Scientists at Washington University in St. Louis have engineered enzymes capable of dismantling the misfolded proteins tied to ALS and Parkinson's disease. The advance, led by associate professor Meredith Jackrel, focuses on a class known as disaggregases. These enzymes can be rapidly produced and screened using a newly developed method.
The work addresses a core driver of neurodegeneration: protein clumps that accumulate in neurons. Current treatments for ALS and Parkinson's only manage symptoms, without targeting the underlying molecular pathology. This approach could open a new therapeutic avenue.
The team's method accelerates the identification of effective disaggregase variants. Misfolded proteins are a hallmark of several neurological disorders, making these enzymes a promising tool. Details of the screening platform were not disclosed in the report.
Should the enzymes prove effective in animal models, human trials could follow. The research remains at an early stage, and challenges include delivery to the brain and long-term safety. Jackrel's team plans to test the enzymes in cellular and animal models.
"This is an important step," an expert unaffiliated with the study noted, "but many hurdles remain before clinical application." The approach highlights growing interest in protein repair as a strategy against neurodegeneration.