 'rigorous assessment of its safety, quality and effectiveness'. It was the first regulator in the world to approve the treatment. Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said: 'This groundbreaking decision to fund the UK's first ever Crispr-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition. 'Previously, only individuals fortunate enough to have a stem cell donor match could access stem cell transplants as a potential cure. 'With Casgevy, we now have a treatment that offers hope to more patients, the majority of whom are from African and African-Caribbean backgrounds and have experienced years of feeling ignored. 'This treatment has the potential to transform lives and offers a glimpse into the exciting possibilities of gene therapy.' Nice approved the treatment for certain patients with a severe form of the blood disorder thalassemia in August last year. The NHS spending watchdog said that it will collect more data while patients receive the treatment on the NHS before it evaluates the medicine again. It said that collecting more data through a managed access agreement may resolve some uncertainty in the evidence, particularly about how long the benefits of treatment with exa-cel last. Health minister Andrew Gwynne said: 'By offering a treatment that could allow patients to live a life free of debilitating illness, we will give people with conditions like sickle cell disease more freedom and independence, all whilst protecting vital NHS emergency services.' Professor Bob Klaber, director of strategy, research and innovation at Imperial College Healthcare NHS Trust – which led the UK arm of the clinical trials for exa-cel, said: 'Together with patients and industry partners, we are proud to be part of the ground-breaking research and international academic collaboration that has made this treatment possible. 'The treatment is an example of true medical innovation and will provide patients with no other options a potential cure for the painful, debilitating symptoms of their diseases. 'It also offers promising research avenues for other genetic diseases.'
