Delivery of genetic therapies to the brain is one of the biggest challenges facing researchers in many fields of neurological disease, including HD. One novel approach, tested only experimentally in mice, is to use a type of harmless virus that can cross from the blood into the brain. Researchers at the University of North Carolina School of Medicine are studying the best ways to adapt these tiny viruses so that someday, safe, low-dose delivery of genetic therapies to the brain might be possible through an IV. While these experiments are not directly related to HD, rest assured that researchers in the field are quick to embrace new technologies and apply them to the development of HD therapies.
Huntingtin-lowering and Hope
A personal story from a participant in the Ionis trial and a discussion of ongoing approaches in HD drug discovery provide a hopeful account of the technological leaps in the field and highlight the importance of clinical research participation.
This week the FDA released a statement acknowledging the urgent need for medical treatments for neurological disorders, sharing their intention to include more patient and family input, and introducing measures to modernize the way they communicate about drug development guidelines. Newly released documents incorporating input from doctors, patients, researchers and advocates have laid out expectations for therapy design across five major brain diseases. Although Huntington’s Disease is not included in the initial release, this lays the groundwork for appropriate trial design and speedier approval of novel therapies for many neurological disorders, and more guidelines will follow. HDSA-backed efforts towards advocacy on Capitol Hill, within industry through HD-COPE, and in partnership with other rare disease organizations will continue to ensure community visibility and engagement that garners FDA attention.