比尔·盖茨:诊断阿尔茨海默症为什么这么难?我们如何能做得更好?
当我在去年秋天首次宣布自己将投资阿尔茨海默症的研究时,我想我知道该期待什么。我知道我将会更深入地与杰出的科学家和倡导者一起努力,阻止阿尔茨海默症的蔓延。对此,我从未失望过。我在过去七个月里看到的事,使我比以往任何时候都更加充满希望。
我从广大阿尔茨海默症群体那里得到了惊人的反馈,这是我没有预料到的。因为在发布声明之前,我的家人没有公开谈论过我父亲的诊断结果,我也从未体验过支持性群体有多么的了不起。你们中的很多人和我分享了你们的个人经历,通过线下和线上的方式(包括在我的博客和公众号上)。听到具有相同经历人的分享,这很有帮助。
阿尔茨海默症的研究是一个前沿领域,这可以极大地改善人类的生活,既包括病患的生活,又包括他们所爱之人的生活。我乐观地认为,如果我们在几个关键领域取得进展,就能实质性地改变阿尔茨海默症的发展进程。我们现在能做的最重要的事情之一,就是开发一个可靠的、价格可负担的、容易获得的诊断方法。
今天诊断阿尔茨海默症的过程并不理想。它从一个认知测试开始。如果你表现不佳,你的医生就需要排除所有其他可能导致记忆丧失的原因,比如中风或营养不良。然后你的医生可以通过脊椎穿刺或者PET扫描来确认你是否患上了阿尔茨海默症。尽管这些测试已相当准确,但唯一能确诊这种疾病的方法只有死后的尸检。
这个诊断过程有两大问题。首先,它可能很贵且具有侵入性。美国的大多数保险计划都不会报销阿尔茨海默症的诊断费用,病人通常得自己负担几千美元。与此同时,脊椎穿刺可能会让人感到害怕和不适,PET扫描则要求患者在40分钟内完全保持静止,这对任何人来说都很难做到,但对阿尔茨海默症的患者而言则尤为困难。
其次,只有患者开始出现认知能力下降的症状,他们才会接受检查。而我们对阿尔茨海默症了解得越多,就越能清楚地认识到,这种疾病的发生要早于我们之前的认知。研究表明,阿尔茨海默症在患者表现出症状的十多年前就开始损伤人的大脑。而这可能就是我们需要开始治疗的时机,以获得有效药物的最佳治疗效果。
在寻求科学突破的过程中,这种延迟是个大问题。目前,找到足够符合条件的患者来进行临床试验非常困难,因此招募试验参与者要比进行这项研究花费更长的时间。在我们能够减缓疾病的发展之前,我们需要一种更好的方法来诊断阿尔茨海默症,就像血液检查或眼科检查那样简单。
这有点像鸡和蛋的问题。如果没有一种成本更低、侵入性更小的方法来及早地诊断病人,就很难研发出一种革命性的新药物。但大多数人都不希望在没有治疗办法的情况下太早地了解自己是否患病。阿尔茨海默症诊断工具的商业市场基本上并不存在。前景光明的研究正在进行,可很少有公司在关注如何将这些研究转化为可用的产品。
因此,我接下来在阿尔茨海默症研究领域的投资将通过一个叫做“诊断法加速器”(Diagnostics Accelerator)的新基金进行。这个项目隶属于阿尔茨海默症药物发现基金会(ADDF),旨在促进产生大胆的新想法,用于更早、更好地诊断阿尔茨海默症。我与伦纳德·劳德(Leonard Lauder)、ADDF、杜比家族、查尔斯和海伦·施瓦布基金会(Helen Schwab Foundation)及其他捐赠者一起,投入超过3000万美元来启动“诊断法加速器”。
“诊断法加速器”是一种风险慈善工具,这意味着它不同于大多数基金项目。政府或慈善机构的投资在发展新想法和前沿研究方面发挥着很好的作用,但它们并不总是善于创造有用的产品,因为没有人会最终赢利。另一方面,风险投资更有可能开发出一种能够真正被病人使用的测试,但其金融模型更青睐于为投资者带来巨额回报的项目。
风险慈善是二者的折中。它鼓励大胆、冒险的研究方法,最终目的是为真正的病人开发真正的产品。如果“诊断法加速器”支持的任何一个项目获得成功,我们意外获得的经济回报将被重新投入到资金池中。
我希望这项投资能搭起一座桥,将学术研究和可靠的、价格可负担的、容易获得的诊断方法联结起来。我预计会有很多新玩家加入,他们有创新的想法,但之前可能没有资源去探索这些想法。如果你认为自己就是这样一个敢于思考的人,我们想听到你的好主意。我鼓励你在“诊断法加速器”的网站上申请资金(点击文末“阅读原文”)。
想象一个这样的世界:诊断阿尔茨海默症就像每年体检验血一样简单。研究表明未来并没有那么遥远,“诊断法加速器”使我们向未来又迈进了一步。
Why diagnosing Alzheimer’s today is so difficult—and how we can do better
When I announced that I was investing in Alzheimer’s research for the first time last fall, I thought I knew what to expect. I knew I would get to engage more deeply with the brilliant scientists and advocates working to stop Alzheimer’s—and I haven’t been disappointed. The things I’ve seen over the last seven months make me more hopeful than ever.
