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

心力衰竭是指由于心肌受损，心脏不能有效地将血液输送到全身。因此，重要器官（如大脑和肺）得不到足够的血液，患者会感到疲倦和呼吸困难，无法运动。随着心力衰竭的进展，患者的生活质量不断下降，对医院服务的依赖程度越来越高。心力衰竭患者的数量继续增加，尽管有新的治疗方法，但许多人仍然有症状，因为目前的治疗方法不能修复心肌（从而使其能够有效地再次泵血）。对于这些病人来说，缓解这些症状的唯一方法是一种非常罕见的、高度侵入性的心脏移植手术。申请者来自世界著名的巴茨心脏中心，该团队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。一旦处理完毕，这些细胞就会被注入心脏，以帮助修复心肌（从而使其更有效地泵血）并改善患者的症状。一个名为“心脏细胞基金会”的慈善机构成立了，目的是促进采用细胞疗法治疗心力衰竭。这个慈善机构每年筹集数十万英镑，并建立了一个富有同情心的治疗单位。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方法商业化，以便在国内和国际上更广泛地使用。为了实现商业化，他们成立了心脏细胞公司（HCC）。HCC需要获得监管机构的批准，以确保大规模试验所需的投资（这可能导致市场授权——在NHS采用这种治疗之前）。然而，监管机构不愿意批准HCC，直到有更明确的测量来评估细胞产品的质量。HCC正在寻求创新分析（A4I）竞赛的支持，以开发这种测量技术（通常称为测定）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。该A4I项目将通过分析参与巴兹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的检测方法。

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.