RARECast - 利用AI开放创新应对ALS 封面

利用AI开放创新应对ALS

Using AI Open Innovation to Tackle ALS

本集简介

崔斯·戴森创立了Challenge Works,旨在激励创新者解决社会问题。被诊断出患有肌萎缩侧索硬化症的戴森,如今正利用这一平台为这种渐进性神经退行性疾病寻找新疗法。Challenge Works推出的1000万美元“ALS经度奖”项目,结合人工智能、开放协作和大数据来探索该病症的新疗法。我们采访了Challenge Works的董事总经理戴森,谈及他的ALS诊断经历、通过奖项推动创新的理由,以及利用人工智能寻找疾病治疗方案的潜力。

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Speaker 0

本期RareCast播客节目由全球基因企业联盟赞助播出。联盟成员支持全球基因的使命与项目,致力于满足罕见病患者的关键需求,并解决他们面临的不平等问题。欲了解更多关于企业联盟的信息或贵机构如何加入,请访问globalgenes.org,并在'关于'选项卡下选择'企业联盟'。我是丹尼尔·莱文,这里是RareCast。特里斯特·戴森创立Challengeworks平台,旨在激励创新者解决社会问题。

The following RareCast podcast is made possible through the support of the Global Genes Corporate Alliance. The members of the Corporate Alliance support Global Genes mission and programs, work to meet the vital needs of people with rare diseases, and address inequities they face. To learn more about the Corporate Alliance or how your organization can become a member, go to globalgenes.org and choose Corporate Alliance under the About tab. I'm Daniel Levine, and this is RareCast. Trist Dyson founded Challengeworks to incentivize innovators to solve societal problems.

Speaker 0

被诊断出肌萎缩侧索硬化症的戴森,现正利用该平台为这种渐进性神经退行性疾病寻找新疗法。1000万美元的Challengeworks ALS经度奖运用人工智能、开放协作和大数据来寻找新治疗方案。我们采访了Challengeworks董事总经理戴森,谈及他自身的ALS诊断经历、通过奖项激励创新的理由,以及利用AI寻找疾病治疗方案的潜力。特里斯,感谢你的参与。

Dyson who was diagnosed with amyotrophic lateral sclerosis, is now using the platform to find new treatments for the progressive neurodegenerative disease. The $10,000,000 ChallengeWorks Longitude Prize on ALS harnesses AI open collaboration and big data to find new treatments for the condition. We spoke to Dyson, Managing Director of Challengeworks about his own diagnosis of ALS, the case for using a prize to spur innovation and the potential for leveraging AI to find treatments for the disease. Tris, thanks for joining us.

Speaker 1

不客气,很高兴能来参加节目,丹尼。

No. It's a pleasure to be here, Danny.

Speaker 0

我们将讨论罕见神经肌肉疾病肌萎缩侧索硬化症(ALS),以及Challengeworks如何通过ALS经度奖激励相关创新与治疗。让我们从ALS开始。对于不熟悉ALS的听众,这是什么疾病?

We're gonna talk about the rare neuromuscular condition amyotrophic lateral sclerosis challenge works and how it's seeking to spur innovation and treatments for the condition with the Longitude Prize on ALS. Let's start with ALS. For listeners not familiar with ALS, what is it?

Speaker 1

在美国,它常被称为卢·格里克病,得名于20世纪初一位罹患此病的著名棒球运动员。本质上这是一种神经退行性疾病,但与痴呆症不同,它主要影响肌肉运动和肌肉控制。随着运动神经元死亡,患者会逐渐丧失肢体、腿部、语言功能,最终影响呼吸能力。

Well, I think in The States, it's often known as Lou Gehrig's disease after a famous baseball player early part of the twentieth century who came down with it. It's basically a neurodegenerative disease, but unlike dementia, for example, it affects muscle movement, muscle control. And as motor neurons die, you lose function of limbs, legs, speech, and ultimately breathing.

Speaker 0

它的症状表现和发展过程是怎样的?

