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Korea Herald
14-05-2025
- Health
- Korea Herald
A NEW HOPE FOR PATIENTS SUFFERING FROM POTENTIALLY FATAL RARE HEART DISEASE: NUHCS IS FIRST IN ASIA TO RECRUIT PATIENTS FOR LANDMARK GENE EDITING CLINICAL TRIAL
The international study explores gene editing therapy to stop the production of proteins causing the disease in ATTR-CM patients SINGAPORE, May 14, 2025 /PRNewswire/ -- While receiving treatment for a car accident ten years ago, Mr Chua learnt that he had an abnormal build-up of protein in his heart vessels. This discovery led to the diagnosis of a condition known as transthyretin amyloid cardiomyopathy (ATTR-CM), a rare disease currently affecting approximately 150 patients in Singapore. ATTR-CM is caused by the build-up of misfolded, deformed transthyretin proteins in the heart, nerves and other organs due to genetic mutation or ageing. Symptoms of this potentially fatal rare disease are often vague and may include numbness in the hands and feet, lethargy and dizziness. If not diagnosed and treated promptly, ATTR-CM can lead to heart failure. Over time, Mr Chua's hands and legs became stiff, and the once active 62-year-old could no longer walk. The subsequent years following his diagnosis were riddled with frequent visits to the hospital due to episodes of heart failure and multiple injuries from falls resulting from nerve issues caused by the disease. Despite being on years of medication, Mr Chua's condition had continued to decline, and his growing need for assistance with simple everyday tasks had greatly affected his spirit. There is currently no cure for this debilitating illness. A new international clinical trial may be set to change the trajectory of this disease for patients suffering from ATTR-CM – including Mr Chua. The MAGNITUDE study involves a single-dose gene editing therapy administered intravenously that will alter the patient's DNA, slowing down the production of the abnormal protein that causes the disease. Novel treatment offers new hope for patients Assistant Professor Lin Weiqin, Clinical Director of the Heart Failure and Cardiomyopathy Programme at the National University Heart Centre, Singapore (NUHCS), is leading the Singapore arm of this trial. He shared that the double-blind study will investigate the impact of the gene editing research medicine Nexiguran Ziclumeran (nex-z, also known as NTLA-2001) on ATTR-CM. "Gene editing therapy has been approved in other countries, for use in some neuro-muscular conditions, cancers and inherited blood disorders. If this trial is successful, it will be the first DNA altering treatment used in the field of adult cardiology and offers new hope to patients living with ATTR-CM," explained Asst Prof Lin, who is also a Senior Consultant at NUHCS. In Asia, the first patient of the double-blind clinical trial was recruited in Singapore and received his infusion in September 2024 at NUHCS. Since then, four more patients have been involved in the study, including Mr Chua, who was the fifth and most recent participant. The gene editing therapy is administered alongside the standard treatment for ATTR-CM as part of the study. While it may take another two to three years to see the outcomes of this study, early results of the trial medication in early phase clinical trials have shown promising results, with "consistent, rapid, and durable reductions" in the abnormal protein that causes ATTR-CM, and minimal side effects. [1] The patients in Singapore have also not reported any side effects thus far. NUHCS is the coordinating site for the Singapore trial. The heart centre is still recruiting patients, who must fulfil the following criteria: About the National University Heart Centre, Singapore (NUHCS) The National University Heart Centre, Singapore (NUHCS) is an academic, national specialist centre under the National University Health System (NUHS). NUHCS brings together the resources, expertise and capabilities in the areas of Cardiology, Cardiothoracic and Vascular Surgery to better meet the needs of the growing number of patients with heart disease and raise the future generation of medical professionals. As one of two national heart centres in Singapore for the treatment and management of complex cardiovascular diseases, NUHCS offers six core clinical programmes including Heart Failure & Cardiomyopathy, Structural Heart Disease, Acute Coronary Syndrome, Heart Rhythm, Congenital & Structural Heart Disease and Women's Heart Health. The centre has been awarded two institutional Peaks of Excellence for its Minimally-invasive Cardiothoracic Surgery and Aortic Centre Programme, and has been ranked top in Singapore for three consecutive years in 2022, 2023 and 2024 for the specialty of Cardiac Surgery in Newsweek's "World's Best Hospital" Award. Comprising a team of internationally-recognised cardiologists and surgeons from the cardiothoracic and vascular specialties, NUHCS serves as a referral national centre for cardiothoracic and vascular conditions and provides a comprehensive approach to the treatment of these patients. The holistic patient-care approach is backed by leading translational research at the Cardiovascular Research Institute (CVRI) and Cardiovascular Metabolic Translational Program, all of which complements these advanced quaternary clinical services to deliver state-of-the-art treatment solutions to the most challenging heart, lung and circulatory diseases.
