Huntington's Disease Culprits Identified

HDAC enzymes underly Huntingtons disease

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) Huntington's disease is stubborn. It clings to a genetic defect passed down through families, and no current treatment slows its progression. But a new discovery may change that.

Researchers have identified a potentially powerful new target for drugs that could have an effect on the disease. Enzymes called HDACs, or histone deacetylase complexes, have been discovered to underlie the cell mutation that drives Huntington's disease.

Inhibiting, or blocking, these enzymes could reduce cell mutation and slow the progression of the disease.

"Ask your doctor about treatments for Huntington's disease."

Professor Robert Lahue of National University of Ireland Galway's Centre for Chromosome Biology was the lead author of the paper, which was published in the online journal PloS Biology.

Huntington's disease is caused by an inherited genetic defect on a chromosome, and many patients have seen it appear in other members of their families. If one of your parents has Huntington's, there's a 50 percent chance of getting the gene that causes Huntington's.

Symptoms of Huntington's can appear in the patient's mid-30s or early 40s. The disease appears earlier and earlier the farther it is passed down a family line.

The defective gene causes a section of DNA to repeat over and over again, more times than it does in a normal person. The effect is that nerve cells waste away, and it manifests in behavioral, physical, and mental changes until death.

One hundred thousand people suffer from the disease, and no treatments are available to stop or even slow its progression.

That's why this new research is so significant. The scientists found that when the HDAC enzymes are active, they exacerbate the cellular mutations that cause the disease.

They theorize that HDACs contribute to the severity of the disease. Using a lab model, they tested experimental drugs to inhibit HDACs and found that the risk of mutation can be reduced.

That means that future drugs may be able to target HDACs in Huntington's patients, and have an effect on the progression of the disease.

Many labs are working in this vein, trying out potential inhibitors for HDACs in Huntington's. A drug is a ways off from development.

The research was supported by Science Foundation Ireland and Millenium Fund of National University of Ireland, Galway, in conjunction with other research grants. The paper was published in February 2012.

Reviewed by: 
Review Date: 
February 28, 2012
Last Updated:
February 29, 2012