According to a story from BioPortfolio, the biopharmaceutical company Collaborative Medicinal Development, LLC has recently announced that the first patient has been enrolled in a clinical trial that will test the company’s investigational drug CuATSM in patients with amyotrophic lateral sclerosis, a rare and fatal disease. Collaborative Medicinal Development is prioritizing the development of drug candidates for neuropsychiatric and neurological diseases.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of the illness, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about this rare disease, click here.
About The Trial
The trial is expected to include a total of four different sites across Australia and will enroll 80 patients. The trial will compare outcomes of six months of treatment with CuATSM against placebo. Changes in patients will be assessed using two different measurement tools. One will be the Edinburgh Cognitive and Behavioral ALS Screen (ECAS), which will measure changes in cognitive function; the other is the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R), which will evaluate motor function.
About CuATSM
CuATSM is an orally active small molecule drug that is capable of penetrating the blood-brain barrier. It is the first drug of its class. Early trials with both humans and mouse models have demonstrated that the drug may have potential in the treatment of amyotrophic lateral sclerosis and Parkinson’s disease. CuATSM has also earned Orphan Drug designation from the US Food and Drug Administration (FDA).
This clinical trial has been supported by FightMND, a non-profit organization that is committed to finding a cure for amyotrophic lateral sclerosis. Learn more about this organization here.