The Print
14-05-2025
- Health
- The Print
A new hope for patients suffering from potentially fatal rare heart disease: NUHCS is first in Asia to recruit patients for landmark gene editing clinical trial
Singapore, May 14: While receiving treatment for a car accident ten years ago, Mr Chua learnt that he had an abnormal build-up of protein in his heart vessels. This discovery led to the diagnosis of a condition known as transthyretin amyloid cardiomyopathy (ATTR-CM), a rare disease currently affecting approximately 150 patients in Singapore. The international study explores gene editing therapy to stop the production of proteins causing the disease in ATTR-CM patients. ATTR-CM is caused by the build-up of misfolded, deformed transthyretin proteins in the heart, nerves and other organs due to genetic mutation or ageing. Symptoms of this potentially fatal rare disease are often vague and may include numbness in the hands and feet, lethargy and dizziness. If not diagnosed and treated promptly, ATTR-CM can lead to heart failure. Over time, Mr Chua's hands and legs became stiff, and the once active 62-year-old could no longer walk. The subsequent years following his diagnosis were riddled with frequent visits to the hospital due to episodes of heart failure and multiple injuries from falls resulting from nerve issues caused by the disease. Despite being on years of medication, Mr Chua's condition had continued to decline, and his growing need for assistance with simple everyday tasks had greatly affected his spirit. There is currently no cure for this debilitating illness. A new international clinical trial may be set to change the trajectory of this disease for patients suffering from ATTR-CM – including Mr Chua. The MAGNITUDE study involves a single-dose gene editing therapy administered intravenously that will alter the patient's DNA, slowing down the production of the abnormal protein that causes the disease. Novel treatment offers new hope for patients Assistant Professor Lin Weiqin, Clinical Director of the Heart Failure and Cardiomyopathy Programme at the National University Heart Centre, Singapore (NUHCS), is leading the Singapore arm of this trial. He shared that the double-blind study will investigate the impact of the gene editing research medicine Nexiguran Ziclumeran (nex-z, also known as NTLA-2001) on ATTR-CM. 'Gene editing therapy has been approved in other countries, for use in some neuro-muscular conditions, cancers and inherited blood disorders. If this trial is successful, it will be the first DNA altering treatment used in the field of adult cardiology and offers new hope to patients living with ATTR-CM,' explained Asst Prof Lin, who is also a Senior Consultant at NUHCS. In Asia, the first patient of the double-blind clinical trial was recruited in Singapore and received his infusion in September 2024 at NUHCS. Since then, four more patients have been involved in the study, including Mr Chua, who was the fifth and most recent participant. The gene editing therapy is administered alongside the standard treatment for ATTR-CM as part of the study. While it may take another two to three years to see the outcomes of this study, early results of the trial medication in early phase clinical trials have shown promising results, with 'consistent, rapid, and durable reductions' in the abnormal protein that causes ATTR-CM, and minimal side effects.[1] The patients in Singapore have also not reported any side effects thus far. NUHCS is the coordinating site for the Singapore trial. The heart centre is still recruiting patients, who must fulfil the following criteria: * Between 18 to 90 years old * Diagnosed with heart failure due to ATTR * On medication for heart failure and have experienced heart failure in the past year * For females, they are only eligible if they can no longer have children. About the National University Heart Centre, Singapore (NUHCS) The National University Heart Centre, Singapore (NUHCS) is an academic, national specialist centre under the National University Health System (NUHS). NUHCS brings together the resources, expertise and capabilities in the areas of Cardiology, Cardiothoracic and Vascular Surgery to better meet the needs of the growing number of patients with heart disease and raise the future generation of medical professionals. As one of two national heart centres in Singapore for the treatment and management of complex cardiovascular diseases, NUHCS offers six core clinical programmes including Heart Failure & Cardiomyopathy, Structural Heart Disease, Acute Coronary Syndrome, Heart Rhythm, Congenital & Structural Heart Disease and Women's Heart Health. The centre has been awarded two institutional Peaks of Excellence for its Minimally-invasive