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From the cancellation of a massive alcohol study to the dissolution of Theranos, 2018 was a year fraught with shutdowns. But amid all the shake-ups, the year also saw a few firsts, including approval of an artificial intelligence–based medical platform that helps clinicians diagnose strokes.
Metformin functions through a gut microbiome–bile acid–farnesoid X receptor axis to lower glucose in type 2 diabetes, revealing a new therapeutic target in this disease.
In 2018, drugs for rare conditions such as beta-thalassemia and forms of amyloidosis made it onto the approval mainstage in addition to drugs for more common diseases such as cancer. The gene-editing technology CRISPR slowly made strides into the therapeutic realm, while drugs for Alzheimer’s disease continued to falter, leaving a wide gap that is still in need of filling.
Disruption of the circadian clock has been linked to cancer and alterations in cancer metabolism and this has implications for therapeutic development.
Single cell dissection of plasma cell heterogeneity in myeloma patients reveals new insights into disease that may inform early diagnosis and clinical management.
Analysis of the UK Biobank genetic and phenotypic data demonstrate the power of including a large population and detailed phenotyping in a prospective study to identify genetic and lifestyle factors related to health and disease.
Protein-specific and shared genetic pathways influence the spread of amyloid-β and tau pathology. Hence, distinct gene expression profiles may induce regional vulnerability of the human cortex to the specific proteinopathies of Alzheimer’s disease.
Mutational signatures in melanoma are associated with prognostic features in patients and suggest distinct disease etiologies associated with the influence of different wavelengths of ultraviolet radiation.
WASP is a novel tumor-suppressor gene in ALK-driven anaplastic large cell lymphoma through modulation of CDC42 and MAPK signaling and provides a rationale for combination therapy.
Nongenetic activation of Aurora kinase A in the majority of patients with non-small-cell lung cancer mediates adaptive resistance to EGFR inhibition and offers an opportunity for combination treatment.
Initial results from a first-in-human study show that PET imaging with PD-L1 antibodies outperforms immunohistochemistry- or RNA-sequencing-based biomarkers for prediction of clinical response to immunotherapy.
ONECUT2 is a targetable transcription factor that antagonizes androgen receptor signaling and drives androgen independence and neuroendocrine differentiation in castration-resistant prostate cancer.