$125,000 awarded to Dr. Agessandro Abrahao, Sunnybrook Health Sciences Centre, in collaboration with Dr. Lorne Zinman, Dr. Nir Lipsman, Sunnybrook Health Sciences Centre, Dr. Kullervo Hynynen, Dr. Simon Graham, Dr. Jamie Near, Sunnybrook Research Institute, Dr. Sanjay Kalra, Dr. Kelvin Jones, University of Alberta, Dr. Isabelle Aubert, and Dr. Sonam Dubey, Sunnybrook Research Institute.
CNM-Au8, developed by Clene Nanomedicine Inc., is an oral liquid suspension of gold nanocrystals that is being investigated as a possible treatment for ALS. In preclinical ALS models in the lab, CNM-Au8 was shown to improve motor neuron function and survival by increasing energy production within cells and lowering oxidative stress. The efficacy of CNM-Au8 as a potential treatment for ALS was previously studied in a Phase 2 clinical trial, called RESCUE-ALS. Although topline results revealed the trial did not meet its primary and secondary endpoints, the company indicated they were pleased with the results which show a potential for efficacy and as such feel it is important to confirm the results in a larger trial. CNM-Au8 is also currently being tested in the HEALEY ALS Platform Trial with topline results expected in the second half of 2022.
With this grant, Dr. Abrahao and his team propose to conduct a Phase 2a clinical trial investigating CNM-Au8 coupled together with a technique called MR-guided focused ultrasound (MRgFUS). Previously supported with a 2017 ALS Canada Project Grant, the team has shown that MRgFUS can safely and temporarily enhance permeability of the blood-brain barrier (BBB), a specialized barrier that protects the brain from substances in the bloodstream including some drugs. Researchers are hopeful that delivering CNM-Au8 alongside MRgFUS will allow for more of the investigational drug to reach affected motor neurons in the brain.
This study will be the first to investigate MRgFUS in conjunction with an experimental ALS therapy. The clinical trial, which aims to recruit 10 volunteers with ALS, will be open label meaning every participant enrolled will receive the active treatment for up to 8 weeks. Participants will be followed to assess the safety and feasibility of the proposed treatment, as well as to measure whether there is enhanced delivery of CNM-Au8 to the brain and how the body breaks down the drug internally. This proof-of-concept study is a promising next step in developing a non-invasive way to deliver ALS therapeutics to the brain.