Science advances by collaborations and publications. Over the last decade, IRBM has done significant research into neurodegenerative diseases such as Huntington’s disease (HD), often with our excellent collaborators at the CHDI Foundation, and other esteemed institutions. Our paper in J. MedChem, with CHDI and researchers in the Huntington communicaty, outlines how a Huntingtons disease (HD) imaging biomarker for use in positron emission tomography (PET) was identified. This is another step forward helping CHDI achieve its mission to rapidly develop therapeutics that slow the progression of HD.
To develop therapies, it helps to understand a disease, and its progression. One way to do this is through imaging technology such as PET. The paper outlines how a HD imaging biomarker for use in PET was identified. HD is characterised by protein aggregates due to misfolding in mutant huntingtin (mHTT) protein. Through a series of systematic structural activity studies, coupled with tests on mouse and human brain tissue samples, a novel high-affinity ligand specific for mHTT aggregates was discovered. This compound is now being advanced to human clinical trials as a first-in-class HD PET radiotracer.