Macquarie University Leads 4-Site Australian Study Focusing on Amyotrophic Lateral Sclerosis


A Macquarie University investigator is leading a Phase 2/3 clinical trial that enrolled its first patient in a multi-center, randomized, double-blinded, placebo-controlled clinical trial to evaluate the efficacy and safety of CuATSM in subjects with Amyotrophic Lateral Sclerosis (ALS)—also known as “Motor Neuron Disease (MND)” and Lou Gehrig’s Disease. The Sponsor, a small biotech venture called Collaborative Medicinal Medicine LLC (CMD), is based in Australia and Mill Valley, CA, and seeks to enroll 80 patients across four sites in Australia. 

The Study

The study will assess the effects of 6-months treatment of the sponsor’s investigational product CuATSM compared with placebo on disease severity measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and on cognitive function measured by the Edinburgh Cognitive and Behavioral ALS Screen (ECAS). The study lasts 18 months and TrialSite News will track until the end and report results.

The Measurements

ALSFRS-R is a validated rating instrument for monitoring the progression of patient disability with ALS. ALSFRS did have some weaknesses—it was afforded disproportionate weighting to limb and bulbar, as compared to respiratory, dysfunction. Hence, the revised model incorporated additional assessments of dyspnea, orthopnea and need for ventilatory support. And as reported by a study commissioned by biopharmaceutical company Regeneron, it also retains the properties of the original scale and shows strong internal consistency and construct validity.

ECAS was designed by Sharon Abrahams, Thomas Bak, and Judy Newton affiliated with University of Edinburgh. It represents a brief multidomain assessment originally designed for people with ALS but useful for other disorders as well.

Principal Investigator Comments

Dominic Rowe, MD, Professor of Neurology at Macquarie University and Coordinating Principal Investigator states “We are excited to have enrolled the first patient in this study. Working together with our patients and the CMD team we are optimistic that this may be the first effective disease-modifying treatment for ALS.”

Who is Collaborative Medicine Development LLC

Collaborative Medicine Development is a clinical stage company developing novel disease modifying drugs for the treatment of both neurodegenerative diseases and cancer. We are building our portfolio by both inventing new chemical entities in house and licensing promising leads from third parties.They are a privately-held US/Australian-based “early-stage” pharmaceutical group dedicated to “breaking the mold” with respect to neurodegenerative disease and cancer. Based on our research, we suspect that the company has under 20 employees and has limited financing seeking out grants where feasible, such as a $1 million grant recently won from FightMND.

What is CuATSM

CuATSM is a first-in-class, orally bioavailable, brain-penetrate small molecule. The potential of CuATSM for treatment of ALS was first demonstrated in five mouse models of ALS. Positive results in the most aggressive mouse model of ALS were replicated by three independent investigators. CuATSM has evidenced success in Phase I study. The potential of CuATSM for treatment of Parkinson’s disease (“PD”) also has been demonstrated in four mouse models of PD A phase 0 clinical trial using 62CuATSM demonstrated target engagement in the substantia nigra in patients with PD. A recently completed phase 1/2a open-label study in patients with PD showed 6-months treatment with CuATSM was associated with marked improvements in disease severity measured by UPDRS score and quality of life measured by PDQ-39 score.

Ann & Robert H. Lurie Children’s Hospital of Chicago

Research at Ann & Robert H. Lurie Children’s Hospital of Chicago is conducted through the Stanley Manne Children’s Research Institute. The Manne Research Institute is focused on improving child health, transforming pediatric medicine and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children’s is ranked as one of the nation’s top children’s hospitals by U.S. News & World Report. It is the pediatric training ground for Northwestern University Feinberg School of Medicine. Last year, the hospital served more than 220,000 children from 48 states and 49 countries.

Lead Research/Investigator

Dominic Rowe, MD Professor of Neurology at Macquarie University

Call to Action: For those interested in monitoring ALS, this may be an important study. TrialSite News will track its progress.