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.
2. 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.
3. 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?