Researchers will evaluate the long-term therapeutic effect of bryostatin-1 (Neurotrope, New York, NY) for Alzheimer disease (AD) in a new phase 2 clinical study (NCT02221947). An estimated 100 participants will be enrolled in study to evaluate bryostatin treatment in the absence of memantine (Namenda; Allergan, Madison, NJ). The trial will last 6 months and there will be 2 dosing cycles of 11 weeks each.
Inclusion criteria include a prespecified moderately severe AD (MMSE-2 baseline score 14-10) or moderate AD (MMSE-2 baseline score 18-15) diagnosis. Participants who demonstrated the most evidence of benefit in a prior study will also be included. Any sustained cognitive benefit will be measured by the Severe Impairment Battery (SIB) score.
“We believe that the valuable insights gained from our previous studies have provided crucial guidance for our next trial to increase the possibility of observing clinically meaningful benefits for AD patients with concrete evidence of dementia, benefits that may also possibly translate to MS, stroke, and Fragile X, as preclinical studies suggest,” said Daniel Alkon, MD, president and chief scientific officer, Neurotrope.
“We are hopeful that this trial can substantiate certain compelling data with bryostatin from previous trials, which suggests patients could potentially see improvement in their disease which, I believe, would be transformative as an AD treatment,” said George Perry, PhD, professor of biology and chemistry, Semmes Distinguished University Chair in Neurobiology, University of Texas at San Antonio. “When I see this in the context of currently available drugs, I believe bryostatin could chart a new path in how we treat AD.”
Diana Castro, MD
Stefan Nicolau, MD, and Elie Naddaf, MD