美国CureJM基金会来信喽～带来三款潜在的JDM药物。

今天早上打开电脑发现来自美国的CureJM的执行董事 Jim Minow 先生发来邮件，邮件中提到了关于目前关注的三款潜在JDM药物的最新研究进展，以及有关肺炎疫情对JDM患儿对影响。

不仅如此，还公布下了一次Zoom的网络会议的时间，是在北京时间的11月02日（周一）早上09:00，感兴趣的小伙伴可以一起听听，主题主要有关以下三款药品的评价。

Executive Director Jim Minow

邮件正文内容如下：

Friends and family,

朋友们和家人们，

I have just finished assessing the package of opportunities that emerged from Cure JM’s first “New Drugs in Juvenile Myositis” symposium that took place earlier this month.  Quite simply, the consensus reaction was, “Wow!”

我刚刚完成了本月早些时候Cure JM举行的首次“幼年皮肌炎新药”研讨会的各项评估。结果一致的反应是，“哇！”效果很惊人。

There has never been a time where there has been more potential for improved treatments for JM than there is today.  If there is one takeaway from the conference, it is this:  we must seize the day and seize the opportunity to scientifically determine if any of the drugs presented at the symposium will help our kids.

从来没有一个时代比今天更有可能有那么多潜在的治疗方法来改善JM。如果说这次会议有什么收获的话，那就是：我们必须抓住时机，科学地确定在研讨会上提出的药物是否有助于我们的孩子。

The goal of the symposium was to inform pediatric rheumatologists and other attending doctors in JM care of new and better treatments that we hope will be available to them soon.  

研讨会的目的是告知儿童风湿病专家和其他JM治疗医生关注新的更好的治疗方法，我们希望他们能很快了解到这些治疗方法。

What we know so far is promising.

到目前为止我们知道的新药物是让人憧憬的。

Vamorolone:  I call vamorolone the “anti-steroid” steroid.  It is a drug that has all of the immunosuppressant benefits of prednisone and virtually none of the side effects.  I don’t need to tell you how big that could be for patients with JM.  Vamorolone is on track for clinical trial results in Duchenne muscular dystrophy, and trial results will be reviewed and hopefully approved by the FDA in 2021.

Vamorolone：我称Vamorolone为“温和激素”的糖皮质激素。它是一种具有泼尼松所有免疫抑制作用，但几乎没有任何副作用的药物，不用我来告诉你这对于JM病人来说意味着什么。Vamorolone正在进行杜氏肌营养不良症的临床试验，试验结果将在2021年得到FDA的审查并有希望获得批准。 

But will it work in JM?  , the CEO of the ReveraGen, the biomedical company that created vamorolone, presented an optimistic case that it will, and that we will need to have our own clinical trial in JM to prove its effectiveness

但它用来治疗JM行吗？生物医药公司Reveagen的首席执行官Eric Hoffman博士，提出了一个乐观的观点，认为它会成功，而且我们需要我们自己的JM临床试验来证明它的有效性。

Baricitinib:  Dr. Hanna Kim presented the findings of her four-patient clinical trial of baricitinib, a drug already approved by the FDA for other inflammatory diseases, most notably rheumatoid arthritis.  Did it work in JM? In a word:  YES.  Of the four JM patients enrolled in the trial, all four saw improvement in skin conditions, and the two patients who suffered muscle weakness also saw significant improvements in muscle strength during the trial.  Dr. Kim is now planning a second trial with 20 JM patients in an effort to replicate the results and create a larger validated data set that doctors can use in the future to prescribe baricitinib for use in JM.

