Today, a study was published in the journal Scientific Reports
in which a European team of scientists discovered what they claim are encouraging early signs for a potential Huntington’s disease (HD) treatment.
Robert Lahue, Ph.D., professor of biochemistry at the National University of Ireland (NUI) Galway and his team of researchers partnered with scientists from the University of Barcelona to assess the impact of blocking histone deactylase 3 (HDAC3), an enzyme believed to alter important biochemical mechanisms in the brain of HD patients.
HD is a is potentially fatal neurodegenerative disease. Primary symptoms include chorea, or random and sudden, involuntary movements and twisting. HD patients experience deterioration in several areas of the brain due to impaired nerve cells, leading to a collection of mental health and cognitive issues.
The study, conducted in mice, focuses on measuring cognitive decline and a delay in the onset of molecular signs of neurodegeneration in a pre-clinical model of HD by blocking HDAC3 with an experimental compound called RGFP966, a selective HDAC3 inhibitor.
“While these results are preliminary, the data shows that the onset of Huntington’s disease is delayed when HDAC3 (the enzyme) is blocked in this pre-clinical setting,” said Lahue. “This is an encouraging first step because currently there are no effective treatments that target the root cause of the disease.”
The study concluded that using an HDAC3-selective inhibitor to target disease progression early provides an array of benefits to HD mice by averting hippocampal-memory deficiencies and overpowering degeneration in the brain’s striatum.
Lahue and Silvia Ginés, Ph.D., his co-researcher from the University of Barcelona, have applied for additional funding to continue development of this potential treatment and evaluation of the safety and efficacy of it.
For more information on studies for potential HD therapies, follow Rare Disease Report