Challenge: Of the top 10 most fatal illnesses in the world, Alzheimer’s disease is the only one for which there is currently no disease-modifying therapy available. Alzheimer’s disease becomes progressively more prevalent as people age, rising from over 11% of the population 65 and older to over 33% of those aged 85 and older. This devastating and deadly illness is becoming an increasingly urgent problem as the worldwide population ages.
Toxic amyloid proteins are known to be a significant contributing factor in the development of Alzheimer’s disease. Abnormal clusters of protein fragments build up between nerve cells in the brainleading to impaired memory. The most important therapies in development currently are biologics, antibodies and peptides that bind and neutralize toxic amyloid. However, delivering sufficient quantities of a safe and effective amyloid-targeting biologic across the blood-brain barrier in humans has not yet been possible.
Solution: KalGene Pharmaceuticals is developing an Alzheimer’s disease therapy based on a naturally-occurring agent that binds specifically to toxic amyloid-beta. When coupled with a blood-brain-barrier carrier molecule developed at the National Research Council of Canada, the compound has been successfully delivered into the brain in Alzheimer’s disease models. KalGene’s project with the Montreal Neurological Institute and the Centre for Imaging Technology Commercialization will proceed to demonstrate this technology in human Alzheimer’s disease patients.
Impact: An effective Alzheimer’s treatment would have profound personal impact for millions of people and their caregivers worldwide, and would transform the trajectory of healthcare costs. Alzheimer’s disease is one of the most feared health diagnoses. An approach to mitigate the progression of Alzheimer’s diseasewould bring hope to patients, families and individuals at high risk. The total economic burden of dementia care in Canada alone is expected to rise from $14 billion in 2008 to $75 billion in 2028 with more than half of that attributable to Alzheimer’s disease.
This project will also accelerate therapies for other brain conditions beyond Alzheimer’s disease. The difficulty of delivering drugs across the blood-brain barrier has profoundly limited the development of effective therapies for diseases such as brain cancers and Parkinson’s disease. By demonstrating that the blood-brain-barrier carrier technology is effective in delivering an Alzheimer’s therapy in humans, the project will provide a path to develop brain-penetrant therapeutics for other neurological diseases.