Resolving quality, standards and productivity related challenges of a ground breaking regenerative therapy for heart failure

解决心力衰竭的突破性再生疗法的质量、标准和生产力相关的挑战

• 批准号: 10073371
• 金额: $ 4.78万
• 依托单位: HEART CELLS COMPANY LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2023
• 资助国家: 英国
• 起止时间: 2023 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10073371
• 关键词: Resolving; quality; standards; productivity; related

Heart failure is when the heart cannot efficiently pump blood around the body due to damage to the heart muscle. As a result, important organs (such as the brain and lungs) do not get enough blood and patients suffer from tiredness and breathlessness and are unable to exercise. As the heart failure progresses, patients experience an ever-decreasing quality of life and an increased dependence on hospital services. The number of heart failure patients continues to increase and, despite new treatments, many remain symptomatic as current treatments do not repair the heart muscle (thereby enabling it to pump blood efficiently again). For these patients, the only way to alleviate these symptoms is a very rare, and highly invasive, heart transplant.The applicants originate from the world-renowned Barts Heart Centre, where this team has developed and tested a cell therapy which uses stem cells taken from the patient's own bone marrow. Once processed, these cells are infused into the heart to help repair the heart muscle (thereby enabling it to pump blood more effectively) and improve the patient's symptoms. A charity, the Heart Cells Foundation, was formed to promote the adoption of cell therapy for heart failure. This charity raises hundreds of thousands of pounds annually, and has created a Compassionate Treatment Unit. The team has treated over 500 heart failure patients, who were told they had no more treatment options, with their own stem cells.The Heart Cells Foundation wishes to commercialise this treatment so it is more widely available nationally and internationally. To lead this commercial step, they formed the Heart Cells Company (HCC). HCC needs approval from regulatory agencies in order to secure the investment needed for a large trial (which could lead to market authorisation - required before the NHS will adopt the treatment). However, regulatory agencies are unwilling to grant HCC approval until it has a more definitive measurement to assess the quality of the cell product.HCC is seeking support from the Analysis for Innovators (A4I) competition to develop this measurement technique (commonly called an assay) to assess the cell product's efficiency and quality. This assay will also enable HCC to improve and research the treatment method to make it more effective in the future.This A4I project will develop this efficiency and quality assay by analysing samples of bone marrow taken from heart failure patients participating in ongoing clinical trials at Barts Heart Centre.

心力衰竭是指由于心肌损伤，心脏不能有效地将血液泵送到全身。因此，重要器官（如大脑和肺）得不到足够的血液，患者会感到疲劳和呼吸困难，无法锻炼。随着心力衰竭的进展，患者的生活质量不断下降，对医院服务的依赖性增加。心力衰竭患者的数量持续增加，尽管有新的治疗方法，但许多患者仍然有症状，因为目前的治疗方法不能修复心肌（从而使其能够再次有效地泵血）。对于这些患者来说，缓解这些症状的唯一方法是进行非常罕见且高度侵入性的心脏移植。申请者来自世界著名的Barts心脏中心，该团队在那里开发并测试了一种细胞疗法，该疗法使用从患者自身骨髓中提取的干细胞。一旦被处理，这些细胞被注入心脏，以帮助修复心肌（从而使其能够更有效地泵血）并改善患者的症状。一个慈善机构，心脏细胞基金会，成立了促进采用细胞疗法治疗心力衰竭。该慈善机构每年筹集数十万英镑，并创建了一个Compounds治疗单位。该团队已经用自己的干细胞治疗了500多名心力衰竭患者，这些患者被告知他们没有更多的治疗选择。心脏细胞基金会希望将这种治疗商业化，以便在国内和国际上更广泛地使用。为了领导这一商业步骤，他们成立了心脏细胞公司（HCC）。HCC需要得到监管机构的批准，以确保大型试验所需的投资（这可能导致市场授权-在NHS采用该治疗方法之前需要）。然而，监管机构不愿意批准HCC，直到它有一个更明确的测量来评估细胞产品的质量。HCC正在寻求创新者分析（A4 I）竞争的支持，以开发这种测量技术（通常称为分析）来评估细胞产品的效率和质量。这项检测也将使HCC能够改进和研究治疗方法，使其在未来更加有效。这个A4 I项目将通过分析来自Barts心脏中心正在进行的临床试验的心力衰竭患者的骨髓样本来开发这种效率和质量检测。