Baricitinib：Hanna Kim博士介绍了试用Baricitinib进行的四名患者临床试验的结果。Baricitinib是一种已经被FDA批准用于治疗其他炎症性疾病，尤其是类风湿性关节炎的药物。它用来治疗JM行吗？答案是肯定的。在参与试验的4名JM患者中，4名患者的皮疹都有所改善，2名肌无力的患者在试验期间的肌力也有显著提高。Kim博士现在正计划对20名JM患者进行第二次试验，试图复制第一次的结果并创建一个更大的有效的数据集，以供将来医生给JM患者开baricitinib时参考。

Abatacept:  Dr. Rodolfo Curiel, the trial’s principal investigator and Director of the Cure JM Center of Excellence at George Washington University reported that nine of ten juvenile myositis patients have been enrolled in the trial, and eight of those nine have completed the study.  A previous abatacept trial in adult dermatomyositis reported that half of the patients who received the treatment met the primary outcome improvement measurements in activity and strength scores.  Dr. Curiel’s current trial in JM should be completed soon and the results of six core set measures will be published in late 2021.  This trial has been active for five years, and anecdotal information suggests that investigators are seeing efficacy in JM.

Abatacept：该试验的主要研究者、Cure JM在乔治华盛顿大学的卓越中心主任Rodolfo Curiel博士报告说，10名青少年肌炎患者中的9名已被纳入试验，其中8名已完成研究。先前的一项成人皮肌炎abatacept试验报告说，接受治疗的患者中有一半在活动和力量方面评分得到改善。Curiel博士目前在JM的试验将很快完成，六项核心指标的结果将于2021年底公布。这项试验已经进行了五年，并且轶事信息表明，研究人员正在观察JM的疗效。

COVID-19 and JM

COVID-19 和JM

Of course, I worry about the second and third waves of COVID-19.  We now know that children and young adults can contract the coronavirus and that younger children especially are not a protected class, but do seem to have milder symptoms.  Whether children with an underlying autoimmune disease are more at risk from COVID-19 is an open scientific question as there is scant data and limited studies.  Also, what we know about the disease itself is in constant change.

当然我担心第二波和第三波的新冠病毒。我们现在知道，儿童和年轻人可以感染新冠病毒，尤其是年幼的不受保护的儿童，但儿童似乎确实有较轻的症状。由于匮乏的数据和很有限的研究，患有潜在自身免疫疾病的儿童是否更容易感染新冠病毒是一个开放的科学问题。而且我们了解到新冠病毒肺炎本身也在不断的变化。

We now know, for example, that COVID-19 is more of a vascular disease than a respiratory disease, and that many COVID patients, even those with mild symptoms, may be so-called “long haulers.”  This is where COVID most likely has a long-term effect on the heart, the vascular system, and other organs.  JM is a vasculopathy, meaning that the vascular system is already under attack from an autoimmune response. Could contracting COVID exacerbate this condition?  We don’t know.

例如，我们现在知道，新冠状病毒肺炎相比较于呼吸系统疾病更像是一种血管疾病，许多感染者，即使症状轻微者，也可能是所谓的“长途跋涉者”。这是说病毒最有可能对心脏、血管系统和其他器官产生长远的影响。JM是一种血管病，意味着血管系统已经受到自身免疫反应的攻击。感染病毒会使JM病情恶化吗？我们目前不知道。

Preliminary data from the COVID-19 Global Rheumatology Alliance Provider Registry indicates that moderate and high-dose steroid use (more than 10 mg per day) is associated with a more severe COVID-19 disease course.  Other studies suggest that steroid use is helpful in treating the later stages of the disease, but harmful in the early stages when the body’s natural immune response is important to combat disease onset.   

来自COVID-19全球风湿病联盟提供商注册处的初步数据表明，中剂量和高剂量类固醇的使用（每天超过10毫克）与更严重的COVID-19疾病进程相关。其他研究表明，类固醇的使用有助于疾病后期的治疗，但在早期阶段有害，因为人体的自然免疫反应对对抗疾病的发生很重要。

The general consensus among pediatric rheumatologists is that if corticosteroid therapies are effective in managing JM they should not be discontinued.  The CDC’s position that immunocompromised individuals are at higher risk is unchanged.  As questions may arise in your own family, please consult with your physician.

