A new target for early onset dementia

As just about everyone knows, there is no cure for dementia. There are treatments and ways to delay progress, but it doesn’t get better. For those unfortunate people who suffer from frontotemporal dementia, early onset dementia, which strikes those between the ages of 40 and 64, it’s a particularly cruel dissent of health. The condition is genetic and is caused by the death of cells in the front and sides of the brain and can lead to dramatic changes in personality and behavior as well as physical limitations like the loss of speech.

UCLA scientists have made a breakthrough with this particular kind of brain ailment. They have discovered that a key signaling pathway plays an important role in the progress of the disease. This signaling pathway may provide a new target for treatment and perhaps one day a cure. The results of the study are published in the journal Neuron.

Previous research linked the disease to a mutation in a gene required for the development of a protein called granulin that regulates cell growth and survival. The mutation reduced granulin by half. “Until now, little has been known about granulin’s function in the brain. We wanted to explore whether a granulin shortage kick-starts the cell death that precedes dementia. We also were searching for naturally occurring protective responses that we could target to help alleviate the disease’s symptoms,” said Dr. Daniel Geschwind, who holds the Gordon and Virginia MacDonald Distinguished chair in Human Genetics, and is a professor of neurology at the David Geffen School of medicine at UCLA and a professor of psychiatry at the Semel Institute for Neuroscience and Human Behavior at UCLA.

“We discovered that a drop in granulin sabotaged brain cells’ survival and boosted activity of WNT, a major signaling pathway,” Geschwind explained. “Within this pathway we identified a major increase in specific receptor that Wnt binds to on the cell surface.” These findings will be key for new treatments and better diagnosis.

Source: UCLA Health Sciences, ScienceDaily