A new class of compounds identified by researchers from the Weill Cornell Medical College, Brandeis University and Columbia University Medical Center could pave way to a new approach for treating Alzheimer's disease, the most common form of dementia that primarily affects the elderly and is characterized by impaired thinking, mood swings and memory loss.
In their study "Pharmacological chaperones stabilize retromer to limit APP processing," which was published in the journal Nature Chemical Biology on April 20, the researchers identified a "chaperone" compound R55, which they say, can significantly increase retromer levels.
The retromer protein complex steers away amyloid precursor protein (APP) from the region of the cell where it splits and create beta-amyloid, protein fragments that contribute to the development of Alzheimer's disease.
The researchers said that the compound's ability to increase retromer levels and reduce amyloid-beta in the neurons in the hippocampus, the part of the brain associated with learning and memory, could have implications in the treatment of neurodegenerative diseases such as Alzheimer's and Parkinson's.
"These findings show that pharmacological chaperones can enhance the function of a multiprotein complex and may have potential therapeutic implications for neurodegenerative diseases," the researchers wrote.
Study researcher Scott Small, director of the Alzheimer's Disease Research Center in the Taub Institute for Research on Alzheimer's Disease, said that treatment involving pharmacological chaperones could be safer and even more effective than conventional methods used for treating neurological diseases as they often target only single proteins.
"Our findings identify a novel class of pharmacologic agents that are designed to treat neurologic disease by targeting a defect in cell biology, rather than a defect in molecular biology," Small said.
Although it isn't yet clear what causes Alzheimer's, many believe that genetics has a crucial role in the development of the disease particularly in those who develop it early. Environmental and lifestyle factors are also considered to play a role in the development of the disease.
The Alzheimer's Association reports that about 5.2 million Americans suffer from Alzheimer's disease as of 2014. The number includes about 200,000 individuals below the age of 65. It also estimates that by 2050, the number of individuals who are at least 65-years old with Alzheimer's may increase from 5 million to up to 16 million unless medical breakthroughs that could prevent and stop the disease could be developed.