And how does it manifest itself and progress?

Speaker 1

每个人的情况都不同。平均而言预期寿命很短,患者通常在两三年内去世。有些人从四肢开始发病,表现为肢体起病型。他们的手臂和腿部会逐渐退化,行走、提物变得困难,最终症状蔓延至胸部,这时呼吸就会变得困难。

Well, it's different for different people. It, on average, has a very short life expectancy, so people are normally dead within two or three years. For some people, it starts in the limbs, so limb onset. And for them, their arms and legs start to deteriorate. It becomes hard to walk, to lift things, and then eventually it spreads to the chest, and that's when breathing becomes difficult.

Speaker 1

最终,嗯,先是言语和呼吸功能丧失,然后走向死亡。但对其他人而言,疾病始于所谓的延髓发病,症状最初体现在声音和喉咙部位。不幸的是,这种发病起点更糟糕,因为呼吸功能恶化的速度会明显加快。但必须说明的是,个体差异极大——有些人一年内就会离世,而有些人能存活很多年。

And then ultimately, well, talking and breathing and then ultimately death. But for other people, it starts with what they call bulbar onset, and this is where it starts really around symptoms begin in your voice and in your throat. And unfortunately, that's a worse starting point because obviously then the deterioration towards poorer breathing is quicker. But it has to be said, it's extremely different and divergent for different people. You know, it can rain some people are dead within a year, but some people live many years.

Speaker 1

比如众所周知的斯蒂芬·霍金,他就存活了几十年。所以这种疾病的病程存在巨大的个体差异。

I mean, the famous person that people will know of is Stephen Hawking. He lived for many decades. So it can have a huge degree of variation from person to person.

Speaker 0

目前有哪些治疗选择?

And what treatment options exist today?

Speaker 1

简而言之,实际上没有真正有效的治疗方法。包括英国在内的大多数国家使用一种叫利鲁唑的药物,已上市二十余年。这种药价格低廉,能适度延缓病情发展但效果有限。还有类似药物如依达拉奉,在某些国家获批使用。

I mean, the short answer is there are no treatments really. In most countries, including The UK, there is a drug called Riluzole, which has been around for a couple of decades. This is a fairly cheap drug. It slows the disease down modestly, but not significantly. And there are some other drugs that are similar to that, like edaravone, which is licensed in some countries.

Speaker 1

这些药物的效果都不显著。

These drugs don't have a huge effect.

Speaker 0

这种药在美国是不是叫tofersen?

Is that drug the one that's known as tofersen in The United States?

Speaker 1

这是种新药,非常令人振奋。Tofersen专为约2%的SOD1基因突变导致的遗传性ALS患者研发。该药物能显著延缓病情发展,部分患者甚至报告功能部分恢复——比如原本行走困难现在有所改善。这确实是种突破性新药。

Well, that's a new drug and this is very exciting. Tufersen is a drug that's been developed for a very small group of people with ALS, about two percent of patients, who have an inherited form of the disease through a gene called SOD1. And this drug does have a very significant effect in slowing the disease down. Some people even report partial reversal as in they're regaining some function, but maybe they couldn't walk so well and now they're able to walk better, that sort of thing. So it's a remarkable new drug.

Speaker 1

这展示了可能性。是的,它即将上市。已在欧洲和美国推出,非常令人振奋。但仅适用于极少数患者群体。

It shows what's possible. And yes, it is coming on to the market. It's been in both Europe and The US and is very exciting. But it is for a small, very, very small group of patients.

Speaker 0

那么考虑到未满足的需求,你认为其重要性如何?

So as you think about the unmet need, how significant would you say that is?

Speaker 1

嗯,我是说,对于其他98%的人群,正如我所说,目前确实没有真正有效的药物。诊断情况也相当糟糕,患者往往需要很长时间才能确诊。这种疾病的治疗和诊断方式近百年来都没有实质性突破,而且这是一种毁灭性的疾病。

Well, I mean, for the other ninety eight percent of people, as I said, there's not really any effective drug. And the diagnosis is pretty poor as well. It takes a long time for people to get diagnosed. So there's been no change in the treatment or diagnosis of the disease of any real note for a hundred years. And it's a devastating disease.

