AFTD is proud to announce the most recent recipients of our Pilot Grants, which provide seed funding to promising FTD researchers: Andrew Arrant, PhD, of the University of Alabama at Birmingham, and David Butler, PhD, of the Neural Stem Cell Institute at the Regenerative Research Foundation.

Dr. Arrant was awarded the 2019 Basic Science Pilot Grant for his proposed investigation into a common cause of genetic FTD: mutations in the progranulin gene. These mutations result in a loss of the progranulin protein the gene encodes, and a corresponding loss of its many protective actions in the brain. Therapies that restore its levels could be effective in FTD – if they target the specific function that’s most critical to preventing degeneration.

Dr. Arrant’s project will tackle this question by modifying progranulin to create a form that acts only on lysosomes, structures that are part of the cell’s waste disposal system. As a result, they’ll be able to determine if this is the key to progranulin’s ability to protect brain cells. The answer could guide drug development toward more precise treatments for progranulin-associated FTD.

Dr. Butler, recipient of the 2019 Susan Marcus Translational Research Pilot Grant, will evaluate a unique type of antibodies, known as intrabodies, to determine if they can be used to reduce the abnormal accumulation of the protein tau that can occur in FTD.

Unlike naturally occurring antibodies, which act outside the cell, intrabodies are laboratory-engineered antibodies that remain inside cells and can be further modified to create designer proteins with additional desirable features. Dr. Butler’s intrabodies will bind to tau and also have a molecular “tag” to direct the bound protein to the cell’s waste disposal system. He will test his intrabodies in a novel 3-D cell culture model; if they are effective, he hopes to ultimately develop them as anti-tau therapies.