Our Story

The Paradigm

Despite decades of research, Amyotrophic Lateral Sclerosis, or ALS – the most common type of motor neuron disease – remains a painfully elusive target.

ALS affects an estimated 350,000 worldwide and is expected to increase in the coming years due to the ageing population.

The root cause is understood in the 10% of ALS patients with “monogenic” disease. Only about 3% of these patients can potentially access a therapy option.  But for “sporadic” cases – the other 90%, the needle has barely moved therapeutically.

Beyond a lack of therapies, there is no definitive, non-invasive diagnostic method for ALS – especially in the early stages. There have been considerable efforts to identify biomarkers that would allow rapid, non-invasive and accurate diagnosis, but with limited success to date. 

Most investigations for ALS and other neurodegenerative diseases (NDDs) have centered on where the damage can be observed – in the brain and central nervous system. 

What if the damage originates somewhere else?

The Shift

It began with an unexpected observation...

…which has led to a fundamental change in understanding the origins of ALS  and brought groundbreaking therapeutic and diagnostic opportunities for this devastating disease, with potential implications for other NDDs, including Alzheimer’s and Parkinson’s.

Vesalic has characterised a previously unknown systemic metabolic dysfunction that creates a toxic exosome cargo in ALS patients, which is carried to the CNS, where it binds to and damages neurons. 

  • The significance: a previously unknown druggable target with disease-modifying potential for ALS

In addition, Vesalic discovered a disease-specific alteration of the lipid composition in the membranes of exosomes circulating in the blood. 

  • The significance: a novel, blood-based biomarker that could enable quicker, more accurate detection of ALS

Learn more about our pioneering discoveries and therapeutic and diagnostic opportunities. 

Our Science

Our Story