What is the Landscape of HD Research and Where does the HDF “Fit”?
Nearly $100 MM a year is dedicated to HD research, worldwide. About half is from the National Institutes of Health (which tends to fund later stage research); and the remainder is from a few private foundations. While each of these funding organizations has a particular focus, and all contribute to research, (from the most basic science levels to human clinical trials), the HDF stands out “for its unbelievably productive track record, singular focus on research to find a cure for the disease, and ability to create a community of scientists dedicated to a common goal”. (Dr. Jim Olson) The HDF is willing to take informed risks with early-stage research, giving purchase to many ideas that other funders would leave aside. Today, their funding is focused on developing RNAi-based treatments that target the mutant HD gene (the one validated target in Huntington’s). Successful RNAi would be a cure. The HDF also encourages trials in HD for a small number of ‘healthiness’ target drugs which are