Speaker 1

它会剥夺你的自理能力,夺走你的声音、语言能力、进食功能,最终连呼吸都无法自主。这是一种极其残酷的疾病。在美国约有3.3万名患者,你可能觉得这个数字不算庞大,但这是因为太多患者迅速离世导致的。

It takes away your ability to your independence. It takes away your voice, your speech, your ability to eat and drink, and then takes away your ability to breathe. So it's a very cruel disease. In The United States, it affects about thirty three thousand people that have it. So you might think that's not a huge number of people, but the reason for that is because so many people are dying so quickly.

Speaker 1

如果患者生存期更长且可治疗,这个数字会高得多。粗略估计,每300人中就有一人可能在一生中罹患此病。所以它不算常见病,但也并非特别罕见。

If it had a longer life expectancy and it was treatable, it would be a much higher number. And roughly speaking, one in three hundred people are expected to get the disease over the course of their lifetime. So it's not a common disease, but it's not particularly rare either.

Speaker 0

你认为为何新疗法难以惠及患者?是因为对疾病认知不足?还是经济因素导致研发动力不足?为何进展如此缓慢?

Why do you think it's been so difficult to bring new therapies patients? Is it a lack of understanding of the disease? Is it a lack of interest for economic reasons? Why haven't we seen greater progress?

Speaker 1

我认为原因有很多。首先,和所有神经退行性疾病一样,其机制极其复杂。大脑中发生的许多变化难以理解和解析。而且由于显而易见的原因,我们很难在生前与大脑进行交互研究,这增加了研究难度。此外,这种疾病具有高度异质性。

I think the reason is that well, there's a number of reasons. I mean, the first is that as with all the other neurodegenerative diseases, it's extremely complicated. There's a lot going on in the brain that's hard to understand and to interpret. It's also very difficult to interact with a brain for obvious reasons until after somebody has passed, so that makes it hard. It's extremely heterogeneous.

Speaker 1

我是说,这对不同人来说完全不同。正如我们刚才讨论的,针对一个小型基因亚群的首个治疗方案已经出现。亚群种类繁多。我认为过去五十年左右资金一直相对不足。近十到十五年情况显著改善,很大程度上得益于大西洋两岸的患者和公众成员筹款并提高了这种疾病的知名度。

I mean, it's it's completely different for different people. As we just discussed, there's the to first in treatment for a small genetic subgroup. There are many subgroups. I think it has been somewhat underfunded over the last fifty years or so. That has changed significantly in the last ten, fifteen years, and a lot of that has been because of patients and members of the public on both sides of the Atlantic raising money and raising the profile of the disease.

Speaker 1

比如,你可能记得冰桶挑战。那筹集了大量资金。在英国,我们有许多橄榄球运动员参与。在美国,则有几位演员和足球明星为提升疾病认知和筹款做出了巨大贡献,这些资金流向了研究和数据收集领域。这意味着人们对这种疾病的了解已从几乎一无所知发展到拥有非常扎实的基础知识。

Mean, you might remember the ice bucket challenge. That raised a lot of money. In in The UK, we have a number of rugby players. In The US, there's some several actors and football stars that have done a huge amount to raise a profile of disease and raise a lot of money, and that's gone into research and it's gone into data gathering. And what that means is the level of understanding of the disease has gone from being something that people didn't know very much about to something where there was really very strong foundational knowledge.

Speaker 1

这就是为什么我们开始看到针对个人的首批治疗方案。希望很快会有更多疗法问世。

And that's why we're starting to see treatments to persons the first one. Hopefully, are others coming along soon.

Speaker 0

对于不熟悉Challengeworks的听众,你能解释一下这是什么机构吗?

And for listeners not familiar with Challengeworks, can you explain what that is?

