Safety and efficacy of a novel amyloid-clearing therapeutic KAL-ABP-BBB in early Alzheimer’s disease patients
McGill University, Douglas Hospital Research Centre
The accumulation of the protein amyloid-beta constitutes the major abnormality in the brain of Alzheimer’s disease patients. This award will support a clinical trial for assessing the safety of a novel therapy targeting a fundamental abnormality in the Alzheimer’s disease brain. This clinical trial will be conducted at the Douglas Research Institute and the Montreal Neurological Institute and Hospital, McGill University, in patients who have mild Alzheimer’s disease and test positive for the presence of amyloid in the brain, using positron emission tomography (PET).
Relevance to the acceleration of therapeutics for neurodegenerative diseases of aging
To date, the vast majority of anti-amyloid therapies have not been successful, since they are not capable of reaching the brain in sufficient concentrations. This novel treatment consists of a carrier molecule that crosses the blood-brain barrier fused to a peptide that clears away amyloid-beta deposits once inside the brain. The carrier molecule ‘tricks’ the blood-brain barrier into allowing its entry into the brain, and delivers the anti-amyloid peptide to the site of disease in high concentrations to enable its therapeutic effect. The anti-amyloid peptide is engineered to target the toxic oligomeric forms of the amyloid. By investigating cerebrospinal fluid, plasma, and PET imaging measures, the study aims to demonstrate that the drug crosses into the human brain and promotes amyloid clearance. Both the carrier molecule and disease-fighting peptide employ novel receptor binding mechanisms and were engineered at the National Research Council of Canada.
Clearing amyloid-beta deposits – the major abnormality observed in the brain of Alzheimer’s disease patients – is expected to slow or even stop the progression of the disease. As such this study will provide crucial information necessary to advance this promising therapeutic agent to an effective treatment for Alzheimer’s disease.