Before a potential FTD drug is tested in clinical trials, it is evaluated in cell or animal models. This is done to confirm that it enters the brain in sufficient amounts, and to identify warning signs of adverse reactions, determine the optimal dose, and demonstrate that it is effective in live animals. Since 2007, AFTD and the Alzheimer’s Drug Discovery Foundation have partnered to fund the Accelerating Drug Discovery for FTD grant program, which supports preclinical research on promising new or repurposed drugs to treat FTD.

AFTD is proud to announce the recipients of the 2020 Accelerating Drug Discovery for FTD awards:

Wen-Hsuan Chang, PhD, the Head of Research for AcuraStem Incorporated, a California-based biotechnology company, received an award for her proposal, “Development of PIKFYVE antisense oligonucleotides (ASO) treatment for FTD.” Dr. Chang will use her award to test an antisense oligonucleotide (ASO) that targets the enzyme PIKFYVE in two mouse models of FTD. Based on previous studies, she believes that blocking the enzyme will increase the clearance of damaged or misshapen proteins, such as tau and TDP-43, to prevent them from accumulating and damaging brain cells.

Anna Orr, PhD, an assistant professor of neuroscience at Cornell University, received an award for her proposal, “Lead optimization of novel site-selective blockers of mitochondrial complex III ROS as FTD therapeutics.” She and her co-investigators, Drs. Adam Orr and Subhash Sinha, will further develop a group of compounds her team developed known as S3QELs, which block harmful free radicals in brain cells that can trigger protein accumulation and inflammation. By improving the chemical and pharmacological features of these compounds, Dr. Orr and her team hope to advance a promising new approach to treating FTD.