First Steps Being Taken Toward Huntington’s Disease Treatment

In late July of this year, the results from a scientific preclinical trial were published in which a research team in Europe discovered initial signs that could pave the way to a Huntington’s disease treatment.
Huntington’s disease is a rare and potentially fatal neurogenerative disease in which parts of the brain deteriorate from impaired nerve cells.

This deterioration leads to a collection of mental health and cognitive issues. Currently, there is a grim prognosis for Huntington’s disease patients, so a cure, or at least a better treatment, is of great need in the rare disease community. To learn more about Huntington’s disease, click here.

This team, led by Robert Lahue, Ph.D., set out to look at the potential impact of inhibiting histone deactylase 3 (HDAC3). HDAC3 is an enzyme currently believed to change vital biochemical processes in the brain of Huntington’s disease patients.

Specifically, the team used an experimental compound known as RGFP966, a selective inhibitor for HDAC3, on mice. Then, the researchers focused on measuring the decline of cognitive abilities, which in turn were used to evaluate any delayed onset of molecular signs of neurodegeneration.
The results proved that RGFP966 provided rats with a spectrum of benefits to the brain. These data are extremely encouraging because even though they are preliminary, they show that blocking HDAC3 could ultimately delay the onset of Huntington’s disease in suspected patients.

Now, Dr. Lahue and his co-researchers have sent in requests for additional financing that would allow them to continue testing their theory and develop a potential treatment from it. Hopefully, this funding will be granted to the team, so they can further prove the safety and efficacy of RGFP966.

Even so, because there aren’t currently any effective nor safe treatments for this disease that target its root cause, any news on the treatment-front is good news.
To learn more about this preclinical study from Rare Disease Report, click here.

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