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Targeting Fibroblast Activation Protein for Precision Medicine.

The advancement of precision medicine depends critically on well-defined molecular targets. Pathologically activated fibroblasts drive disease progression in cancer, fibrosis and chronic inflammation through sustained inflammation, extracellular matrix remodeling and pathological microenvironment formation. Among available markers, fibroblast activation protein (FAP) stands out due to its selective overexpression in diseased tissues versus minimal expression in healthy organs. This unique profile makes FAP a promising target for molecular imaging and targeted therapy, enabling precision theran…

Precision medicine strategy in pancreatic ductal adenocarcinoma.

BACKGROUND: Pancreatic ductal adenocarcinoma (PDAC) is a highly lethal cancer with limited therapeutic options. Integration of molecular profiling may enable personalized treatment approaches. We evaluated the clinical utility of molecularly matched treatment (MMT) in a real-life cohort of PDAC patients. PATIENTS AND METHODS: A retrospective chart review of clinical/molecular data was carried out, including all PDAC patients with a contributive molecular profile. Survival outcomes were compared across three groups: patients with actionable molecular alterations (MAs) who received MMT (MA/MMT)

CD95/Fas stoichiometry in future precision medicine.

CD95, also known as Fas, belongs to the tumor necrosis factor (TNF) receptor superfamily. The main biological function of this receptor is to orchestrate and control the immune response since mutations in CD95 or deregulation of its downstream signaling pathways lead to auto-immunity and inflammation. Interestingly, more than twenty years ago, pioneer studies highlighted that like TNFR1, TRAILR1 or CD40, CD95 pre-associates at the plasma membrane in a ligand-independent fashion. This self-association occurs through a domain designated pre-ligand assembly domain or PLAD. Although the disruption

Patient-Derived Microphysiological Systems for Precision Medicine.

Patient-derived microphysiological systems (P-MPS) have emerged as powerful tools in precision medicine that provide valuable insight into individual patient characteristics. This review discusses the development of P-MPS as an integration of patient-derived samples, including patient-derived cells, organoids, and induced pluripotent stem cells, into well-defined MPSs. Emphasizing the necessity of P-MPS development, its significance as a nonclinical assessment approach that bridges the gap between traditional in vitro models and clinical outcomes is highlighted. Additionally, guidance is provi…

Defining neuroblastoma: From origin to precision medicine.

Neuroblastoma (NB), a heterogenous pediatric tumor of the sympathetic nervous system, is the most common and deadly extracranial solid malignancy diagnosed in infants. Numerous efforts have been invested in understanding its origin and in development of novel curative targeted therapies. Here, we summarize the recent advances in the identification of the cell of origin and the genetic alterations occurring during development that contribute to NB. We discuss current treatment regimens, present and future directions for the identification of novel therapeutic metabolic targets, differentiation

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… medicine, as it can affect treatment response. … Del Bufalo F, De Angelis B, Caruana I, et al. ; Precision Medicine Team–IRCCS Ospedale Pediatrico Bambino Gesù. GD2-CART01 for relapsed or refractory high-risk neuroblastoma. N Engl J Med. 2023;388(14):1284–1295. 154. Bosse KR, Raman P, Zhu Z, et al. …

Immunotherapy guided precision medicine in solid tumors.

Cancer is no longer recognized as a single disease but a collection of diseases each with its defining characteristics and behavior. Even wi … s its oncogenic transformation in a particular patient. This has ushered in the era of personalized precision medicine (PPM). Immunotherapy, on the other hand, involves leveraging the body's immune system to recognize and attack cancer cells and spare healthy cells from the damage induced by radiation and chemotherapy. Combining PPM and immunotherapy represents a pa …

Research Priorities for Precision Medicine in NAFLD.

NAFLD is a multisystem condition and the leading cause of chronic liver disease globally. There are no approved NAFLD-specific dugs. To advance in the prevention and treatment of NAFLD, there is a clear need to better understand the pathophysiology and genetic and environmental risk factors, identify subphenotypes, and develop personalized and precision medicine. In this review, we discuss the main NAFLD research priorities, with a particular focus on socioeconomic factors, interindividual variations, limitations of current NAFLD clinical trials, multidisciplinary models of care, and novel app…

Precision Medicine in Sepsis and Septic Shock.

Sepsis is defined as a potentially fatal organ dysfunction induced by a dysregulated host response to infection [...].

From cryptogenic to ESUS: Toward precision medicine?

Cryptogenic infarctions are infarctions without a defined cause, despite a complete work-up. They differ from infarctions of undetermined ca … d, ESUS-ATH with stenosis<50%, ESUS-AF (covert AF & atrial cardiomyopathy), ESUS-cancer and others. Precision medicine is the ability to make targeted healthcare decisions based on the specific risks of individual patients. One preliminary stage is therefore to identify homogeneous groups suitable in the future for new therapeutic trials and, at the end, for new spe …

Artificial intelligence in precision medicine in hepatology.

