Why Animal Models Matter in NARS1 Research
From fruit flies to mice, and what this really means
You might have seen us talk about mouse models, and more recently fruit fly research.
On the surface, it can feel quite removed from real life.
Why flies? Why mice? And how does this actually help our children?
It’s a fair question.
Because before any treatment can even be considered for people, researchers need to understand what’s happening in the body, and test whether anything can safely change it.
That’s where these models come in.
What can fruit flies (Drosophila Melanogaster) tell us about NARS1?
As part of our research programme, we funded a $25,000 grant for Dr. Farzeneh Larti to study NARS1 using fruit fly models.
It might sound unexpected, but fruit flies are widely used in neurological research. They share important biological processes with humans, particularly in how brain cells develop and function. They’re also quick to study and easier to genetically adapt, which means researchers can explore genetic changes much faster.
In NARS1 research, this allows scientists to:
Measure seizure activity
Observe how seizures affect the body and recovery
Study movement and behaviour
Understand how genetic variants impact brain and body functions
This stage of research helps answer some of the most important early questions:
What is going wrong?
Why are these symptoms happening?
It gives researchers direction, showing which biological pathways are affected and where potential treatments might need to act or what symptoms may be improved or resolved.
The mouse (Mus Musculus) model: a key step forward
Alongside this, we’ve also reached an important milestone in our mouse model research.
Through our partnership with The Jackson Laboratory, one of our NARS1 mouse models has now entered Phase 2 breeding.
This means researchers are now producing mice that carry two NARS1 variants, allowing them to study the condition in a whole living system. And this is where things start to come together.
While flies help us understand biology at an early stage, mouse models allow researchers to see how that biology plays out across the brain and body over time in mammals, which is more similar to our body systems..
They can:
Track how the condition develops
Study symptoms like seizures, movement, and behavior in a complex system
Explore whether treatments could make a meaningful difference
The next step is viability, the mice need to live long enough for researchers to properly observe and measure what’s happening. Phenotyping or observing symptoms in mice can take 6 months to a year to gather enough data in enough individual mice to prove consistency.
If successful, this opens the door to therapeutic testing, especially genetic-based therapeutics, which require more information before using in humans.
Why both models are important
These models don’t replace each other, they work together.
Fruit flies help researchers move quickly and understand the basics
Mouse models allow those findings to be tested in a way that’s much closer to the human body
It’s a step-by-step process:
Understanding the biology → testing in a living system → exploring treatments
There isn’t a shortcut around this.
What this means for families
This doesn’t mean a treatment is ready. And it doesn’t mean a clinical trial is just around the corner. But it does mean something important:
We are building the foundations properly.
Researchers now have the tools to:
Understand NARS1 in much more detail
Study how it develops over time
Begin exploring what could change that biology
Every step like this reduces uncertainty and moves research forward in a meaningful way.
The reality behind the research
This kind of work is complex, and it’s expensive.
Creating a single mouse model can cost around $45,000 per genetic variant
Detailed testing (phenotyping) can range from $100,000 to $250,000, depending on the depth of study
It’s a significant investment. But it’s also what makes the next stage of research possible.
Without these models, we simply wouldn’t be able to test treatments safely or understand whether they have real potential.
Where fundraising fits in
Everything we’ve shared here, from fruit fly research through to mouse models, is only possible because of funding - whether your generous donations or grants.
It’s not always the most visible part of the journey. But it’s one of the most important. Because this is how progress happens.