Category: Edmonton

The ability to accurately measure people’s brain degeneration may help researchers to find much needed biomarkers that are essential to understanding, diagnosing and ultimately treating ALS. Using advanced brain imaging techniques (magnetic resonance imaging, or MRI), this observational study will monitor over time the degree of change that occurs in the brains of people who are living with ALS. Each study participant will have 3 MRI scans over a period of 8 months, along with neurological and cognitive evaluations. This study is led Dr. Sanjay Kalra and will operate within the Canadian ALS Neuroimaging Consortium (CALSNIC), a cross-Canada imaging network funded by the largest-ever grant provided by the ALS Canada research program.

Ibudilast (also referred to as MN-166) is an experimental therapy being developed to treat ALS. This broad target drug is thought to reduce the activity of immune cells in the brain, thereby supressing inflammation. It is also believed to promote the production of neurotrophic factors which play a role in the growth and survival of motor neurons. The Phase 2b/3 COMBAT-ALS clinical trial will enroll 230 participants living with ALS and last 12 months. Researchers will monitor participants to ensure that the drug is safe. Researchers will also evaluate the impact of ibudilast on the progression of ALS by evaluating changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, as well as muscle strength, quality of life, and respiratory function. Ibudilast will be taken by mouth in combination with a dose of riluzole. Researchers believe the anti-inflammatory and neuroprotective characteristics of ibudilast make it a promising treatment option for ALS.

To learn more, please click here to view a webinar hosted by the study sponsor, MediciNova.

Antibodies are proteins that are produced by the immune system to protect the body against foreign invaders like bacteria and viruses, and work by binding to specific proteins on the harmful agents and triggering their removal and/or destruction. In this Phase 2 clinical trial, researchers will be testing the safety of a human antibody (called AP-101) designed to target an ALS-linked protein called SOD1. Evidence suggests that the misfolding of SOD1 in cells can cause the protein to take on a toxic gain of function. Researchers are hopeful that targeting this protein may represent a promising strategy for the treatment of ALS. A previous Phase 1 study found the drug to be safe and well-tolerated at the tested doses. In this follow-up study, researchers expect to enroll 63 participants who will be randomly divided to either receive the active drug (AP-101) or placebo intravenously (IV), for 48 weeks. Both individuals with familial, SOD1-ALS and sporadic disease will be eligible to participate. Researchers will monitor participants to ensure that the drug is safe, identify any side effects, determine the appropriate dosage, and learn more about how the body breaks down the drug internally.

Abnormalities in a protein called TDP-43 are present in approximately 97 percent of all ALS cases. Preclinical studies have shown that when the amount of functional TDP-43 is decreased within cells, the level of another protein, STMN2, is substantially decreased. Patient tissues analyzed by researchers also showed that STMN2 levels are lower than expected specifically in motor neurons. These findings support the idea that a reduction in STMN2 resulting from TDP-43 dysfunction contributes to ALS and suggest that methods to preserve the levels of STMN2 within motor neurons may have a therapeutic benefit. QRL-201 is a genetically targeted therapy that aims to restore normal STMN2 levels in people living with ALS. This Phase 1 study will enroll 64 participants who will be randomly assigned to have either the active drug (QRL-201) or placebo delivered into the spinal fluid through a procedure known as an intrathecal injection. Researchers will monitor participants to ensure that the drug is safe, determine the appropriate dosage, and learn more about how the body breaks down the drug internally.