Should you donate your DNA to help cure diseases? - Greg Foot

譯者: Yi-Fan Yu 審譯者: Amanda Zhu

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

事情是這樣的：開發一個 可以交到你手上的藥物，

can take a long time.

需要花很長的一段時間。

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

當我們想要了解疾病的原因，

for example, with multiple sclerosis or heart disease—

比如，多發性硬化症或心臟疾病，

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

開發一個新藥物 要大量的反覆試驗而且很花錢。

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

這也是為什麼只有 一小部分疾病有藥物治療。

But you could change all this.

但你可以改變這個現狀。

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

你可以幫忙為尚無法治癒方法的疾病 開發便宜新藥物。

It's all about medical crowdsourcing.

這將依賴醫療群募。

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

但，研究人員不是要你捐錢，

they’re asking you to donate something more personal...

他們希望你可以捐出 一些比較私人的東西

First, though, some drug development history.

首先，談談有關藥物開發的歷史。

Many of the first medicines were discovered by chance.

初期新藥物的開發大多都是偶然的。

Natural philosophers then took these

自然哲學家們

and identified the active chemicals inside.

會從中找出有效的化學分子。

And pharmaceutical companies then turned those into drugs.

然後藥廠就會把這些成分變成藥物。

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

問題是，有很長一段時間， 我們不懂為什麼這些藥物有效。

Until scientists figured out that disease happens when the molecular machines

最後科學家終於搞懂，

病發的原因與身體裡的 「分子機器」蛋白質有關，

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

原來是維持身體運作的蛋白質在搗蛋。

Drugs treat disease by targeting those disruptive proteins.

藥物是藉由破壞這些 製造混亂的蛋白質來治療疾病。

Researchers realized that if they can identify

研究人員明白，如果他們可以識別出

which malfunctioning proteins cause a specific disease,

造成特定疾病的異常蛋白質，

they can then try to find or develop a drug

那他們就可以試著找出或開發出藥物，

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

來阻擋這些蛋白質作怪，並預防疾病。

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

這是個很棒的藥物開發計劃， 但流程很緩慢。

So far, they’ve only identified these therapeutic targets

到目前為止，它們只能識別出

for a small proportion of diseases.

少部分疾病的治療標靶。

However, this is where you can help.

然而，這就是你能幫忙的地方。

Researchers are now turning their attention to DNA,

研究人員開始把注意力

放在 DNA 這個人體裡的 基因操作手冊，

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

看它如何指導我們的身體 製造出這些蛋白質。

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

他們想要知道 是哪些基因組的微小變化

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

造成壞壞蛋白質的大量生產 進而使你生病。

The thing is, that’s a big job.

問題是，這是個大工程。

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

DNA 很複雜，

每個疾病可能都有 成千上百的蛋白質參與其中。

of proteins involved.

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

但如果科學家有很多人的基因組， 他們就能做比對，從中找出規律。

They can look at multiple people suffering from

從中他們可以找出 目前尚無治療方法的疾病

the same currently untreatable disease,

在不同人身上的致病原因，

find any small genetic changes they share,

找出共同在基因上發生的微小變化，

identify the faulty proteins they code for,

進而辨別出根據基因編碼 製造出的作怪蛋白質，

and there you go: those are brand new therapeutic targets

然後就能找出全新的治療標靶，

for a currently untreatable disease.

來治療目前尚未有療法的疾病。

Now the researchers have three options:

現在，研究人員有三個選項：

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

1. 這些新的蛋白質標靶當中，

是否其中一個已經與 其它可治癒疾病的發病有關?

to a different disease that is treatable?

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

如果有，治療該疾病的藥物 也許就能摧毀掉這個蛋白質，

and work for this disease, too.

並發揮功效。

To find out, start a clinical trial.

為了找到答案，就要展開臨床試驗。

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

2. 如果沒有，是否在這些 新的蛋白質標靶當中

有一個與其他疾病有關，

to a different disease that had a promising drug

該疾病曾有人研發 很看好的治療藥物，最後卻無效。

that didn’t ultimately work?

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

如果有，當初覺得很看好 可能是因為該藥物能對付這種蛋白質，

and it may work for this disease.

所以這種藥也許會發生作用。

Start a clinical trial to find out.

可以用臨床試驗確認看看。

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

3. 如果是全新的蛋白質， 從來沒有在其它疾病中被標定過，

could they design a new drug to affect it?

是否可以設計新藥來影響它?

This involves AI machine learning and some very cool chemistry.

這就牽扯到人工智慧的機器學習， 還有一些相當酷炫的化學。

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

當然還得付出很多時間、精力與成本。

Researchers are excited about all this because they think

研究人員對這一切都相當興奮，

因為他們認為你身體裡面 有五分之一的蛋白質，

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

多多少少都有藥物可以與之結合。

a drug that will bind to them.

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

任何常見的疾病

很可能都有成千上百的 蛋白質參與其中，

of proteins involved,

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

他們期望能夠利用現有的藥物

they’ve already got a drug for.

來辨識出這些蛋白質。

But this all relies on finding those new therapeutic targets,

但這都寄望在能找出 這些新的治療標靶，

and that's why they need you.

這也是為什麼科學家需要你的原因。

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

是的，你的資料， 包括你的基因資料與病歷資料，

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

這樣他們就可以拿 相同疾病患者的基因組做比對。

So would you give your data for research?

所以你願意把你的資料 提供給他們做研究嗎?

There are two questions you may have:

你可能會有兩個問題：

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

誰有權使用我的資料？他們會做甚麼?

One group is health care providers who are starting to consider

其中一個團體是提供健康照顧的機構，

他們已經開始考量利用基因分析 提供病人更多的個人照顧。

using genetic analysis to give patients more personal care.

Another group is private consumer genetic testing companies.

另一個團體是為一般消費者 做基因測試的私人公司。

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

有些公司藉由銷售 基因資料給藥廠營利，

but that was with their customers consent.

但要經客戶同意才可以。

However, it raises another question:

然而，這又產生另一個問題：

if your data goes towards making new drugs,

如果你的資料是用來做新藥研究，

should pharmaceutical companies recognize that contribution

那藥廠是不是應該認同我們的貢獻，

and offer drugs more cheaply?

而提供更便宜的藥物給我們?

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

最好的做法是， 你要去研究向你要資料的機構，

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

了解他們拿你的資料做什麼， 要如何保密你的資料。

We’ll each have our own take on this,

我們每個人對此都有自己的看法，

but what is clear is genomics could be a powerful tool

但很顯然的， 基因學是個強而有力的工具，

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

它能縮減新藥開發的時間與成本，

for currently untreatable diseases.

治癒目前無法治癒的疾病。

So, what do you think?

所以，你認為呢?