Breakthrough discovery in treatment for deadly motor neurone disease

Scientists are set for a £78 million financial injection to advance the development of a groundbreaking treatment for motor neurone disease (MND). Among the various forms of MND, amyotrophic lateral sclerosis (ALS) is most prevalent in the UK, impacting around 5,000 individuals with no known cure so far.

Trace Neuroscience, an emerging biopharmaceutical company, is paving the way with their work on replenishing a key brain and spinal cord communication protein, following the pivotal "breakthrough discovery" of UNC13A protein's role in nearly all ALS cases. The disease rapidly leads to muscle wasting, paralysis, and often death from respiratory failure in three to five years.

The restoration of UNC13A could allow for healthier nerve and muscle cell interaction, potentially slowing the disease's fatal progression. UCL Queen Square Institute of Neurology's Professor Pietro Fratta shared his insight: "UNC13A is critical for neurons to communicate amongst each other and with muscles, and is lost in nearly all ALS cases."

He lauded the approach, stating that "Being able to re-establish this is groundbreaking."

Additionally, he remarked on the significance of the funding, "This large funding round is pivotal for us to rapidly translate this science into a life-changing medicine and advance our lead program toward the clinic."

Eric Green, co-founder and chief executive of Trace Neuroscience, has expressed optimism about the potential for a significant breakthrough in ALS treatment, stating: "Trace Neuroscience was founded on the breakthrough discovery that loss of UNC13A is a driver of disease in nearly all people with ALS."

He further elaborated on the company's mission, saying, "We have now brought together academic leaders, experienced drug developers and a leading syndicate of investors committed to urgently turning this discovery into a medicine."

The company, which has licensed intellectual property exclusively from UCL as part of its formation, recently announced a substantial $101 million USD financing round. The funding effort was spearheaded by Third Rock Ventures and saw contributions from Atlas Venture, GV, and RA Capital Management.

Dr Caitriona O’Rourke, UCLB senior business manager, acknowledged the significance of the funding, calling it a "huge milestone" while also noting the early stage of the research. She conveyed her optimism about the future, adding: "I’m optimistic about what comes next and look forward to following Trace as it progresses into clinical trials."

Dr Brian Dickie, Director of research development at the MND Association, highlighted the importance of the investment for MND research, stating: "This announcement marks a substantial investment into the further development of this specific area of MND research and will allow what we hope will be the rapid development of a promising new approach to treating motor neurone degeneration, targeting a pathological process which has been linked to 97% of all cases of MND."

"It is really exciting to see the direct impact the MND Association’s support is making in the search for effective treatments for this devastating disease."