Should you donate your DNA to help cure diseases Greg Foot

So here’s the thing: developing a new
drug and getting it to you

can take a long time.

When we have to work
out the cause of a condition—

for example, with multiple sclerosis
or heart disease—

developing a new drug takes significant
trial and error and lots of money.

Which is why we only have drugs
for a small proportion of diseases.

But you could change all this.

You could help discover new, cheaper drugs
for currently untreatable diseases.

It’s all about medical crowdsourcing.

However, researchers aren’t asking
you to donate your money,

they’re asking you to donate
something more personal…

First, though, some drug development
history.

Many of the first medicines
were discovered by chance.

Natural philosophers
then took these

and identified the active chemicals
inside.

And pharmaceutical companies
then turned those into drugs.

The thing is, for a long time, we didn’t
know why those drugs worked.

Until scientists figured out that disease
happens when the molecular machines

that keep your body going— your proteins—
start misbehaving.

Drugs treat disease by targeting
those disruptive proteins.

Researchers realized
that if they can identify

which malfunctioning proteins cause
a specific disease,

they can then try to find
or develop a drug

that stops those proteins acting up,
and that will prevent the disease.

It’s a great plan,
but it’s a slow process.

So far, they’ve only identified
these therapeutic targets

for a small proportion of diseases.

However,
this is where you can help.

Researchers are now turning
their attention to DNA,

to the genetic instruction manual that
tells our bodies how to make our proteins.

They want to know which small changes
in someone’s genome

can lead to the production of those
dodgy proteins that cause a disease.

The thing is, that’s a big job.

DNA is huge, and each disease is likely
to have hundreds, possibly thousands,

of proteins involved.

But if they have lots of people’s genomes,
they can compare them and spot patterns.

They can look at multiple people
suffering from

the same currently untreatable disease,

find any small genetic changes they share,

identify the faulty proteins
they code for,

and there you go:
those are brand new therapeutic targets

for a currently untreatable disease.

Now the researchers have three options:

  1. Has one of those new target proteins
    been previously linked

to a different disease that is treatable?

If so, the drug for that disease
may target this protein

and work for this disease, too.

To find out, start a clinical trial.

  1. If not, has one of those new target
    proteins being previously linked

to a different disease that had
a promising drug

that didn’t ultimately work?

If so, its promise may have come
from successfully targeting this protein

and it may work for this disease.

Start a clinical trial to find out.

  1. If this is a brand new protein target
    never identified before for any disease

could they design
a new drug to affect it?

This involves AI machine learning
and some very cool chemistry.

And a lot of time, effort, and cost too.

Researchers are excited about all this
because they think

1 in 5 of the proteins in your body
either have, or are likely to have,

a drug that will bind to them.

And, as any common disease is likely
to have hundreds, possibly thousands,

of proteins involved,

they’re hopeful they’ll be able
to identify a few of those proteins

they’ve already got a drug for.

But this all relies on finding
those new therapeutic targets,

and that’s why they need you.

Well, your data— both your genetic data
and your health history data,

so they can compare the genomes
of people with similar conditions.

So would you give your data for research?

There are two questions you may have:

who will have access to my data,
and what could they do with it?

One group is health care providers
who are starting to consider

using genetic analysis to give patients
more personal care.

Another group is private consumer
genetic testing companies.

Some have already sold genetic data
on to pharmaceutical companies for profit,

but that was with their customers consent.

However, it raises another question:

if your data goes towards
making new drugs,

should pharmaceutical companies
recognize that contribution

and offer drugs more cheaply?

Your best bet is to research the
organizations who are asking for your data

to find out what they will do with it
and how they will protect it.

We’ll each have our own take on this,

but what is clear is genomics
could be a powerful tool

to cut the current time and cost
it takes to develop new drugs

for currently untreatable diseases.

So, what do you think?

