Researchers have made significant strides in understanding frontotemporal dementia (FTD), a disease that can manifest during middle age, through a recent study published in Nature Aging. The study, led by Rowan Saloner, PhD, an Assistant Professor at the UCSF Memory and Aging Center, analyzed over 4,000 proteins in cerebrospinal fluid samples from 116 individuals with inherited FTD. The findings are expected to identify these first specific biomarkers for the disease, which could revolutionize approaches to early detection and treatment.
The study was primarily aimed at contrasting these samples with those from 39 healthy controls matched to the probands with FTD. These biological changes were found to be associated with RNA processing, synaptic health, and immune responses. These alterations are directly associated with the disease severity. This month’s landmark news has renewed hope in the battle against frontotemporal dementia. As Saloner noted, just as Alzheimer’s disease now has accepted biomarkers and emerging disease-modifying treatments, frontotemporal dementia is still without any approved therapies.
James Giordano, PhD, MPhil, a professor emeritus at Georgetown University Medical Center and frequent TMC collaborator, communicated the vital nature of this study. He noted that because it used advanced proteomic analytic techniques, it was able to quickly and thoroughly identify potential biomarkers for FTD. This study uncovered important protein alterations that may cause FTD during middle age. Taken together, these findings lay the groundwork for future personalized treatments that directly address the disease’s underlying biology.
Saloner explained that studying inherited forms of FTD allows researchers to understand the underlying brain pathology in living patients confidently. He said their approach involved measuring concentrations of tens of thousands of proteins in cerebrospinal fluid. This enabled them to map biological changes underlying RNA processing, synaptic health, and immune response that were all connected to increasing disease severity. This combined approach offers a unique and potent model for identifying physiological changes before they become behavioral symptoms.
The study’s results might lay the foundation for creating molecular tests to diagnose FTD more quickly and track its progression. Saloner said their study was especially important. It gets us one step closer to creating molecular tests that help detect diseases sooner, track their evolution, and direct personalized treatments customized to a patient’s biology. He added it will be very important to replicate many of the findings in patients with the sporadic forms of FTD. This means that the biological changes found may be important to a high percentage of patients.
Beyond policy implications, Giordano noted that the study has a major limitation. The application of a very specialized proteomic scanning tool likely limited their results. Despite this constraint, he emphasized that early identification of proteomic biomarkers could lead to new drug development trajectories aimed at preventing or mitigating the progression of FTD and other neurodegenerative conditions.
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