Breakthrough represents a new hope for HD patients

Scientists from the Weizmann Institute in Israel have published the results of an animal study which holds tremendous promise for the treatment of Huntington’s Disease.  They have discovered that two small molecules, called SPI-24 and SPI-77, can effectively reduce mutant huntingtin expression in the brain while leaving the normal gene expression unaffected. These compounds traverse the blood-brain barrier, making them suitable for direct delivery to the brain through oral administration or subcutaneous injection. 

In a mouse model SPI-24 and SPI-77 administration resulted in improved motor and emotional functions as well as a delay in disease progression. Importantly, long-term use has shown no adverse side effects or global changes in gene expression. 

In announcing the results, Professor Rivka Dikstein of the Weizmann Institute said “These findings suggest that the SPIs are superior to other Htt-lowering approaches in terms of mutant allele selectivity, delivery methods (no surgery), and cost”. IHA Present Svein Olaf Olsen welcomed the results, saying “This is great news indeed. Although it’s early days, these findings are tremendously important and will give enormous hope to HD families.”   

Link to the press release: https://wis-wander.weizmann.ac.il/life-sciences/toward-treatment-huntington%E2%80%99s-disease

Link to the research article: https://www.embopress.org/doi/full/10.1038/s44321-023-00020-y