There is an urgent need to develop novel and effective therapies for patients with ALS. Stem cell transplantation and gene therapy (i.e., insertion of a protective gene into living neurons using a flu-like virus) techniques are two promising new strategies. The delivery of stem cells and gene therapy to the brain remains challenging as the blood-brain barrier (BBB) limits the passage of large molecules to the central nervous system. This barrier works as a protective barrier to prevent the passage of unwanted substances in the bloodstream into the brain.
Evaluate the safety and the precise technical parameters of FUS to reversibly open the BBB of the motor cortex of patients with ALS. We also plan to test and validate novel biomarkers, including specific bloodwork and electrophysiological test (called MUNIX), which are promising tools to better demonstrate a potential effect of a treatment earlier than the physical examination. This stage is currently under regulatory assessment and we expect to start recruiting patients in 2017 after approval is obtained and funding is secured. .
Develop and validate a reliable, safe and potentially efficacious source of stem cell (such as mesenchymal stem cells). Experiments using ALS animal models may be required in this stage to assess stem cell dosing and specific parameters of delivery.
World-first, proof-of-concept study using FUS to deliver stem cells to the brain of patients with ALS. Along with the results of stage 1A and 1B, we would have the necessary data to design this human interventional trial using the most promising delivery tool and biotherapeutic approach for humans with ALS.
Develop viral vectors with transgenic neurotrophic factor genes to support neurons and glia cells, which are affected by ALS. These vectors will be tested in ALS animal models using FUS to allow for the targeted delivery of these biotherapeutics to the brain. This initiative will provide the necessary preclinical data before possible translation of gene therapy to humans with ALS.
Statements in this plan other than historical data and information constitute "forward-looking statements" and involve uncertainties regarding study processes and results. The final approved protocol may differ from this plan.