UCLA researchers in March 2012 reported the development of a molecular compound called CLR01 that prevents toxic proteins associated with Parkinson's disease from binding together and killing the brain's neurons. Building on those findings, they have now turned their attention to Alzheimer's disease, which is thought to be caused by a similar toxic aggregation or clumping, but with different proteins, especially amyloid-beta and tau.
What they've found is encouraging. Using the same compound, which they've dubbed a "molecular tweezer," in a living mouse model of Alzheimer's, the researchers demonstrated for the first time that the compound safely crossed the blood-brain barrier, cleared the existing amyloid-beta and tau aggregates, and also proved to be protective to the neurons' synapses - another target of the disease - that allow cells to communicate with one another.
The report was published in the journal Brain.
"This is the first demonstration that molecular tweezers work in a mammalian animal model," says Gal Bitan, PhD, associate prof-essor of neurology. "Most important, no signs of toxicity were observed in the treated mice."
Molecular tweezers are complex molecular compounds capable of binding to other proteins. Dr. Bitan and his colleagues have been working with a particular molecular tweezer called CLR01.
The situation for humans is more proble-matic than for mice because the neurons gradually die in Alzheimer's disease, Dr. Bitan said. "That's why we must start treating as early as possible. The good news is that the molecular tweezers appear to have a high safety margin, so they may be suitable for prophylactic treatment starting long before the onset of the disease."
"Protection of Primary Neurons and Mouse Brain from Alzheimer's Pathology by Molecular Tweezers," Brain, November 26, 2012