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合作寻找治愈方法

一种罕见癌症的药物治疗多年来一直困扰着研究人员. David Drewry’s work at the 北卡罗来纳大学埃舍曼药学院 has scientists and survivors optimistic that big breakthroughs are within reach.

大卫·德鲁里站在外面.
David Drewry. (摄影:Jeyhoun Allebaugh)

At the age of 12, 就在几个月前,他死于一种叫做脊索瘤的罕见癌症, Justin Straus在国际脊索瘤研究研讨会上发表了演讲. 穿着白色t恤坐在桌子旁, 他的头发因为最近的化疗而变短了, 贾斯汀请求脊索瘤研究人员:一起努力.

斯特劳斯在2008年的那次会议上说:“如果我们通力合作,我们就能走得更远。. “我认为,如果医生们开始互相分享他们的进展, 这样进步就会来得更快.”

2002年,7岁的贾斯汀被诊断出患有脊索瘤, 治疗方法是有限的, 今天仍然如此. 大多数病人接受手术和放疗, 但脊索瘤通常形成于脑干和脊髓附近, 给外科医生留下很小的余地来切除周围组织,以减少复发的机会. 复发的脊索瘤很少能治愈, 如果进一步的手术和放射治疗无效,也没有药物治疗.

多年来,科学家们一直在寻找治疗脊索瘤的药物 北卡罗来纳大学埃舍曼药学院 希望重大突破即将到来. 研究人员正在寻找一种有前途的新药物靶标, it turns out that Justin’s call for collaboration represented much more than a flash of youthful idealism. 事实上,它为那些在脊索瘤治疗方面取得进展的科学家提供了剧本.

“非常有前途的研究”

The observant gaze of his wife of 40 years led to an early chordoma diagnosis for John Watson ’77. 华生在刷牙的时候,他的妻子, 贝齐·布莱克威尔77年, told him that his left eye was not tracking properly with his right eye and that he should see an ophthalmologist.

“她一直在看着我,”沃森打趣道.

他去看了眼科医生后,又预约了更多的医生来做最后的诊断. 最后,核磁共振成像显示沃森大脑底部有一个肿瘤,医生鉴定为脊索瘤. “这让我很震惊,因为我根本就没有头痛, no fatigue, 我看不出有任何症状,” Watson said.

向专家咨询, 沃森和布莱克威尔, 谁住在教堂山, quickly decided that Watson would undergo surgery in November of 2020 at UNC Hospitals to remove the tumor.

手术成功后, 医生建议用质子束放射治疗以减少复发的机会. 北最正规外围足球网站州没有提供这种治疗的中心, 所以华生和布莱克威尔暂时搬到了华盛顿, D.C.他在那里的一家医院接受了每周5天的治疗.

沃森出现癌症, 但他对整个治疗方案的难度感到震惊, from the grueling surgery and recovery to the demands of undergoing radiation therapy so far from home. 他和布莱克威尔会见了乔希·萨默斯, 他是脊索瘤基金会的执行董事,也是一名脊索瘤幸存者, for guidance on how they could support research into new drug therapies that would offer more options for chordoma patients.

“我们在手术和放疗的另一边说, ‘We would really like to do something to push research in a direction that would be less intrusive,” Watson shared. “Josh said, “你这么说很有趣,因为北卡罗来纳大学正在进行非常有前途的研究.“我们都是毕业生,并致力于大学, 了解到这里发生的一切真是令人震惊.”

从个人到职业

贾斯汀·施特劳斯在2008年发表脊索瘤研究研讨会演讲的几年前. 大卫·德鲁里知道贾斯汀的故事和他与脊索瘤的斗争. Drewry was a chemist and researcher at the pharmaceutical company GlaxoSmithKline in North Carolina before he joined the 北卡罗来纳大学埃舍曼药学院 as an associate professor in 2017. 他的女儿是贾斯汀在达勒姆学院的同学, 达勒姆的一所私立学校, North Carolina, 德鲁里回忆自己曾试图帮助孩子理解她的朋友正在经历的事情.

“贾斯汀的家人对此非常开放,这是孩子们经历的一部分,”德鲁里说. “有时贾斯汀在上课,有时他的治疗不是基于他的治疗. 他会和他的朋友们谈论这件事, and we would talk about it with our daughter to help her with this difficult and hard-to-understand thing.”