Speaker 1

Challengeworks是一家设计和实施挑战赛奖项的组织。这意味着我们会寻找那些我们认为存在巨大创新潜力、能实现突破并解决特定问题或开拓新市场的领域。然后我们发起竞赛,提供现金奖励和其他激励措施,鼓励人们在从农业到太空等众多领域开发这类解决方案和突破性技术。

Challengeworks is an organization that designs and delivers challenge prizes. And that what that means is we look for gaps where we think there's a lot more potential for innovation to make a breakthrough and solve a particular problem, crack a new market. And then we launch a competition with a number of cash prizes and other types of incentives to encourage people to develop those sorts of solutions and breakthroughs in lots of different areas from agriculture to space.

Speaker 0

那么用奖项激励创新来解决重大社会挑战的依据是什么?

And what's the case for using a prize to encourage innovation to address large social challenges?

Speaker 1

是的。这适用于存在市场缺口或部分市场失灵的情况。你总是在寻找存在需求、会有资金投入、会有投资和客户的机会领域。但在初期阶段存在不确定性,不清楚这些要素会从何而来,这意味着尚未有明确的主导者出现。这时你设立奖项吸引创新者解决特定问题,从而培育新市场或新投资领域的雏形。

Yeah. I mean, it's where there's a market gap or a market a partial market failure. So you're always looking at an opportunity where there's demand and where there will be money and where there will be investment and where there will be customers. But where it's a bit unpredictable to begin with and it's unclear where that's going to come from and that means that no one's yet emerged. And then you set the prize to attract in innovators to address that particular problem and then feed the beginnings of a new market or new investor space.

Speaker 0

能否举例说明你们迄今为止在过往挑战和奖项设置方面的工作,这些奖项如何推动了关键创新?

Are there some examples of what you've done to date in terms of previous challenges and the prizes that have been awarded that have led to key innovations?

Speaker 1

是的。我们的核心奖项类别之一是'经度奖'。大约十年前,我们针对抗生素耐药性(AMR)设立了这项全球性挑战奖项,旨在邀请创新者开发能快速区分细菌性与病毒性感染的诊断技术。

Yeah. One of our key prize categories is called the Longitude Prize. And we launched a prize, it was about ten years ago now, on AMR, antimicrobial resistance. And this was a global challenge. And we were inviting innovators to develop a diagnostic that would be able to quickly differentiate between a bacterial and a viral infection.

Speaker 1

若确诊为细菌感染,该技术还需精确告知所需服用的抗生素。全球约250支团队参与角逐,去年由一支瑞典团队胜出,随后被日本公司Sysmex Astrego收购。他们研发的设备完全实现了这些功能。可以想象,未来几年当人们去诊所甚至药房时,只需做个检测——可能无需看医生——就能知道是细菌还是病毒感染。若是细菌感染,设备会精准推荐应使用的抗生素。

And if it was bacterial, tell you exactly what antibiotic you needed to take. And this include teams about 250 teams from around the world, and it was won last year by a Swedish team that then got acquired by a Japanese company, Sysmex Astrego, and they have developed this device that does exactly that. And if you can imagine in a few years' time, when you go to a clinic or possibly even a pharmacy, you will take a test. You may not need to see a doctor, and the test will tell you if it's bacterial or viral. If it's bacterial, it will tell you exactly the right antibiotic to take.

Speaker 1

因此我们认为这将是颠覆性的变革。

So we think this is an enormous game changer.

Speaker 0

需要说明的是,您个人对此挑战有着深切关注。2022年您被确诊患有ALS(肌萎缩侧索硬化症),当时是如何确诊的?

I should note you have more than a passing interest in this challenge. In 2022, you were diagnosed with ALS. How did you come to get diagnosed?

Speaker 1

某种程度上这是个常见故事。我注意到左手拇指活动异常,当时没太在意——其实本该早点就医的。

Well, it's quite a common story in some ways. I mean, I noticed that my thumb wasn't working properly, my left thumb. I didn't think too much of it. I probably should have seen somebody earlier. I didn't.

