Compound Reverses Huntington's Symptoms in Mice

TUESDAY, Sept. 16 (HealthDay News) -- An experimental compound called HDACi 4b reversed Huntington's disease symptoms in mice genetically altered to develop the disease, say researchers at the Scripps Research Institute in California.

Huntington's is an inherited neurological disease that affects people's movement and thinking ability. There is no cure or treatment that can reverse or slow progression of the physical and mental deficits caused by the disease.

"We found that (HDACi 4b) can target the expression of several hundred genes in the brain and reverse the abnormalities caused by a single mutant protein. This suggests that a treatment for Huntington's disease that targets a core pathogenic mechanism might be close at hand -- closer than previously imagined," study author Elizabeth A. Thomas, an assistant professor in department of molecular biology, said in a Scripps news release.

In this study, the mice were given the compound after they first started to develop symptoms of Huntington's. The treatment slowed the loss of body weight and reduction of brain size, and improved their appearance and motor functioning.

"The benefit seen was surprising, and immensely exciting, because it suggests this compound could form the basis of truly relevant therapeutic treatment for Huntington's disease," Thomas said. "The mice that were destined to develop Huntington's disease receiving the treatment did significantly better than the mice who didn't receive the drug."

The findings were published this week in the early edition of the Proceedings of the National Academy of Sciences. The study received funding from Massachusetts-based Repligen Corp., which holds several patent applications related to HDAC (histone deacetylase) inhibitors, a class of agents that include HDACi 4b.

Other HDAC inhibitors produced some beneficial results in test animals but were deemed too toxic.

"[HDACi 4b] proved to be therapeutically superior, as well as less toxic than other HDAC inhibitors that had been tested for Huntington's disease," Thomas said.