Huntington’s disease, also called Huntington’s chorea, is a rare disease. It is a genetic neurodegenerative disorder that, on average, develops around the age of 40 and is characterized by involuntary muscle movements, cognitive decline and psychiatric problems. For years Huntington’s has been a stigmatized disorder, resulting in manifestations of rage, depression, mood swings as well as uncoordinated movements, dementia and paralysis. There is currently no cure, but research has finally brought a glimmer of hope.
A long journey that includes the efforts of six Italian centers
Italy is among the nations that, in 2019, will begin experimenting with an innovative treatment that has never been tried before. The project is coordinated by University College London (UCL) and has joined the forces of Centers of Excellence both in the United States and in Europe. Participants in Italy include:
- Carlo Besta Neurological Institute in Milan
- University of Genova
- University of Florence
- La Sapienza University in Rome (Sant’Andra)
- University of Bologna
- IRCCS Home for Relief of Suffering in San Giovanni Rotondo, (project coordinator in Italy).
A total of 90 centers will be involved in the care of 660 patients (50-60 in Italy). The study will last 25 months and will use “antisense therapy” to stop the gene from developing the toxic element that causes cell damage. The main objective of the project is to identify a rapid solution to slow the progression of symptoms. Professor Ferdinando Squitieri, head of the Huntington and Rare Diseases Unit at the IRCCS Home for Relief of Suffering Research Hospital and of the Neurology Department at the CSS-Mendel Institute as well as Scientific Officer of the LIRH Foundation, explains that initially the project will only address patients already affected by the disease. In an eventual second phase it will focus on people who carry the gene, but have yet to manifest symptoms. This experimental therapy would directly target the cause of the disease, but would not have effects on every aspect of the disease. Despite these limits, it does represent an important step in fighting Huntington’s disease in hopes that young carriers of the gene can be included in these trials and that, in time and with new treatments, life expectancy in patients will increase.
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