Speaker 1

我辗转多位临床医生,他们起初认为可能是肌肉损伤。最终转到神经科医生那里,他们发现了典型症状:肌肉痉挛、抽搐以及部分肌肉萎缩。随后伦敦国王学院的专科诊所确诊了ALS。这个过程耗时很长,但很常见——人们往往早期难以察觉这些症状。

I went to various clinicians and doctors who thought that it might be muscular or that I damaged it in some way. And eventually, I got directed towards neurologists who spotted the other telltale signs, so spasticity in the muscles twitching and other atrophy in parts of my muscles. And they then sent me on to a specialist clinic at King's in London, and and they confirmed ALS. So it took a long time, and that's quite common. I mean, people often don't spot the signs very early on.

Speaker 1

你知道,目前还没有诊断方法,就像我之前说的。所以你实际上是在依赖经验丰富的神经科医生通过观察症状来做出判断。

You know, there's no at the moment, there's no diagnostic, as I said earlier. So you're literally relying on experienced neurologists to basically look at your symptoms and determine it that way.

Speaker 0

六月份,Challengeworks宣布了关于ALS的经度奖。这个奖项是什么?资金如何筹集?以及它是如何运作的?

In June, Challengeworks announced the Longitude Prize on ALS. What is the prize? How is it funded? And how does it work?

Speaker 1

是的。好的。我们认为破解这个奖项的关键在于新兴的人工智能形式,这个人工智能的新时代。我们相信这对于处理和把握这种疾病的复杂性以及理解其生物学机制至关重要。因此,我们汇集了同类中最大的疾病数据集,包括来自美国和加拿大患者的大量基因组和多组学数据,还有许多其他国家的数据。

Yeah. Okay. So we think the key to cracking this prize is the new emerging forms AI, the new the new era of of artificial intelligence. And we think that that's gonna be critical in handling and getting to grips with the complexity of this disease and understanding the biology. So we've pulled together the largest disease dataset of its kind, huge genomic, multiomics data from all around the world, including from patients in The United States and Canada, but from many, many other countries as well.

Speaker 1

这些数据将提供给参赛团队。我们预计AI公司、研究机构或大学会申请。申请截止日期是12月3日,但他们需要具备一定的疾病专业知识,因为AI可以发现各种模式,但大多数在没有专业知识的情况下是无意义的。所以,如果有AI领域的人士认为自己具备工具但缺乏疾病专业知识,请联系我们,我们可以帮你对接相关专家。总之,这些团队会申请。

And this data will be available to competing and participating teams. Now what we're expecting is that AI companies or research institutions, universities will apply. The application is closes on December 3, but they will need some disease expertise because AI can find all sorts of patterns, most of which would be irrelevant without their expertise. And so if there are AI folks listening here that think that they've got the the tools but don't have the disease expertise, please do reach out because we can connect you with people who do. Anyway, these teams will then apply.

Speaker 1

我们将从全球选出20个团队。他们将获得数据访问权限,大约13万美元(汇率允许的话),并有九个月的时间。在这九个月里,他们需要识别目标。所谓目标,指的是如果你能改变疾病的生物学要素,就能改变疾病进程的那些点。

They will get we will select 20 from around the world. They'll get access to this data. They'll get about a $130,000 exchange rates permitting, and they'll have nine months. And nine months from which they are looking to identify targets. And by targets, we mean elements of the biology of the disease that if you could alter it, you would change the the course of the disease.

Speaker 1

因此,他们在寻找可以开启或关闭的东西。之后会有第二轮筛选,我们将缩减到10个团队。这10个团队将获得更多数据,以及大约25万到30万美元的资金。如果需要实验室支持,他们也能获得。我们认为在那个阶段很可能需要。

So they're looking for things that you might switch on and off. There will then be a second round where we would down select to 10 teams. Those 10 teams will get more data, and they will get about quarter of a million to 300,000 US dollars. And and they will also get lab access if they need lab access. We think at that stage, probably will.