Cardiothoracic Surgery and Aortic Centre Programme, and has been ranked top in Singapore for three consecutive years in 2022, 2023 and 2024 for the specialty of Cardiac Surgery in Newsweek's 'World's Best Hospital' Award. Comprising a team of internationally-recognised cardiologists and surgeons from the cardiothoracic and vascular specialties, NUHCS serves as a referral national centre for cardiothoracic and vascular conditions and provides a comprehensive approach to the treatment of these patients. The holistic patient-care approach is backed by leading translational research at the Cardiovascular Research Institute (CVRI) and Cardiovascular Metabolic Translational Program, all of which complements these advanced quaternary clinical services to deliver state-of-the-art treatment solutions to the most challenging heart, lung and circulatory diseases. NUHCS services span across four locations to serve the western and central locations in Singapore: * NUHCS at National University Hospital (NUH), Kent Ridge – Main Operations * NUHCS Heart Clinic @ Ng Teng Fong General Hospital (NTFGH) * NUHCS Heart Clinic @ Jurong Medical Centre (JMC) * NUHCS Heart Clinic @ Alexandra Hospital (AH) For more information, visit: [1] Fontana M, Solomon SD, Kachadourian J, Walsh L, Rocha R, Lebwohl D, Smith D, Taubel J, Gane EJ, Pilebro B, Adams D, Razvi Y, Olbertz J, Haagensen A, Zhu P, Xu Y, Leung A, Sonderfan A, Gutstein DE, Gillmore JD. CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. N Engl J Med. 2024 Dec 12;391(23):2231-2241. doi: 10.1056/NEJMoa2412309. Epub 2024 Nov 16. PMID: 39555828. (ADVERTORIAL DISCLAIMER: The above press release has been provided by PRNewswire. ANI will not be responsible in any way for the content of the same) This story is auto-generated from a syndicated feed. ThePrint holds no responsibility for its content.
Straits Times
14-05-2025
- Health
- Straits Times
New drug based on gene-editing tech could treat rare, potentially fatal heart disease
Mr Chua Ah Hai (2nd from left), the most recent participant in the Singapore arm of the trial, with (from left) Prof Lin Weiqin, Dr Kay Ng and clinical research coordinator Nur Faezah Md Fadzillah. ST PHOTO: AZMI ATHNI SINGAPORE – A new drug based on gene-editing technology could soon provide respite to patients suffering from a rare and potentially fatal genetic heart disease. Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare disease of the heart muscle that involves deformed transthyretin (TTR) proteins building up in the heart, nerves and other organs. As a result of the protein build-up, the walls of the heart become thickened and stiff. The heart cannot then relax properly and get filled with blood, and it cannot pump it efficiently to the rest of the body. The symptoms of ATTR-CM can be vague and may include numbness in the hands and feet, lethargy and dizziness ; but left undiagnosed and not treated promptly, it could lead to heart failure and death. The disease affects about 150 people in Singapore, but Assistant Professor Lin Weiqin – clinical director for the heart failure and cardiomyopathy programme at the National University Heart Centre, Singapore (NUHCS) – believes the number could be much higher. This is because those affected often dismiss the symptoms – including swelling in the legs, numbness in the hands and unsteady walking – as normal consequences of ageing, Prof Lin said, noting that such symptoms can also be attributed to other conditions. 'Not many patients know about this condition, and not many doctors can diagnose this condition accurately as well,' he said, noting that NUHCS first set up a registry for ATTR-CM patients in 2019. Nexiguran Ziclumeran, a new drug that is also known as nex-z or NTLA-2001, seeks out and removes a portion of the TTR gene, slowing down the production of the disease-causing protein. It is based on Crispr-Cas9 technology, the Nobel Prize-winning gene-editing tool which has been used to alter DNA sequences. US biotechnology firm Intellia Therapeutics, which developed nex-z, is now conducting a trial involving 765 patients with ATTR-CM and a history of heart failure worldwide to evaluate its efficacy. Recruitment for the double-blind trial – where neither the participants nor the researchers know who received the actual treatment and who received a placebo – in Singapore began in 2024. Eight patients have already been recruited from NUHCS and the National Heart Centre Singapor e, and Prof Lin, who is leading the study here, said they hope to recruit a total of 14 patients by end-2025 . Prof Lin noted gene editing therapy has already been approved in other countries for treating certain neuromuscular conditions, cancers and inherited blood disorders. 