Discovery of Tiny Cell ‘Tunnels'

FAU scientists find brain cells use nanotubes to spread toxic huntingtin protein to neighbors.

Huntington’s disease is a devastating inherited brain disorder caused by a toxic protein that gradually spreads from neuron to neuron, leading to progressive movement, cognitive and psychiatric symptoms. There is currently no cure, and existing treatments only manage symptoms without slowing the underlying disease, which typically unfolds over 10 to 20 years. Understanding how this harmful protein spreads through the brain is critical to developing therapies that can stop the disease before widespread damage occurs.

In a new study published in Science Advances, FAU scientists and collaborators have discovered that brain cells use tiny structures called tunneling nanotubes to pass the toxic huntingtin protein to neighboring cells. The researchers identified a previously unknown molecular partnership between two proteins, Rhes and SLC4A7, that drives the formation of these cellular tunnels. By disrupting this pathway in both cells and a mouse model of Huntington’s disease, they significantly reduced the spread of the toxic protein in the brain, revealing a promising new target for therapies aimed at slowing or halting disease progression.

“This research shines a spotlight on an entirely new way cells communicate in health and disease,” said Randy Blakely, Ph.D., executive director of the FAU Stiles-Nicholson Brain Institute, the David J.S. Nicholson Distinguished Professor in Neuroscience, and a professor of biomedical science in the FAU Charles E. Schmidt College of Medicine. “By learning how harmful proteins physically move from cell to cell, we gain powerful new leverage points for therapy. The idea that we could slow or even halt disease progression by blocking these microscopic tunnels opens an exciting frontier for treating not only Huntington’s disease, but a wide range of neurological disorders and cancers in the future.”

Read the press release.