Current drug programs targeting Huntington’s disease utilize subjective measures to evaluate a patient’s overall physical and neurological health to measure efficacy. However, symptoms such as motor and cognitive impairment can vary from day-to-day. “The use of objective biomarkers to assess Huntington’s Disease in clinical trials will enable us to more accurately determine if treatments are effective. sRNAlytics’ discovery of an objective biomarker will help transform the way we think about therapeutic development and treating patients,” said Dr. Blair Leavitt, Professor at the University of British Columbia.
In this recent study, sRNAlytics used their sRNA-FIND platform to discover small RNA biomarkers in the brain tissue of deceased Huntington’s patients. In collaboration with researchers at the Boston University Medical School, they showed that these biomarkers could monitor Huntington’s disease progression 20 years prior to symptomatic onset in a liquid biopsy. Additional work showed correlations with neuronal cell death and loss of motor functions in patients. Dr. Richard Myers, a Professor of Neurology and study lead commented: “The small RNA profiles that sRNAlytics identified represent the most promising approach to test efficacy that I have seen to date. The FDA’s recognition of these findings is an important step forward in validating their technology and accelerating their future use in clinical trials.”
Source: Business Wire