'If this trial is successful, it will be the first DNA-altering treatment used in the field of adult cardiology and offers new hope to patients living with ATTR-CM,' he said. Patients in Singapore who wish to take part in the trial must be aged between 18 and 90, and diagnosed with heart failure due to ATTR. They must also be on medication for heart failure and have experienced heart failure in the past year. As a precaution, only women who can no longer have children are eligible to participate in the trial, as the edited genes could be passed down, said Prof Lin. One of those recruited is Mr Chua Ah Hai, who began experiencing numbness and weakness in his hands following a car accident in 2018. Doctors originally diagnosed Mr Chua, who was working as a baker then, with carpal tunnel syndrome – a common condition among those who work with their hands. An abnormal heart scan, however, suggested a deeper problem, and a heart biopsy confirmed that the father of two had ATTR-CM. 'I couldn't carry heavy things after my condition deteriorated. I stopped work in March 2024, as my employers encouraged me to rest,' said the 61-year-old, noting he often fell down and sustained fractures as a result of his condition. He took part in the trial as he believed it could benefit his condition. 'I wanted to give myself a new hope, a new lease of life,' he said. Mr Chua's son and daughter, aged 27 and 26, were tested and found to be positive for the gene mutation that causes the disease, though they have shown no symptoms and have been asked to monitor their conditions. A parent with the gene mutation that causes familial ATTR-CM has a 50 per cent chance of passing it to each of his or her children, according to the Cleveland Clinic; but not every child who inherits this gene mutation develops ATTR-CM. Though the outcomes of the study will only be known in about two to three years, early phase clinical trials – whose findings were published in the New England Journal of Medicine in November 2024 – showed 'consistent, rapid, and durable reductions' in the abnormal protein that causes ATTR-CM, with minimal side effects. Patients enrolled in the trial here have also not reported any side effects, Prof Lin noted. Another trial will begin to study the effectiveness of nex-z on patients with transthyretin amyloid neuropathy, where the accumulation of TTR proteins results in nerve damage. The National University Hospital (NUH) and the Singapore General Hospital are recruiting patients for this, with Dr Kay Ng – a senior consultant at NUH's neurology division – leading recruitment. Should the trials prove successful, those who initially received a placebo will also receive a free dose of nex-z, said Prof Lin, adding that he expects the drug to become commercially available by the end of the decade. Zhaki Abdullah is a correspondent at The Straits Times. He is on the health beat, in addition to occasionally covering science, environmental, tech and Muslim affairs issues. Join ST's WhatsApp Channel and get the latest news and must-reads.
Yahoo
14-05-2025
- Health
- Yahoo
A NEW HOPE FOR PATIENTS SUFFERING FROM POTENTIALLY FATAL RARE HEART DISEASE: NUHCS IS FIRST IN ASIA TO RECRUIT PATIENTS FOR LANDMARK GENE EDITING CLINICAL TRIAL
The international study explores gene editing therapy to stop the production of proteins causing the disease in ATTR-CM patients SINGAPORE, May 14, 2025 /PRNewswire/ -- While receiving treatment for a car accident ten years ago, Mr Chua learnt that he had an abnormal build-up of protein in his heart vessels. This discovery led to the diagnosis of a condition known as transthyretin amyloid cardiomyopathy (ATTR-CM), a rare disease currently affecting approximately 150 patients in Singapore. ATTR-CM is caused by the build-up of misfolded, deformed transthyretin proteins in the heart, nerves and other organs due to genetic mutation or ageing. Symptoms of this potentially fatal rare disease are often vague and may include numbness in the hands and feet, lethargy and dizziness. If not diagnosed and treated promptly, ATTR-CM can lead to heart failure. Over time, Mr Chua's hands and legs became stiff, and the once active 62-year-old could no longer walk. The subsequent years following his diagnosis were riddled with frequent visits to the hospital due to episodes of heart failure and multiple injuries from falls resulting from nerve issues caused by the disease. Despite being on years of medication, Mr Chua's condition had continued to decline, and his growing need for assistance with simple everyday tasks had greatly affected his spirit. There is currently no cure for this debilitating illness. A new international clinical trial may be set to change the trajectory of this disease for patients