Promising Update on Efficacy and Safety of “Molecular Tweezers”

Posted: May 7, 2014

Five new publications by Gal Bitan, Ph.D., and colleagues of the David Geffen School of Medicine at UCLA have been released on developing the "molecular tweezer"* CLR01 as a therapeutic drug for Alzheimer's disease and other amyloidoses (conditions involving the build-up of insoluble amyloid proteins).

For the past several years, Dr. Bitan has been studying "molecular tweezers". Initial studies show that administration of low doses of CLR01 leads to a significant reduction of Abeta and Tau in transgenic mice (mice which have been engineered to show human-like Alzheimer's traits). Bitan has also conducted trials on its safety and pharmacokinetics - the study of the effects of the drug on the body. The results were promising, showing no signs of toxicity in mice when CLR01 was administered daily, even at doses higher than those needed for efficacy of the drug.

The next step of this study will be to optimize the administered dose of the drug and examine its effect on prevention of appearance of brain pathology in young mice, compared to treating already-existing pathology in older transgenic mice.

*A molecular tweezer is an open molecule shaped like the letter "C", capable of binding to proteins and modulating their aberrant misfolding, thereby preventing the formation of toxic aggregates of several disease-related proteins, including Abeta and tau which are implicated in Alzheimer's disease.


For more on these studies, follow the links below:

Modulators of amyloid protein aggregation and toxicity: EGCG and CLR01

Bio-nanoimaging: Protein misfolding and aggregation

A Shortened Barnes Maze Protocol Reveals Memory Deficits at 4-Months of Age in the Triple-Transgenic Mouse Model of Alzheimer's Disease

Molecular Tweezers Targeting Transthyretin Amyloidosis

Safety and pharmacological characterization of the molecular tweezer CLR01 – a broad-spectrum inhibitor of amyloid proteins’ toxicity