Speaker 1

然后他们将优先考虑并寻找最具潜在成药性的目标,即他们认为可以转化为药物或针对其开发药物的生物学要素。接着进入第三轮,剩下5个团队。这5个团队将进入实验室,测试目标在成药性方面的潜力,他们将获得75万美元。最终,一个团队将赢得100万英镑的大奖,约合120万美元,用于被认为最具成药潜力的目标。

And they will then look to prioritize and look for which is the most potentially druggable target, which element of the biology they think could be turned into a or or you could train a drug at. And then we move into a third round, and the third round is five teams. And the five teams will then go into the lab, and they will test the targets against their potential drug ability, and they will get three quarters of a million dollars. And then one team will be awarded will be awarded the the winning prize, which is a million pounds. So I don't know, $1,200,000, something like that, for the target which is deemed to have the greatest potential to become a drug.

Speaker 0

所有这些资金是从哪里来的?

Where does the funding for all this come from?

Speaker 1

嗯,正如我之前提到的,这个领域的大量研究资金来自患者和公众筹款。我们的资金很多来自各类组织,包括美国的机构,但也遍布全球,如欧洲和澳大利亚,那里的患者和公众筹集了这些资金。我们还在美国与约翰霍普金斯大学相关的PACCARD中心、ANTS ALS合作,这些机构提供了大量数据来源,同时在西海岸还与'万脑计划'合作。

Well, as I mentioned to you before, a lot of the research funding in this space has come from patients who have raised and members of the public who have raised money. And so a lot of our funding comes from organizations, including in The United States, but all around the world, around Europe and Australia as well, where patients and members of the public have raised those funds. We're also working with in The United States with PACCARD Center, which is associated with Johns Hopkins, ANTS ALS, which is also associated with Johns Hopkins, which is where a lot of the data comes from, and on the West Coast with 10,000 brains.

Speaker 0

我想这在五到十年前可能还无法实现。从数据和技术的角度来看,是什么变化使之成为可能?

This is a challenge that I imagine five, ten years ago wouldn't have been possible. What's changed in the landscape from a data point of view and a technology point of view to to enable it?

Speaker 1

嗯,我认为AI模型确实起到了关键作用。你说得对,十年前的技术水平与现在完全不同。如今的可能性令人难以置信。我们开始看到这些AI模型在药物研发中的影响,但它们更多被用于已有治疗方案的疾病领域,比如COVID或心血管疾病。

Well, I mean, I think the AI models have yeah. You're right. I mean, ten years ago, the level of sophistication I mean, it's just it's completely changed over that period of time. And what's now possible is unbelievable. We're also starting to see the effect of these AI models in drug discovery, but we're seeing them used often in existing disease areas where there are already existing treatments, so things like COVID or cardiovascular disease.

Speaker 1

这些是新药和有效药物,但可能不会成为头条新闻,因为它们只是众多药物中的一部分。但你可以看到其中的可能性。另一个因素是数据——我们进行了大规模的数据收集工作。在美国,数据开放程度高且共享意愿强,这很棒,但数据也相当分散。

So they're new drugs and effective drugs, but maybe not headline grabbers because they're just, you know, part of part and parcel of lots of other drugs. But you can see what's possible. The other thing is the data. There's been a huge exercise in gathering data. In The United States, you've got very open data and a willingness to share data, which is fantastic, but it's quite dispersed.

Speaker 1

因此需要开展数据整合工作,这项工作仍在进行中。在欧洲我们面临相反的问题:由于医疗体系特性存在大量数据集,但保护严密难以获取。所以我们不得不与一些组织合作来开放这些数据的访问权限。这两方面都发生了变化——AI的潜力以及数据的可获得性。此外正如之前提到的,我们对疾病的基础认知建立在相当扎实的现有理解上,而这在十年前也是不存在的。

So there's needed to be an exercise in bringing it together, and that's still happening. In Europe, we have kind of the opposite problem, which is that there's large data sets in sort of you know, because of the nature of the health care systems, but they're very well guarded and hard to get into. So in that case, we've had to sort of work with some of the organizations to to open up the access to that data. So those two things have changed, the the the the potential of the AI and also the availability of the data. And then also, as I mentioned earlier, the foundational knowledge of the disease, you're building on pretty strong existing understanding, and that wasn't true ten years ago either.

