Healthcare (Commonwealth Union) – Neurological conditions continue to be devastating for seniors. Among them motor neurone disease (MND) or ALS is one of the most concerning.
A groundbreaking experimental therapy for MND, produced by the University College London (UCL) spinout Trace Neuroscience, is set to enter human testing through a new global clinical trial programme.
The biopharmaceutical company, co-founded by Professor Pietro Fratta of the UCL Queen Square Institute of Neurology, has launched two clinical studies investigating TRCN-1023, a potential treatment for MND, also referred to as amyotrophic lateral sclerosis (ALS), the most prevalent form of the condition.
The innovative investigational medicine has been developed to address UNC13A, a key genetic factor believed to play a major role in the development of MND/ALS.
The drug’s advancement is based on Professor Fratta’s research at UCL, which helped reveal the crucial role of the UNC13A protein in MND/ALS progression. The discovery has since positioned UNC13A as one of the leading therapeutic targets being explored for treating the disease.
The new clinical programme brings together two early-stage trials aimed at advancing the development of TRCN-1023 and generating initial evidence from patients worldwide. FUNCTION ALS, a Phase 1/2 study approved to begin in the UK, is expected to expand into additional countries in 2026, while LAUNCH ALS, an investigator-led trial already taking place in China, has begun dosing its first participants. Together, the studies are intended to speed up the drug’s clinical development and provide early insights into its potential as a treatment for MND/ALS.
MND/ALS is a devastating and ultimately fatal neurological disorder in which motor neurons in the brain and spinal cord progressively deteriorate, leading to worsening muscle weakness. Over time, people living with the condition can lose the ability to move, communicate, swallow and breathe. The disease is estimated to affect around one in 300 people during their lifetime, and despite ongoing research efforts, there are currently no approved disease-modifying treatments that can halt its progression.
UNC13 loss in about 97% of people with MND/ALS, an activity is believed to contribute to disease progression, making it one of the most important possible therapeutic targets in the field.
TRCN-1023 is an antisense oligonucleotide (ASO) therapy intended to restore normal production of the UNC13A protein, which is important for communication between nerve cells and muscles.
Sometimes called a “genetic medicine,” TRCN-1023 is not a traditional gene therapy as it does not make permanent changes to a person’s DNA. Instead, the treatment targets RNA, the molecule that conveys instructions from genes to help make proteins.
ASOs are short stretches of genetic material that scientists build in the lab. They bind to particular RNA molecules inside cells, changing how certain proteins are made. TRCN-1023 is designed to correct how UNC13A RNA is processed so that cells can produce a functional UNC13A protein.
TRCN-1023 is delivered through an intrathecal injection, a method that introduces the medicine directly into the cerebrospinal fluid surrounding the spinal cord.
This delivery technique is widely used for antisense oligonucleotide (ASO) treatments, as it enables the therapy to access the central nervous system, the area primarily affected by MND/ALS.
Professor Fratta indicated that UNC13A plays a vital role in allowing neurons to communicate with one another and connect with muscles, and its function is disrupted in almost all cases of MND/ALS.
“Our research at UCL uncovered just how central this protein is to the disease process and developed ASOs to rescue it and restore neuronal function – making it one of the most compelling and widely relevant targets for new treatments.
“Seeing this discovery translated into a clinical trial is a major milestone. It brings us a step closer to a therapy that could make a meaningful difference for the vast majority of people living with MND/ALS.”