Supporting Justin through the rigors of his illness became a cause for the close-knit Durham Academy community, 2008年贾斯汀去世后, 他的同学为脊索瘤研究举办募捐活动. 德鲁里收藏了这些活动的t恤, including one with the word perseverance written across the front — the word Justin managed to write across a whiteboard as a final message to his family and loved ones shortly before he died.

在一次募捐活动中,德鲁里遇到了脊索瘤基金会的索默. 当时,德鲁里正在葛兰素史克研究用于治疗其他类型癌症的化合物. His conversations with Sommer and his daughter’s experience led Drewry to think about what he could do as a researcher.

德鲁里说:“这对我女儿来说非常重要. “对我来说,买一件t恤是一件很容易的事. It seemed more tangible to me as a scientist to put my training and expertise in play to make a long-term contribution.”

德鲁里赞同贾斯汀的观点,认为研究人员应该更加合作. At GSK, Drewry had helped start an open science project that facilitated easier and more routine sharing of compounds that GSK had developed with academic institutions and labs around the world.

“We realized that it would be much more powerful for the scientific community and patients if we could send our work to someone studying malaria or a cancer like chordoma that wasn’t our focus.” Drewry said. “我们成功地在GSK实施了这一点,并开始广泛分享. 我希望脊索瘤研究成为其中的一部分.”

索默把德鲁里介绍给了一位在伦敦大学学院研究脊索瘤的病理学家. Drewry sent her compounds developed at GSK that she was then able to test in chordoma cell lines supplied by the Chordoma Foundation. 她的研究显示出很有希望的结果,并最终发表.

德鲁里说:“那是我第一次觉得自己真的可以做出贡献。. “I’ve held onto what Justin said about persevering and collaborating because discovering new drugs is an extremely hard job. It’s such a complicated path from an idea to a medicine that you need lots of people working together on it. 贾斯汀告诉我们,如果我们合作,我们会走得更远,这是绝对正确的.”

绑定的突破

德鲁里在伦敦大学学院的合作者. Adrienne Flanagan, had discovered a critical link between chordoma and a protein called brachyury. 在胚胎发育期间,brachyury在身体中是活跃的,然后在大多数人体内关闭. 弗拉纳根的研究表明,brachyury在脊索瘤患者中仍然活跃, 弗拉纳根将这个灵光一现的时刻描述为脊索瘤的“盔甲上的裂缝”.

Further research revealed that almost all chordoma patients have an inherited alteration in at least one copy of the brachyury gene, 进一步证实该蛋白为药物开发提供了一个有希望的靶点.

在北卡罗来纳大学埃舍曼药学院, Drewry began developing molecules that he hoped could bind to the brachyury protein and block its function. 他得到了牛津大学拥有x射线晶体学专业知识的科学家的帮助, 这样德鲁里就能知道是否有化合物附着在靶标上了. 在x光照射的1000种化合物中,近50种与brachyury相关.

作为一名药物化学家, 这对我来说是一个顿悟的时刻, 因为现在我可以把这些化合物变得更好,” Drewry said.

The research team has now identified several compounds that bind to brachyury and appear to disrupt its functioning, 德鲁里正在研究因果关系. “That’s the next big thing we need to work on is understanding that the effect we see in a chordoma cell is due to this molecule binding to brachyury,” Drewry said. “这将是推动该领域向前发展的下一个科学挑战.”

还有一种可能性,德鲁里的工作将应用于脊索瘤以外的治疗. Data collected through the years from cancer patients have shown that many aggressive cancer cases are linked to the presence of the brachyury protein. Compounds that Drewry develops could be used as tools to explore brachyury’s role in a variety of cancer types, 也可以作为药物的模板,有朝一日这些药物可能代表有效的治疗方法.

靶向brachyury蛋白是抗癌药物研究的新策略, 使私人慈善事业成为德鲁里工作的重要资金来源. 约翰·沃森和贝特西·布莱克威尔承诺捐出50美元,来支持德鲁里的实验室. The funding will help Drewry continue his research until further grants from the federal government and other funders are secured.

“他们正试图解开我们未知的谜团,”沃森说. “他们正在进行一场非常重要的知识开发之旅. 他们还没到那一步,我们想帮忙.”

脊索瘤基金会已经奖励了超过1美元.给德鲁里的研究拨款五百万, 索默用了一个词来描述德鲁里的作品给他的感觉:希望.

“我们现在真的有理由希望能解决这个问题,”Josh说. “这是知道我们正处于重大最正规外围足球网站的前沿. 这就是为什么支持大卫的工作很重要.”

支持卡的研究人员

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