Cheeran Binith, Cohen Leonardo, Dobkin Bruce, Ford Gary, Greenwood Richard, Howard David, Husain Masud, Macleod Malcolm, Nudo Randolph, Rothwell John, Rudd Anthony, Teo James, Ward Nicholas, Wolf Steven
Sobell Department of Motor Neuroscience, Institute of Neurology, London, United Kingdom.
Neurorehabil Neural Repair. 2009 Feb;23(2):97-107. doi: 10.1177/1545968308326636.
Major advances during the past 50 years highlight the immense potential for restoration of function after neural injury, even in the damaged adult human brain. Yet, the translation of these advances into clinically useful treatments is painstakingly slow.
Here, we consider why the traditional model of a "translational research pipeline" that transforms basic science into novel clinical practice has failed to improve rehabilitation practice for people after stroke.
We find that (1) most treatments trialed in vitro and in animal models have not yet resulted in obviously useful functional gains in patients; (2) most clinical trials of restorative treatments after stroke have been limited to small-scale studies; (3) patient recruitment for larger clinical trials is difficult; (4) the determinants of patient outcomes and what patients want remain complex and ill-defined, so that basic scientists have no clear view of the clinical importance of the problems that they are addressing; (5) research in academic neuroscience centers is poorly integrated with practice in front-line hospitals and the community, where the majority of patients are treated; and (6) partnership with both industry stakeholders and patient pressure groups is poorly developed, at least in the United Kingdom where research in the translational restorative neurosciences in stroke depends on public sector research funds and private charities.
We argue that interaction between patients, front-line clinicians, and clinical and basic scientists is essential so that they can explore their different priorities, skills, and concerns. These interactions can be facilitated by funding research consortia that include basic and clinical scientists, clinicians and patient/carer representatives with funds targeted at those impairments that are major determinants of patient and carer outcomes. Consortia would be instrumental in developing a lexicon of common methods, standardized outcome measures, data sharing and long-term goals. Interactions of this sort would create a research-friendly, rather than only target-led, culture in front-line stroke rehabilitation services.
过去50年的重大进展凸显了神经损伤后功能恢复的巨大潜力,即使是在受损的成人大脑中。然而,将这些进展转化为临床可用的治疗方法的过程却极其缓慢。
在此,我们探讨为何将基础科学转化为新型临床实践的传统“转化研究管道”模式未能改善中风患者的康复治疗。
我们发现:(1)在体外和动物模型中试验的大多数治疗方法尚未在患者身上带来明显有益的功能改善;(2)中风后恢复性治疗的大多数临床试验仅限于小规模研究;(3)招募患者参加更大规模的临床试验很困难;(4)患者预后的决定因素以及患者的需求仍然复杂且定义不明确,因此基础科学家对他们所研究问题的临床重要性没有清晰的认识;(5)学术神经科学中心的研究与大多数患者接受治疗的一线医院及社区的实践联系不佳;(6)与行业利益相关者和患者压力团体的合作发展不佳,至少在英国是这样,在英国,中风转化性恢复神经科学的研究依赖于公共部门研究资金和私人慈善机构。
我们认为患者、一线临床医生以及临床和基础科学家之间的互动至关重要,这样他们才能探讨各自不同的优先事项、技能和关注点。可以通过资助研究联盟来促进这些互动,该联盟包括基础和临床科学家、临床医生以及患者/护理人员代表,资金针对那些作为患者和护理人员预后主要决定因素的损伤。联盟将有助于制定通用方法词汇表、标准化结局测量、数据共享和长期目标。这种互动将在一线中风康复服务中营造一种有利于研究的文化,而不仅仅是以目标为导向的文化。