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NP-C Corner: Will I be a lab rat? An exploration of clinical trials in the world of Niemann-Pick.

“Will I be a lab rat?” …. “Is it safe?”

When facing an NP-C clinical trial for the first time, these questions are understandable.

Movies and shows can give the impression that scientists are people in white lab coats running risky experiments without care of consequence. You may have also seen stories in the news that showcase the rare event of serious complications occurring in research.  

Screenshot from a 'Back to The Future' movie
Source: Screenshot from a 'Back to The Future' movie.

Today, I want to give you a more realistic picture of what research is like.

As you may already know, I am a scientist.

Throughout my studies and via personal experience, I’ve become quite familiar with research. Even though I don’t wear a lab coat (sadly because they feel so cool!), I hope I can share some truths about this space.

It is a big topic, but I’ll do my best to tell you the key bits!

The reality of research

Research is a major part of science, and without it, we wouldn’t have some of the amazing medical advancements we do today.

Research has helped us work toward treating terminal cancers, extending the average lifespan of people, and diagnosing children with rare conditions like NP-C so they can access treatments earlier.

In the Niemann-Pick Disease (NPD) community, we rely on research to bring forward new possible treatments and therapies that can help to manage these diseases.

But I understand that if you’ve never been involved in research before, it can seem a little scary.

So, how does it all work?

Scientific research is not done simply because someone wants to do it.

There are certain hoops researchers need to jump through to get their intervention (this could be a new drug, a specific therapy, or something else they want to try) to the clinical trial stage.

Lots of evidence needs to be collected (usually costing millions of dollars and years of work) to demonstrate that the intervention significantly helps people and to justify why it should be used in human trials.

According to Harvard Business School, for every 5,000 drugs designed in a lab, only 5 are tested with people and only 1 will make it to the market.

Infographic from Saurav Patayl via ‘BenevolentAI: Revolutionizing drug discovery using Artificial Intelligence’
Source: Saurav Patayl via ‘BenevolentAI: Revolutionizing drug discovery using Artificial Intelligence’

Two of the many reasons why drugs may not get to the trial stage are that they do not meet the strict global research guidelines, or they are not safe enough for people to use.

As NPD is a rare condition, there are certain ways to fast-track these processes to shorten the time-to-market for treatments. But there are still extensive follow-ups and strict requirements to make sure it is generally safe for people.

Human ethics boards, like the Australian Human Research Ethics Committee (HREC), ultimately decide if a research project can go ahead or not. They scrutinise the project plan and the intervention to make sure it meets a range of set criteria that are intended to maximise the safety of people and their data.  

So, you can argue that participants (people who voluntarily participate in the study) are considered the most important people in the room. Without participant help, the research could simply not be done.

Doctor using a stethoscope on an adult patient
Source: Antoni Shkraba via Pexels

Medications will generally affect people in different ways because we are all different people. So, there is always the possibility of positively or negatively reacting to a new drug.

But even though treatments may have side effects, so do most common medications. You can easily react to a new sunscreen or feel unpleasant from an over-the-counter painkiller.  

To address the chance of side effects, clinical trial researchers closely monitor participant health so that they can do their best to ensure participants are not significantly harmed.

So, are clinical trials “safe?”

The benefit of the drug is likely to outweigh the risk, otherwise, the drug would not be in the human trial stage at all. Risk will always be there, however, in clinical trials, it’s closely controlled and monitored.

I hope this blog post helps you understand more about clinical trials. Keep an eye out on our website for more information coming soon!

Stay supportive and supported,


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