The Alzheimer's Drug Discovery Foundation (ADDF) announces two investments in potential novel treatments for Alzheimer's disease and frontotemporal degeneration. The two programs are targeting mitochondria and the progranulin gene, which have been implicated in the development of these types of dementia.
Dr. Howard Fillit, Founding Executive Director and Chief Science Officer of the ADDF, says: "The ADDF was founded on the belief that new ideas for treating dementia needed support. We have never wavered in our commitment to funding novel approaches to Alzheimer’s and the other causes of dementia."
Steven Finkbeiner, MD, PhD, The J. David Gladstone Institutes
Novel Human FTLD Neuron and Microglia Cell Models for Drug Discovery
Mutations in the progranulin gene are one of the most common causes of frontotemporal degeneration (FTD), a rare type of dementia. These mutations reduce levels of functional progranulin protein, which increases inflammation in the brain and can result in cell death. The FTD field lacks robust cellular platforms that represent human disease biology for drug discovery research. Dr. Finkbeiner plans to create human cell models to mimic the complex biology observed in patients with progranulin mutations. With this funding, he will generate neurons and microglia (the brain's immune cells) using skin cells from FTD patients with progranulin mutations. He and his team will then use the cell models to help identify potential new drugs targeting progranulin mutations. This project is co-funded through the ADDF's partnership with the Association for Frontotemporal Degeneration, now in its 11th year.
Eugenia Trushina, PhD, Mayo Clinic Rochester
Small Molecule Inhibitors of Mitochondrial Complex I for Treatment of Alzheimer's Disease
Mitochondria are the energy powerhouses of cells. Abnormal mitochondrial function has been shown to contribute to the onset of Alzheimer's disease. Dr. Trushina is developing novel drugs to restore mitochondrial function in diseased neurons. With ADDF funding over the last several years, she has translated her basic research findings into a large-scale drug discovery project. Dr. Trushina will now work with Dr. Lars Knutsen, an experienced medicinal chemist, on the next stages of drug development. They plan to test novel optimized molecules that target mitochondrial dysfunction for potential as effective Alzheimer’s treatments. This will enable Dr. Trushina to move the most promising compounds forward toward the goal of initiating human clinical trials in the next two to three years.