Clinical trials

• Phases 1 and 2 were done in Professor Schapira lab in London by S Mullin. Mullin2020.pdf
• There are additional ongoing studies in Canada and Norway.
• Stephen Pasternak, MD, PhD, FRCPC, Robarts Research Institute, Western University, London, Ontario, Canada, discusses a Phase II trial (NCT02914366) investigating the safety and efficacy of ambroxol in patients with PDD.
• Ambroxol crosses the blood brain barrier, has a 20-year history of safety, and has been used in pregnant mothers with fetuses with respiratory complications.

Mechanism

• Despite the unique characteristics of Ambroxol regarding the stimulation of TFEB, you have to go about 10 years before you see physical symptoms of Alzheimer’s. If you go much later, the system becomes much more difficult to clear. The advances in biomarkers enable us to pick a time, 10 years out.