What I didn’t see coming was the amazing response I got from the Alzheimer’s community at large. Because my family didn’t talk publicly about my dad’s diagnosis before the announcement, I had yet to experience how remarkable the support community is. So many of you have shared your personal experiences with me, both in person and online (including here on TGN). It helps to hear from others who are going through the same thing.
Alzheimer’s research is a frontier where we can dramatically improve human life—both the lives of people who have the disease and their loved ones. I’m optimistic that we can substantially alter the course of Alzheimer’s if we make progress in several key areas. One of the biggest things we could do right now is develop a reliable, affordable, and accessible diagnostic.
The process of getting diagnosed with Alzheimer’s today is less than ideal. It starts with a cognitive test. If you don’t perform well, your doctor needs to rule out all other possible causes for memory loss, like stroke or a nutritional deficiency. Then your doctor can order a spinal tap or PET scan to confirm you have Alzheimer’s. Although these tests are fairly accurate, the only way to diagnose the disease definitively is through an autopsy after death.
There are two big problems with this process. First, it can be expensive and invasive. Most insurance plans in the United States won’t reimburse tests for Alzheimer’s. Patients often pay thousands of dollars out of their own pockets. Meanwhile, spinal taps can be scary and uncomfortable, and PET scans require the patient to stay perfectly still for up to 40 minutes. That’s difficult for anyone to do—but especially someone with Alzheimer’s.
Second, patients aren’t being tested for the disease until they start showing cognitive decline. The more we understand about Alzheimer’s, the clearer it becomes that the disease begins much earlier than we previously thought. Research suggests Alzheimer’s starts damaging the brain more than a decade before symptoms start showing. That’s probably when we need to start treating people to have the best shot at an effective drug.
This delay is a huge problem in the quest for a scientific breakthrough. It’s currently so difficult to find enough eligible patients for a clinical trial that it can take longer to enroll participants than to conduct the study. We need a better way of diagnosing Alzheimer’s—like a simple blood test or eye exam—before we’re able to slow the progression of the disease.
It’s a bit of a chicken and egg problem. It’s hard to come up with a game changing new drug without a cheaper and less invasive way to diagnose patients earlier. But most people don’t want to find out if they have the disease earlier when there’s no way to treat it. The commercial market for Alzheimer’s diagnostics simply isn’t there. There’s promising research being done, but very few companies are looking at how to turn that research into a usable product.
That’s why my next investment in Alzheimer’s research is in a new fund called Diagnostics Accelerator. This project of the Alzheimer’s Drug Discovery Foundation (ADDF) aims to accelerate bold new ideas for earlier and better diagnosis of the disease. Today I’m joining Leonard Lauder, ADDF, the Dolby family, the Charles and Helen Schwab Foundation, and other donors in committing more than $30 million to help launch Diagnostics Accelerator.
Diagnostics Accelerator is a venture philanthropy vehicle, which means it’s different from most funds. Investments from governments or charitable organizations are fantastic at generating new ideas and cutting-edge research—but they’re not always great at creating usable products, since no one stands to make a profit at the end of the day. Venture capital, on the other end of the spectrum, is more likely to develop a test that will actually reach patients, but its financial model favors projects that will earn big returns for investors.
Venture philanthropy splits the difference. It incentivizes a bold, risk-taking approach to research with an end goal of a real product for real patients. If any of the projects backed by Diagnostics Accelerator succeed, our share of the financial windfall goes right back into the fund.
My hope is that this investment builds a bridge from academic research to a reliable, affordable, and accessible diagnostic. I expect to see lots of new players come to the table, who have innovative new ideas but might not have previously had the resources to explore them. If you think you’re one of these bold thinkers, we want to hear your great ideas. I encourage you to apply for funding on the new Diagnostics Accelerator website by clicking on "Read More".
Imagine a world where diagnosing Alzheimer’s disease is as simple as getting your blood tested during your annual physical. Research suggests that future isn’t that far off, and Diagnostics Accelerator moves us one step closer.