More than 200 proteins are affected in Huntington’s disease, researchers reported on Thursday in a study that offers scientists many potential routes to finding treatments for the fatal brain disease.
Tests on fruit flies show that the mutated Huntington’s protein that underlies the disease interacts with 200 other proteins, the researchers report in the Public Library of Science journal PLoS Genetics.
Many of these interactions damage brain cells.
“It’s the gene producing something that seems to interfere with the normal activities of the cell in many, many different places and ways,” Dr. Eugene Oliver, who oversees some Huntington’s disease work at the National Institute for Neurological Disorders and Stroke, said in a telephone interview.
Dr. Juan Botas of the Baylor College of Medicine in Houston, Texas, who worked on the study, said researchers can experiment with the proteins and the genes responsible for their production.
“When you tinker with some of these genes, you find that some of them improve the symptoms. These could be potential therapeutic targets,” Botas said in a statement.
“When you tinker with others, it makes the Huntington’s more aggressive. These might be ones that accelerate the age of disease onset. Not everyone with Huntington’s develops symptoms at the same age.”
No cure, effective treatment
An estimated 30,000 people have Huntington’s disease in the United States alone and it occurs worldwide in about 1 in every 10,000 people.
It is caused by a single copy of a mutated gene and people who inherit it always develop the disease.
Huntington’s can start off with confusion and personality changes, but sufferers later lose the ability to move, think and communicate. They often die from choking, heart failure or infection.
There is no cure or effective treatment.
Botas, a team at privately held Prolexys Pharmaceuticals, in Salt Lake City, Utah, and others studied fruit flies genetically engineered to have a disease that resembles Huntington’s in humans.
They used high-tech screening of genes and proteins to identify the 200 that interact with the mutated Huntington’s gene.
“We are hoping that researchers will look at this study and that those with specific expertise in a particular protein will move forward with their own inquiries,” said Robert Hughes of the Buck Institute for Age Research in Novato, California, who worked on the study.
“It points in the direction for future work, which is important,” said Oliver, who was not involved in the research.