A very delicate balance Could blocking lysosomal gatekeeper PIKfyve slow neurodegeneration?
Earlier this year, the U.S. Food and Drug Administration approved the drug Aduhelm for Alzheimer’s disease. Its manufacturers say Aduhelm blocks aggregation of the protein amyloid beta in the brain. But evidence that the drug can help patients requires a very hopeful eye; clinical studies were so inconclusive that the agency’s advisory panel recommended against clearing the drug for marketing to patients. FDA leaders’ willingness to override that recommendation speaks to patients’ unmet need.