Protein Replacement Therapy May Be Useful in Treating FTD-GRN, Recent Study Finds

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Preclinical studies analyzing GRN mutations in FTD have uncovered a new approach to enhancing the brain’s intake of progranulin, a commonly mutated protein linked to genetic forms of FTD.

As highlighted in a recent study published in Cell, researchers from Denali Therapeutics have developed the Protein Transport Vehicle (PTV) to increase progranulin levels in persons with FTD. The study provided new insight into the molecular and cellular mechanism that may contribute to FTD. Additionally, the research provided a greater understanding of how lysosomes — the “digestive system” of cells — are impacted by progranulin deficiencies and how the process of peripherally administered progranulin (PTV:PGRN) could be useful in treating certain types of FTD. The PTV:PGRN approach could be most beneficial in future treatment of FTD-GRN caused by progranulin deficiency, the study suggests.

Dr. Joseph Lewcock, chief scientific officer at Denali, said in a press release that the therapeutic strategy with PTV:PGRN may be “the most direct and effective way to increase progranulin levels in lysosomes for the potential treatment of people with FTD-GRN.”

He continued: “These new insights from our preclinical research suggest that FTD-GRN may be an atypical lysosomal storage disorder, and that lysosomal function can be restored by PTV:PGRN. Our work also identified candidate clinical biomarkers indicative of lysosomal dysfunction, such as BMP and GlcSph, which may help to evaluate the future therapeutic efficacy of PTV:PGRN and other therapeutics in people with FTD-GRN.”

Read the full Denali Therapeutics press release here.

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