May 15 was Huntington’s Disease Awareness Day. According to professor Eric Reits, awareness for the disease remains a major issue to be addressed. “The taboo on Huntington’s disease is overwhelming, partly explained by the severity of symptoms - being a combination of Alzheimer, Parkinson and ALS - and the absence of a therapy to even delay onset of disease.” the professor of Cellular Imaging explains.
A day in the life of the professor
Reits’ regular working day consists of three different aspects: research, teaching, and interaction with the Huntington’s disease community. Together with his research team he focuses on the visualization and manipulation of protein degradation in Huntington's disease (HD). “Besides discussing ongoing research, results and ideas, we are starting up the recently awarded NWA consortium CureQ which I coordinate, that aims to use HD patient-derived cells to validate new therapeutic strategies for different groups of patients, and to improve better prediction of the age-of-onset.” Reits elaborates.
Transforming hope to first trials
He gets inspired by the many Huntington family members that Eric Reits personally knows, mainly through his activities for the Campagneteam Huntington and the many meetings and discussions with people at risk or gene carriers, their partners and friends. “Once you understand the huge impact of HD on families, the taboo, and severity of symptoms, but also their hope for a cure and need for awareness for HD, you are highly motivated.” Reits says. “Moreover, the idea that since the gene that causes HD was discovered in 1993, it took 30 years to come to the first trials that recently started, aiming for mutant Huntington lowering, and the identification of various potential therapeutic strategies to delay or prevent HD.”
Optimistic future
The first trials aim to lower synthesis of the mutant protein, and recent insights in the intracellular proteostasis machinery provide leads to improve selective turnover of the mutant protein. There is now real hope among scientists, neurologists and HD families that one or more strategies will succeed and provide a therapy that could even be started before the predicted age-of-onset, thereby delaying or preventing disease. If so, genetic counselling will become a serious option for many at-risk.