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圣地亚哥生物技术公司Samumedrecently announcedthat it will be moving to phase 3 clinical trials of its drug lorecivivint (SM04690) for the treatment of knee osteoarthritis.

What is osteoarthritis?

骨关节炎是膝关节炎最常见的形式,也是导致成人残疾的主要原因。尤其是老年人。这种退化,“磨损性”关节炎的特点是关节软骨的破坏和骨骼的结构变化,which leads to pain,inflammation,以及关节功能和活动性的丧失。It occurs most often in people who are at least 50 years old,但也可能发生在年轻人身上。

During the progression of osteoarthritis,膝关节软骨逐渐磨损,磨损粗糙,骨骼之间的保护空间减小。This results in bone grinding on bone,会造成疼痛的骨刺。

一些估计表明大约有3000万成人患有骨关节炎,the majority of which is due to aging.这种状况剥夺了人们的生活质量和独立性,同时损害了工作场所的生产力,增加了医疗保健成本。The current treatment options for patients are very limited in nature,因为目前还没有批准的疾病改良疗法。

Moving to Phase 3

This phase 3 trial,known as STRIDES (SM04690 Trial Evaluating a Randomized Injection for Determination of Efficacy and Safety),will evaluate the ability of lorecivivint to ease the symptoms of knee osteoarthritis,重点关注患者结局和疾病进展的改善。

In the previous phase 2 trial,lorecivivint demonstrated a reduction in pain along with improvement of function and medial joint space width,a marker of disease progression,in patients with knee osteoarthritis.The data was promising and was enough to encourage the launch of this phase 3 trial.

We recently completed the investigator meeting for STRIDES X-ray and anticipate enrolling our first subjects in the near future.启动这项试验是洛雷西万特的一个重要里程碑,Samumedand potentially for the millions of patients with OA of the knee.We look forward to providing further updates on this study,以及其他的跨步测试,throughout 2019.

——尤素夫·亚齐兹,M.D.,Samumed首席医疗官

What is Lorecivivint?

Lorecivivint (SM04690) is a small-molecule inhibitor of the Wnt pathway,它调节细胞命运决定的关键方面,细胞迁移,cell polarity,神经模式,and organogenesis during embryonic development.

Samumed is focused on the part of the Wnt pathway that regulates the self-renewal and differentiation of adult stem cells and is critical for tissue maintenance and organ health.Wnt是一种重要的信号通路,它控制干细胞分化的时间,从而支持干细胞的形成。replenishment,and repair of all bodily tissues.

问题开始于Wnt通路功能紊乱,其精心调节的平衡失败,导致组织维护和修复不良,最常见的是the onset of disease in a particular tissue or organ.洛雷西文特靶向Wnt通路,以恢复平衡和恢复正常功能,使健康组织的维护可以再次恢复。

The company claims that its preclinical data suggests that lorecivivint affects joint health in three key ways: generation of new cartilage material,slowing of cartilage loss,减少炎症。

Conclusion

There are currently no approved effective therapies for osteoarthritis that modify disease progression,so a successful drug could improve the quality of life of many older people who suffer from this age-related disease.

至于Wnt通路是否是最佳途径,这是另一个问题;it is likely that a full repair approach,比如一个由衰老或SENS Research Foundation,may ultimately prove more effective;however,until such technologies are available,像洛雷西文特这样的药物至少代表了一个不久的将来的权宜之计,假设第三阶段进展顺利。If it does,then approval may not be far away.

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About the author

Steve Hill

作为科学作家和健康长寿的忠实倡导者,以及促进长寿的技术,Steve has provided the community with hundreds of educational articles,interviews,and podcasts,帮助公众更好地了解老龄化及其动态变化的方法。他的资料可以在H+杂志上找到,Longevity reporter,今日心理学与奇点博客。He is a co-author of the book "Aging Prevention for All" – a guide for the general public exploring evidence-based means to extend healthy life (in press).
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