Researchers have reportedly spotlighted new strategies targeting the prevention and treatment of diseases facilitated by protein mis-folding, including amyotrophic lateral sclerosis (ALS), Huntington’s disease, Alzheimer’s, cystic fibrosis, and cancer. The two studies; the first genetic and the second a nature chemical biology study, were conducted at Northwestern University, headquartered in Chicago, Ill.

The research team indicates that the studies’ data pinpoints new genes and pathways that may prevent protein mis-folding and toxic aggregation, keeping cells healthy. Researchers add that the studies also highlight small molecules with potential therapeutic implications for damaged cells. Richard I. Morimoto, PhD, led the study. “These discoveries are exciting because we have identified genes that keep us healthy and small molecules that keep us healthy,” Morimoto explains.

According to the research team, the first genetic study was conducted in transparent roundworm C. elegans. The study reports that the expression of each gene was reduced one at a time and observed to note any suppressed protein aggregation in the cells. Nine genes were identified that researchers say made all proteins in the cell healthier. The study suggests that these nine define a core homeostasis network that protects the animal’s proteome from protein damage.

During the second, nature chemical biology study, small molecules in human tissue culture cells were screened. Morimoto reports that seven classes of compounds were identified that expand the cell’s ability to make more protective molecular chaperones, which in turn restores proper protein folding. According to researchers, upon treatment with these small molecules, diseased animals were restored to health. 

“We don’t yet know the detailed mechanisms of these small molecules, but we have identified some good drug targets for further development,” Morimoto adds.

Source: Northwestern University