The advancement of investigation tools and electronic health records (EHR) enables a paradigm shift from guideline-specific therapy toward patient-specific precision medicine. The multiparametric and large detailed information necessitates novel analyses to explore the insight of diseases and to aid the diagnosis, monitoring, and outcome prediction. Artificial intelligence (AI), machine learning, and deep learning (DL) provide various models of supervised, or unsupervised algorithms, and sophisticated neural networks to generate predictive models more precisely than conventional ones. The data…

Lipid-lowering therapy: Guidelines to precision medicine.

Dyslipidemia is correlated with a series of health problems, such as obesity, insulin resistance, and the development of cardiovascular dise … t to treating patients with dyslipidemia according to LDL-C reduction as an individualized clinical precision medicine.

Endotype-driven precision medicine in chronic rhinosinusitis.

Introduction: Chronic rhinosinusitis (CRS) is a heterogeneous disease spectrum with variable phenotypes and endotypes that are not well characterized. Conventional treatment options are insufficient; recent research in precision medicine focuses on providing endotype-driven care based on underlying mechanisms.Areas covered: A comprehensive overview of CRS phenotypes and endotypes is provided. Biomarkers have been identified to predict prognosis and guide personalized pharmacotherapy, surgery, and innovative treatments. For CRS with nasal polyps (CRSwNP), type 2-targeting biologics, such as ant…

Cardiovascular Precision Medicine in the Genomics Era.

Precision medicine strives to delineate disease using multiple data sources-from genomics to digital health metrics-in order to be more precise and accurate in our diagnoses, definitions, and treatments of disease subtypes. By defining disease at a deeper level, we can treat patients based on an understanding of the molecular underpinnings of their presentations, rather than grouping patients into broad categories with one-size-fits-all treatments. In this review, the authors examine how precision medicine, specifically that surrounding genetic testing and genetic therapeutics, has begun to ma…

Precision medicine in head and neck cancer.

Head and Neck cancer is among the most common cancers worldwide, with a high prevalence in south East Asia, Brazil and central Europe. Head … diotherapy or surgery) and improved survival. In future years we expect to see the establishment of precision medicine modalities in an attempt to extend survival and improve quality of life of advanced stage HNSCC patients. Several phase III clinical trials are in progress to evaluate the utility of checkpoint inhibitors at different treatment settings, including c …

A Precision Medicine Approach to Oxytocin Trials.

In this chapter, we introduce a new area of social pharmacology that encompasses the study of the role of neuromodulators in modulating a wi … ies for social dysfunctions and anxiety disorders. We will highlight the significance of adopting a precision medicine approach to optimize targeted treatments with oxytocin in neuropsychiatry. Oxytocin effects on social behavior and brain function can vary from one individual to another based on external factors, such as heterogeneity in autism phenotype, childhood …

Prospects for Precision Medicine in Glomerulonephritis Treatment.

BACKGROUND: Glomerulonephritis (GN) consists of a group of kidney diseases that are categorized based on shared histopathological features. … ngs in podocyte biology and advances in experimental approaches that have opened the possibility of precision medicine in GN treatment. We provide an integrative basic science and clinical overview of new developments in GN research and the discovery of potential candidates for targeted therapies in GN. FINDINGS: Advances in podocyte biology have identified many ca …

Biomarkers and Precision Medicine in IgA Nephropathy.

The field of biomarker research in IgA nephropathy has experienced a major boost in recent years with the publication of a large number of s … imize the specific value of each biomarker in a multimarker panel. This is a promising approach for precision medicine and personalized therapy in IgA nephropathy.

Precision Transplant Medicine: Biomarkers to the Rescue.

The concept that individuals with the same disease and a similar clinical presentation may have very different outcomes and need very different therapies is not novel. With the development of many innovative tools derived from the omics technologies, transplant medicine is slowly entering the era of precision medicine. Biomarkers are the cornerstone of precision medicine, which aims to integrate biomarkers with traditional clinical information and tailor medical care to achieve the best outcome for an individual patient. Here, we discuss the basic concepts of precision medicine and biomarkers,…

Precision medicine in myeloma demands precision imaging: is whole body MRI the solution?

Outcomes for patients with multiple myeloma have improved substantially over the past two decades, with median overall survival more than doubling in many cohorts. Management of smouldering myeloma has also shifted, with randomised trials demonstrating that early intervention in high-risk disease delays progression and improves survival compared with observation alone. Imaging diagnostics must therefore evolve in parallel to provide minimally invasive, sensitive and quantitative insights into disease for patients who will live with myeloma for many years. Here we review the latest evidence for