Innovation Trends in AD Research –the shift to increasingly individualised trial design, therapeutics and care.

Alzheimer’s disease (AD) research is entering a new phase of innovation, highlighted by a number of advances presented at recent conferences such as CTAD 2024 in Madrid and AD/PD 2025 in Vienna. As an organisation deeply embedded in the AD scientific community, we are seeing first-hand the encouraging rapid progress towards more accessible, earlier, and personalized approaches in both therapy and biomarker development. Here are some of the trends we are most excited about:

Blood-based biomarkers (BBMs)

BBMs continue to be a key contributor to this shift. Both CTAD and AD/PD featured updates on the accuracy of BBMs for detecting amyloid and tau pathology, with growing momentum toward regulatory approval and integration into clinical trials. The US Food and Drug Administration (FDA) has recently cleared Fujirebio’s amyloid and tau ratio measure as the first BBM to aid AD diagnosis and several other BBMs hold the agency’s Breakthrough Device Designation, including Roche’s amyloid and tau panels.

At CTAD, Roche presented data from the largest global clinical trial of its kind, showing its amyloid plasma panel’s ability to rule out AD pathology. At the same conference, IXICO showcased results from the Global Alzheimer Platform’s (GAP) Bio-Hermes trial, demonstrating a two-stage screening model where indeterminate BBM results are followed up with PET scans. Beckman Coulter’s study presented at AD/PD quantified how often BBMs return indeterminate results, offering practical insights for implementing this approach.

As BBMs move toward broader use, organizations like the Global CEO Initiative are working on adoption guidelines. The WHO also published guidance to support global BBM development, highlighting their potential to improve diagnostic equity.

Vascular co-pathology

Towards a more holistic understanding of the disease, vascular co-pathology is gaining recognition as an important contributor to AD progression. CTAD featured several presentations showing that cerebrovascular pathology—such as white matter hyperintensities, microbleeds, and cerebral amyloid angiopathy—often coexists with amyloid and tau pathology and can drive cognitive decline independently.

At AD/PD, the MODEL-AD consortium presented preclinical findings that vascular dysfunction—such as blood-brain barrier breakdown and impaired cerebral perfusion—amplifies neurodegeneration and inflammation. Such insights are pushing the field toward an integrated disease model in which vascular and neurodegenerative processes interact across the disease continuum and supports improved trial design that integrate multi-biomarker stratification.

Accounting for vascular heterogeneity among participants can support more sensitive detection of treatment effects and biomarker signals. MRI-based imaging offers a potential solution: by quantifying vascular burden, it supports better participant stratification, adjustment for co-pathology, and enrichment of trial populations. As BBMs evolve, brain imaging remains key to linking molecular pathology with clinical expression.

Therapy development is expanding beyond amyloid.

Anti-tau therapies like UCB’s bepranemab and Eisai’s E2814 have shown early promise in slowing tau accumulation, with effects on fluid biomarkers and cognition. Biogen’s ASO BIIB080, administered intrathecally, has shown dose-dependent reductions in CSF tau and early clinical benefit. If validated in larger trials, it could offer a new treatment option, especially for early-stage patients.

Other novel strategies include Lexeo Therapeutics’ LX1001, a gene therapy targeting APOE expression, or Roche’s trontinemab, a modified gantenerumab combined with the Brainshuttle technology. Abbvie recently acquired rights to a similar platform, allowing drug molecules to cross the blood-brain barrier. Interest is also rising in repurposed metabolic drugs, such as GLP-1 receptor agonists (e.g., semaglutide), which may reduce neuroinflammation and support neuronal metabolism—mechanisms increasingly implicated in AD.

Anti-amyloid therapy is also becoming more practical and inclusive.

New efforts aim to reach broader populations, including those in preclinical stages, with the help of scalable BBM-based screening. Subcutaneous administration routes—for e.g. Eisai’s lecanemab—are being developed to reduce infusion burden and enable treatment in community settings.

Together, these advances in the understanding of disease processes, biomarker development, therapeutics, and trial design point to a future where AD trial design and care is increasingly individualized, shifting the focus from symptom management to early intervention and prevention.

 

---