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Background: Convergent evidence indicates that deficits in the endosomal recycling pathway underlies pathogenesis of Alzheimer's disease (AD). SORL1 encodes the retromer-associated receptor SORLA that plays an essential role in recycling of AD-associated cargos such as the amyloid precursor protein and the glutamatergic AMPA receptor. Importantly, loss of function pathogenic SORL1 variants are associated with AD.
View Article and Find Full Text PDFBackground: Lomecel-B is a novel cell-based therapy with potential to demonstrate clinical benefit on Alzheimer's disease (AD) and its progression. Here we present the results of a phase 2a proof-of-concept trial (n = 49) to further define the potential of Lomecel-B in patients with mild AD dementia.
Methods: This double-blind, randomized, placebo-controlled 45-week trial (ClinicalTrials.
Drug Development.
Alzheimers Dement
December 2024
Washington University in St. Louis, School of Medicine, St. Louis, MO, USA; Washington University School of Medicine, St. Louis, MO, USA; Knight Alzheimer Disease Research Center, St. Louis, MO, USA.
Although amyloid b immunotherapies offer great potential for prevention or delay of symptoms in dominantly inherited AD (DIAD), the mechanism of action of this class of medications does not address the underlying mechanism of most DIAD mutations. Moreover, the need for repeated IV infusions or sub-cutaneous injections with Ab immunotherapies may prove challenging for long-term prevention approaches. The majority of DIAD mutations appear to affect the interaction of the gamma-secretase enzyme with the Amyloid Precursor Protein (APP) making this enzyme an attractive target for disease modification.
View Article and Find Full Text PDFDementia Care Research and Psychosocial Factors.
Alzheimers Dement
December 2024
Soleo Health, Frisco, TX, USA.
Background: The approval of anti-amyloid monoclonal antibodies (mAbs) provides a novel approach to the treatment of Alzheimer's disease. Infusions in alternative sites of care can benefit the patient financially and logistically, but coverage is largely payor dependent. The purpose of this study is to describe observations from this national complex specialty pharmacy around the safety of anti-amyloid mAb infusions in alternative sites of care, including the home.
View Article and Find Full Text PDFDementia Care Practice.
Alzheimers Dement
December 2024
Washington University School of Medicine, Saint Louis, MO, USA.
Implementation of amyloid-lowering treatments in clinical care for early symptomatic Alzheimer disease (AD) raises many challenges. The Memory Diagnostic Center (MDC), the dementia specialty practice associated with Barnes-Jewish Hospital/Washington University School of Medicine (BJH/WUSM), has 16 clinicians (12 physicians and 4 advanced practice providers) who see over 2,000 patients with memory disorders per year. BJH is the academic flagship of BJC HealthCare (BJC), an integrated health system in St.
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