In an encouraging study published in early March, researchers reported on a new drug that may prove effective in preventing and treating Alzheimer’s disease. In preclinical animal studies, this pharmacological breakthrough, known as Compound C, was shown to safely and effectively reduce the formation of amyloid plaques in the brains of mice, rats, and monkeys. These plaques, which are mangled proteins that accumulate in the brain and cause mild to severe cognitive impairment, are formed by the action of several enzymes, one of which is γ-secretase. Previously trialed drugs have attempted to prevent their formation by inhibiting this enzyme, but such agents caused unacceptably severe toxic effects. In contrast, Compound C is designed to modulate, rather than inhibit, this enzyme, and–importantly–it had no toxicity in the animals that received the drug, even as it demonstrably reduced plaque formation in their brains. The researchers are cautiously optimistic, as we are at MCI911, that human trials of Compound C will be able to begin in the not-too-distant future.1
Rynearson KD, et al. Preclinical validation of a potent γ-secretase modulator for Alzheimer’s disease prevention. J Exp Med. 2021;218(4):e20202560.
- LaFee S. Novel drug prevents amyloid plaques, a hallmark of Alzheimer’s disease. UC San Diego News Center. March 2, 2021. https://ucsdnews.ucsd.edu/pressrelease/novel-drug-prevents-amyloid-plaques-a-hallmark-of-alzheimers-disease. Accessed March 9, 2021.