Speaker 0

其中一个有趣的方面是你们在为竞争对手提供数据。这个数据集有多大?包含哪些内容?来源是什么?

One of the interesting aspects of this is that you are providing data for competitors. How big a dataset is this? What does it include, and where does it come from?

Speaker 1

目前,我们拥有约10,000名患者的全基因组数据集和完整的多组学数据。还包括部分临床数据,因此规模相当庞大。在美国,数据来源包括纽约基因组中心和ALS协会的ANTS项目——他们在东海岸收集了大量数据。美国国立卫生研究院正在简化申请流程,以便我们的参赛团队能使用这些数据。

Well, at the moment, it's about 10,000 patients, and it's whole genome data sets, full multiomic data. There's some clinical data as well, so it's it's vast. Yeah. In The US, it includes data from the New York Genome Center, from ANTS at ALS who collected a lot of this data on the East Coast. NIH are streamlining the application process to use their data for our competing teams.

Speaker 1

虽然这部分不属于我们的数据提供范围,但我们也能协助获取这些数据。是的,数据量很大,而且我们还在持续扩充。

So although that's not part of our data offer, we're able to facilitate access to that data as well. Yeah. So it's a lot, and we're adding to it all the time.

Speaker 0

比赛结果会如何处理?会公开分享吗?还是由参赛者自行决定用途?

What happens to the results from the competition? Is this going to be shared publicly? Is it for the individual competitors to take and do with it as they wish?

Speaker 1

是的,我们不想阻碍成果商业化的可能性。但对于那些被发现却未被推进的靶点——我们预计会有很多这类情况,因为团队通常只会选择一两个进入下一阶段——我们将公开这些数据。这样所有人都能查看和获取未被继续研究的信息和数据。当然,团队可以自由分享他们正在研究的任何数据目标。

Yeah. So we don't want to discourage the ability to commercialize the results. But if targets that are discovered that are not taken forward, and we expect there to be quite a lot of those because the teams will pick one or two to take through to the next stages, etcetera, we will publish that. So everyone will have visibility and access to all the information and data of things that are not being pursued. Teams will, of course, be very free to share as widely as they wish data targets that they are pursuing.

Speaker 1

我们还将为他们对接潜在投资者和制药公司等可能推动项目发展的资源。

And we will be connecting them with potential investors and pharma, etcetera, that might wanna take it forward.

Speaker 0

你之前提到的一个有趣现象是:由于本次挑战赛的性质,有机会吸引通常不关注罕见病或ALS的智力资源来帮助识别潜在靶点。你们观察到这种情况了吗?目前是否有非常规背景的人士加入挑战?

One Yeah. Of the things you touched on earlier, but I find interesting is that because of the nature of this challenge, there's an opportunity here to bring brainpower that isn't normally focused on rare diseases or ALS and put them to work in helping identify potential targets. Are you seeing that? Are you getting unconventional people joining the challenge at this point?

Speaker 1

确实。我们正获得来自其他疾病领域建模的生物科技公司的关注,他们特别被这些数据吸引。当然奖金也有吸引力,但主要还是数据本身及其带来的机遇。我认为这里存在巨大的商业价值——如果能开发出ALS疗法,目前市场上几乎没有其他治疗方案,仅ALS领域就有巨大的市场空间。

Yeah. We're getting interest from biotech that are running models in other diseases that are particularly interested by the data. And, you know, they're not adverse to the money either, but it's mostly about the data and the opportunity that that presents. I mean, I think there is a big commercial case here, which is that if you can come up with the treatment for ALS, there are no other treatments pretty much. And so you've got an enormous market just for ALS.

