Scientists receive funding for development of MND treatment

A funding boost of more than £78 million will help scientists working to develop a potential treatment for motor neurone disease (MND).

Amyotrophic lateral sclerosis (ALS) is the most common form of MND and there is currently no cure for the condition which affects 5,000 people in the UK.

Trace Neuroscience, a new biopharmaceutical company, is working on developing a way to restore the production of a protein that is essential for communication in the brain and spinal cord.

It was founded on the “breakthrough discovery” that loss of the protein called UNC13A is a driver of disease in nearly all people with ALS.

The condition is a rapidly progressive neurodegenerative disorder leading to wasting, paralysis, and eventual death from respiratory failure within three to five years.

Experts suggest that by restoring UNC13A protein levels, healthy communication between nerves and muscle cells impacted by neurodegenerative disease can be re-established thereby slowing the progression of the disease.

Pietro Fratta, professor of cellular and molecular neuroscience at UCL Queen Square Institute of Neurology, said: “UNC13A is critical for neurons to communicate amongst each other and with muscles, and is lost in nearly all ALS cases.

“Being able to re-establish this is groundbreaking.

“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, said: “Trace Neuroscience was founded on the breakthrough discovery that loss of UNC13A is a driver of disease in nearly all people with ALS.

“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.”

Trace Neuroscience licensed intellectual property exclusively from UCL as part of the company formation, and the 101 million-dollar USD financing was led by Third Rock Ventures and included participation from Atlas Venture, GV and RA Capital Management.

UCL Business (UCLB) supported the academic team working closely to protect the intellectual property and facilitate the collaboration.

UCLB senior business manager Dr Caitriona O’Rourke, said the funding announcement is a “huge milestone”, but added that it is still very early in the study.

She added: “I’m optimistic about what comes next and look forward to following Trace as it progresses into clinical trials.”

Director of research development at the MND Association Dr Brian Dickie said: “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.”