suffering from ATTR-CM – including Mr Chua. The MAGNITUDE study involves a single-dose gene editing therapy administered intravenously that will alter the patient's DNA, slowing down the production of the abnormal protein that causes the disease. Novel treatment offers new hope for patients Assistant Professor Lin Weiqin, Clinical Director of the Heart Failure and Cardiomyopathy Programme at the National University Heart Centre, Singapore (NUHCS), is leading the Singapore arm of this trial. He shared that the double-blind study will investigate the impact of the gene editing research medicine Nexiguran Ziclumeran (nex-z, also known as NTLA-2001) on ATTR-CM. "Gene editing therapy has been approved in other countries, for use in some neuro-muscular conditions, cancers and inherited blood disorders. If this trial is successful, it will be the first DNA altering treatment used in the field of adult cardiology and offers new hope to patients living with ATTR-CM," explained Asst Prof Lin, who is also a Senior Consultant at NUHCS. In Asia, the first patient of the double-blind clinical trial was recruited in Singapore and received his infusion in September 2024 at NUHCS. Since then, four more patients have been involved in the study, including Mr Chua, who was the fifth and most recent participant. The gene editing therapy is administered alongside the standard treatment for ATTR-CM as part of the study. While it may take another two to three years to see the outcomes of this study, early results of the trial medication in early phase clinical trials have shown promising results, with "consistent, rapid, and durable reductions" in the abnormal protein that causes ATTR-CM, and minimal side effects.[1] The patients in Singapore have also not reported any side effects thus far. NUHCS is the coordinating site for the Singapore trial. The heart centre is still recruiting patients, who must fulfil the following criteria: Between 18 to 90 years old Diagnosed with heart failure due to ATTR On medication for heart failure and have experienced heart failure in the past year For females, they are only eligible if they can no longer have children. About the National University Heart Centre, Singapore (NUHCS) The National University Heart Centre, Singapore (NUHCS) is an academic, national specialist centre under the National University Health System (NUHS). NUHCS brings together the resources, expertise and capabilities in the areas of Cardiology, Cardiothoracic and Vascular Surgery to better meet the needs of the growing number of patients with heart disease and raise the future generation of medical professionals. As one of two national heart centres in Singapore for the treatment and management of complex cardiovascular diseases, NUHCS offers six core clinical programmes including Heart Failure & Cardiomyopathy, Structural Heart Disease, Acute Coronary Syndrome, Heart Rhythm, Congenital & Structural Heart Disease and Women's Heart Health. The centre has been awarded two institutional Peaks of Excellence for its Minimally-invasive Cardiothoracic Surgery and Aortic Centre Programme, and has been ranked top in Singapore for three consecutive years in 2022, 2023 and 2024 for the specialty of Cardiac Surgery in Newsweek's "World's Best Hospital" Award. Comprising a team of internationally-recognised cardiologists and surgeons from the cardiothoracic and vascular specialties, NUHCS serves as a referral national centre for cardiothoracic and vascular conditions and provides a comprehensive approach to the treatment of these patients. The holistic patient-care approach is backed by leading translational research at the Cardiovascular Research Institute (CVRI) and Cardiovascular Metabolic Translational Program, all of which complements these advanced quaternary clinical services to deliver state-of-the-art treatment solutions to the most challenging heart, lung and circulatory diseases. NUHCS services span across four locations to serve the western and central locations in Singapore: NUHCS at National University Hospital (NUH), Kent Ridge - Main Operations NUHCS Heart Clinic @ Ng Teng Fong General Hospital (NTFGH) NUHCS Heart Clinic @ Jurong Medical Centre (JMC) NUHCS Heart Clinic @ Alexandra Hospital (AH) For more information, visit: [1] Fontana M, Solomon SD, Kachadourian J, Walsh L, Rocha R, Lebwohl D, Smith D, Täubel J, Gane EJ, Pilebro B, Adams D, Razvi Y, Olbertz J, Haagensen A, Zhu P, Xu Y, Leung A, Sonderfan A, Gutstein DE, Gillmore JD. CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. N Engl J Med. 2024 Dec 12;391(23):2231-2241. doi: 10.1056/NEJMoa2412309. Epub 2024 Nov 16. PMID: 39555828. View original content to download multimedia: SOURCE National University Heart Centre, Singapore (NUHCS)