儿科风湿病学家的普遍共识是，如果皮质类固醇治疗对JM有效，则不应停止治疗。疾病预防控制中心一致的观点是免疫功能受损的人处于更高的风险中。如有疑问，请咨询您的医生。

Cure JM’s Finacial Condition

Cure JM的财务状况

I am happy to report that, thanks to you, Cure JM weathered the first wave of the COVID-19 pandemic well and remains financially strong.  As you know, we have expanded our grant program in recent years and have created and funded two new JM Centers of Excellence to increase access to the highest quality of JM medical care and expertise.  We are also in the third year of funding a $650,000 national biorepository and medical registry of JM patients that will be instrumental to the research that will eventually lead us to the causes of—and cures for—juvenile myositis.

我很高兴地报告，多亏了你们，CureJM很好地经受住了COVID-19的第一波大流行，并且财政上仍然很强大。如您所知，我们近年来扩大了我们的补助金计划，并创建并资助了两个新的JM卓越中心，以增加获得最高质量的JM护理和专业知识的可能。我们也正处于资助一个65万美元的JM患者的国家生物存储和医疗登记的第三年，这将有助于我们最终找到JM病因和治疗方法的研究。

Despite the challenges presented by COVID-19, we were able to meet our grant obligations to fourteen research institutions and four Centers of Excellence in 2020.  Thank you for that.  We could not have accomplished this without you.

尽管COVID-19提出了挑战，我们还是能够在2020年履行对14个研究机构和4个卓越中心的资助义务。谢谢你们，没有你们的支持我们不可能完成这件事。

We were able to fund several “early-career” doctors and researchers who represent the next generation of expertise in research and clinical care of JM children and adults.  This next generation of doctors with JM expertise is settling into careers at children’s hospitals around the country and helping to advance our mission to improve access to high-quality JM care from coast-to-coast.

我们资助了几位处在“早期职业生涯”的医生和研究人员，他们代表了JM儿童和成人研究和临床护理方面的下一代专家。新一代具有JM专业知识的医生正在全国各地的儿童医院安顿下来，并帮助推进我们的使命，以改善全球各地JM获得更高质量护理的机会。

It is clear to me that 2021 holds the potential to be a most important and pivotal year in Cure JM’s history.  We simply must advance these three new drugs (and possibly others) down the “path of access” for JM.  The clinical trials we need to conduct will focus on safety and efficacy in JM, and if successful, would represent great leaps forward.

我很清楚，2021年有可能成为Cure JM历史上最重要和最关键的一年。我们只需推动这三种新药（可能还有其他药物）沿着JM的“通路”前进。我们需要进行的临床试验将专注于JM的安全性和有效性上，如果成功，将代表着巨大的飞跃。

This Year’s Giving Tuesday Holiday Challenge

今年的给力星期二假日挑战赛

We kick off the Giving Tuesday Holiday Challenge, Cure JM’s most important family fundraising program, on November 1.  If ever there was a year we needed you, this is it!  The work ahead of us in 2021 to develop and fund the clinical trials necessary for new and better treatments, to continue the basic research to understand the genetic and environmental causes of JM, and to improve access to quality care by training, and engaging more clinicians and hospitals around the country could not be more important.

11月1日，我们将开始“给力星期二”假日挑战赛，这是JM最重要的家庭筹款项目。如果说什么时候我们需要你，那就是现在！2021年，我们将要开展并资助新的更好的治疗方法所需的临床试验，继续进行基础研究以了解JM的遗传和环境因素，并通过培训改善获得优质护理的机会，以及让更多全国各地的临床医生和医院参与其中，这些工作再重要不过了。

Please consider joining us on the Zoom call this Sunday, November 1, at 8 PM Eastern Time.  You can register here.  Once you register, you’ll receive details on how to join the call.

请考虑加入我们的Zoom会议吧，这个星期天，11月1日，在美国东部时间晚上8点（北京时间11月2日 星期一 早上09:00）。你可以在这里注册。一旦注册完成，您将收到有关如何加入会议的详细信息。

With appreciation

致敬

Jim Minow

Cure JM Executive Director

Cure JM 执行董事

报名链接：点击注册 注：根据收到的邮件进行操作，会议当天保持会议安静，请关闭摄像头及麦克风，谢谢

扩展阅读：瑞士的ACTELION制药公司与REVERAGEN公司合作开发治疗DMD的药物VAMOROLONE（温和激素）

萌芽公益志愿者：红映 译 小欣 小维整理