The Florey Institute of Neuroscience (FIN), one of the world’s largest and most respected brain research centers, recently announced trial results for the drug CuATSM, developed by FIN, the School of Chemistry and Bio21 Institute at the University of Melbourne for treatment of motor neurone disease, also called amyotrophic lateral sclerosis (ALS).
One of the main characteristics of ALS is the death of the brain cells that control muscle movement. It’s known to be a progressive, fatal neurodegenerative disease that leads to muscle weakness, paralysis and eventually to death. There is currently not sufficient treatment or disease-modifying therapies available. To learn more about ALS, click here.
The Phase 1 single and multiple (oral) dose escalating study consisted of 32 ALS patients. Lung function and cognitive ability improved in the group of patients treated with the highest amount of the CuATSM compared to expected declines in standard-of-care patients.
According to measurement by a global disability score, patients treated with CuATSM presented a much slower disease progression. To learn more about the study, click here.
The trial was sponsored by Collaborative Medicinal Development, LLC with support from FightMND. Once the therapeutic potential was evident through testing with mice, the researchers founded Collaborative Medicinal Development, which was subsequently licensed to move forward into human studies.