Speaker 1

但另一方面,这种疾病极为复杂,与其他神经退行性疾病存在大量交叉。尤其是帕金森病和额颞叶痴呆最为典型,不过整体而言都是如此。所以如果能攻克渐冻症(ALS),很可能就能一并解决其他病症,你明白我的意思吧。因此这里蕴藏着巨大机遇,目前大多数神经退行性疾病都缺乏有效治疗手段——最典型的当属阿尔茨海默症。

But the other thing is that the disease is extreme there's a lot of crossover with other neurodegenerative diseases. Parkinson's in particular and frontotemporal dementia also in particular, but just in general. So if you can crack ALS, you can probably crack the others as well, if you see what I mean. So so there is a big opportunity, and I think that is starting to get some in I mean, neurodegenerative diseases, currently, most of them don't have very many effective treatments. I mean, the obvious one is is is Alzheimer's.

Speaker 1

但整体来看,这是个巨大的市场机遇,正是这些AI工具能产生颠覆性影响(而非渐进式改进)的领域——相比那些已有治疗方案的疾病而言。

But taken as a whole, it's an enormous market opportunity, and it's really where these AI tools can have a massive impact rather than a kind of incremental but significant impact with diseases that have existing treatments.

Speaker 0

人们可以通过什么渠道了解更多挑战赛信息?如果想报名该怎么做?

And where can people go to learn more about the challenge, how can they sign up if they want to?

Speaker 1

可以通过我们的网站注册,地址是als.longitudeprize.org。当然在谷歌搜索'经度奖'或通过LinkedIn等平台也能找到。目前我们正邀请各方注册,以便建立潜在合作联系。比如拥有丰富神经退行性疾病研究经验的学术团队若想与AI生物技术公司合作(或反之),我们都希望能促成这类对接。

They can sign up on our website. So it's als.longitudeprize.org. But, you know, if you Google Longitude Prize or you punch it into LinkedIn or something, you should find it. At the moment, we're asking people to register so that we can make connections where we think it might be useful. So, you know, if you're an academic group with a strong neurodegenerative experience looking for to partner with an AI biotech or the other way around, we we hope to make those connections.

Speaker 0

所以你们是在协助组建参赛团队?

So you're helping facilitate the formation of teams?

Speaker 1

没错,有点像婚恋配对服务吧(笑)。

Yeah. Sort of a date a dating service, I guess. Yeah.

Speaker 0

太棒了。Challengeworks董事总经理特里斯·戴森,非常感谢您今天抽空接受采访。

Great. Tris Dyson, managing director of Challengeworks. Tris, thanks so much for your time today.

Speaker 1

谢谢。

Thank you.

Speaker 0

感谢您的收听。如需了解更多关于罕见病的信息并与罕见病社区建立联系,请访问globalgenes.org。要获取影响罕见病社区的最新新闻和趋势,请务必访问raredaily.org。您可以通过raredaily.org或SoundCloud、iTunes、Stitcher及您常用的播客管理器订阅RareCast的RSS订阅源。RareCast由Lavune Media Group为Global Genes制作。

Thanks for listening. For more information about rare disease and to connect to the rare disease community, go to globalgenes.org. To keep up on the latest news and trends affecting the rare disease community, be sure to visit raredaily.org. You can subscribe to the RareCast RSS feed through raredaily.org or through SoundCloud, iTunes, Stitcher, or your preferred podcast manager. The RareCast is produced for Global Genes by the Lavune Media Group.

Speaker 0

您还可以在这些热门播客网站上找到我们的播客节目《生物报告》。我们的主题音乐由Jonah Levine创作,Jonah Levine Collective演奏。我们期待您的反馈,请发送邮件至Danny@LevineMediaGroup.com与我们联系。

You can also find our podcast, the bio report, on these popular podcast sites. Our theme music is composed by Jonah Levine and performed by the Jonah Levine Collective. We'd love to hear from you. Drop us a note at Danny@LevineMediaGroup.com.

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