所以事情是这样的:开发一种
新药并将其提供给您

可能需要很长时间。

当我们必须
找出某种疾病的原因时——

例如,多发性硬化症
或心脏病——

开发一种新药需要大量的
试验和错误以及大量的资金。

这就是为什么我们只有
一小部分疾病的药物。

但你可以改变这一切。

您可以帮助发现新的、更便宜的药物
来治疗目前无法治愈的疾病。

这都是关于医疗众包的。

然而,研究人员并没有要求
你捐出你的钱,

他们要求你捐出
一些更私人的东西……

不过,首先,一些药物开发的
历史。

许多最早的药物
是偶然发现的。 然后

自然哲学家
拿走了这些

并确定了里面的活性化学物质

然后制药公司
将它们变成药物。

问题是,很长一段时间以来,我们都不
知道这些药物为什么会起作用。

直到科学家们发现,
当维持身体运转的分子机器——

你的蛋白质——
开始出现异常行为时,疾病就会发生。

药物通过靶向
那些破坏性蛋白质来治疗疾病。

研究人员意识到
,如果他们能够确定

哪些功能失常的蛋白质会导致
特定疾病,

那么他们就可以尝试寻找
或开发一种药物

来阻止这些蛋白质发挥作用
,从而预防这种疾病。

这是一个伟大的计划,
但这是一个缓慢的过程。

到目前为止,他们只

为一小部分疾病确定了这些治疗靶点。

但是,
这是您可以提供帮助的地方。

研究人员现在将
注意力转向 DNA,

转向
告诉我们身体如何制造蛋白质的遗传指导手册。

他们想知道
某人基因组中的哪些微小变化

会导致产生
那些导致疾病的不可靠蛋白质。

问题是,这是一项艰巨的工作。

DNA 是巨大的,每种疾病都可能
涉及数百甚至数千

种蛋白质。

但如果他们有很多人的基因组,
他们可以比较它们并发现模式。

他们可以查看多个

患有同一种目前无法治愈的疾病的人,

找到他们共有的任何微小的基因变化,

识别他们编码的有缺陷的蛋白质

然后就可以了:
这些

是目前无法治愈的疾病的全新治疗目标。

现在研究人员有三种选择:

  1. 这些新的靶蛋白

中的一种以前是否与另一种可治疗的疾病有关?

如果是这样,那么针对这种疾病的药物
可能会针对这种蛋白质

并且对这种疾病也有效。

要找出答案,请开始临床试验。

  1. 如果没有,这些新的靶
    蛋白中是否有一种先前

与另一种疾病相关,而这种疾病有
一种有前途的药物

,但最终没有奏效?

如果是这样,它的前景可能
来自成功靶向这种蛋白质

,它可能对这种疾病有效。

开始临床试验以找出答案。

  1. 如果这是一个全新的蛋白质靶点,
    以前从未发现任何疾病

,他们是否可以设计
一种新药来影响它?

这涉及人工智能机器学习
和一些非常酷的化学。

还有很多时间、精力和成本。

研究人员对这一切感到兴奋,
因为他们

认为你体内五分之一的蛋白质
要么有,要么可能有,

一种可以与它们结合的药物。

而且,由于任何常见疾病都可能
涉及数百甚至数千

种蛋白质,

他们希望
能够识别出一些

他们已经获得药物治疗的蛋白质。

但这一切都依赖于找到
那些新的治疗目标

,这就是他们需要你的原因。

好吧,你的数据——你的基因数据
和你的健康史数据,

这样他们就可以比较
患有类似疾病的人的基因组。

那么你会提供你的数据进行研究吗?

您可能有两个问题:

谁可以访问我的数据
,他们可以用这些数据做什么?

一组是医疗保健提供
者,他们开始考虑

使用基因分析为患者提供
更多的个人护理。

另一组是私人消费者
基因检测公司。

有些人已经将基因数据出售
给制药公司以获取利润,

但这是在他们的客户同意的情况下进行的。

然而,它提出了另一个问题:

如果您的数据用于
制造新药,

制药公司是否应该
认识到这一贡献

并以更便宜的价格提供药物?

您最好的选择是研究
那些要求您提供数据的组织,

以了解他们将如何处理这些数据
以及他们将如何保护这些数据。

我们每个人都会对此有自己的看法,

但很明显,基因组学
可能是一种强大的工具,

可以减少当前

为目前无法治愈的疾病开发新药所需的时间和成本。

所以你怎么看?