Huntington’s Disease (HD) is a severe genetic disease that causes progressive death of nerve cells in the brain. HD results in a loss of a person’s physical and mental abilities and there are currently very limited treatment options and no curative treatments. AstraZeneca are working on diverse approaches to evaluate/assess new potential treatment options for HD, including efforts to decrease the harmful Huntingtin protein itself and strategies to alleviate the detrimental response of the brain in HD.
A mutation within the Huntingtin gene causes neurodegeneration in HD. Each one of us carries a version of the Huntingtin gene, but only those with a large, expanded version of the gene develop Huntington’s Disease. We are working hard to understand how such a mutation causes degeneration of the brain, and how we might intervene to provide vital treatment options to patients.
There are currently no treatments or therapies that slow or prevent the decline of brain function in Huntington’s Disease, but there are many promising research avenues that are shedding light on how the disease develops that provide exciting areas to drive the development of new medicines.
Our approach to HD is multi-faceted, with approaches seeking to reduce the mutant Huntingtin protein known to cause degeneration in HD, approaches to block cellular responses to mutant Huntingtin that cause neurodegeneration, and gene therapy approaches to directly intervene in the disease-causing gene itself.