Revolutionary treatment for motor neurone disease on the horizon

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

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.

READ MORE: Rugby league player Rob Burrow dies age 41 after battle with motor neurone 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.”

Motor Neurone Disease hit the headlines when former Rugby League England international Rob Burrow died after a lengthy battle with the disease at the age of 41. Burrow spent his entire rugby league career with Leeds Rhinos and helped them win eight Super League titles.

Eric Green, co-founder and CEO 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 USD financing was led by Third Rock Ventures and included participation from Atlas Venture, GV and RA Capital Management.