Nature Reviews Drug Discovery

Bridging the translational innovation gap through good biomarker practice ()
Few biomarkers progress from discovery to become validated tools or diagnostics. To bridge this gap, three European biomedical research infrastructures — EATRIS-ERIC (focused on translational medicine), BBMRI-ERIC (focused on biobanking) and ELIXIR (focused on data sharing) — are paving the way to developing and sharing best practices for biomarker validation.
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Synthetic lethality screens point the way to new cancer drug targets ()
A resurgence in synthetic lethality screening, enabled by CRISPR gene editing, is unlocking targeted drugs for patients with cancerous loss-of-function mutations.
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FDA OKs first in vitro route to expanded approval ()
In vitro data can be used to accelerate the approval of drugs that target specific disease-causing mutations for additional subpopulations of patients with rare diseases such as cystic fibrosis.
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FDA approves first-in-class cancer metabolism drug ()
The FDA approved Agios' and Celgene's enasidenib for acute myeloid leukaemia (AML), validating metabolism-modulating drugs as a means of killing cancer cells.Enasidenib (formerly AG-221) is a first-in-class inhibitor of mutated isocitrate dehydrogenase 2 (IDH2). The IDH enzymes normally metabolize isocitrate into α-ketoglutarate. When
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Diabetes drug shows promise in Parkinson disease ()
There are no approved disease-modifying drugs for Parkinson disease, a neurodegenerative condition that affects 2–3% of people aged ≥65 years. Accumulating evidence suggests that GLP1 receptor agonists — incretin mimetics that researchers first developed for the treatment of diabetes — may have neuroprotective properties. A
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FDA rejects first-in-class osteoporosis drug ()
The FDA rejected Amgen's and UCB's romosozumab, an anti-sclerostin monoclonal antibody that the firms are developing for the treatment of osteoporosis. The rejection followed on the heels of a cardiovascular safety signal that the companies reported in May. Amgen and UCB are now working
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Regulatory watch: Structural and procedural characteristics of international regulatory authorities ()
Drug reviewers in regulatory authorities (health authorities) around the world have the responsibility of evaluating safety, effectiveness and quality control data for a new drug before it is approved for marketing. The regulatory requirements, structure and processes of various regulatory authorities are different, making it
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Deal watch: IL-2 focus switches to stimulating Tregs ()
Recombinant interleukin-2 (IL-2) has been used since the early 1990s for the treatment of renal cell carcinoma and melanoma, as high-dose IL-2 has immune-stimulating properties. A spate of deals in 2017 — including Eli Lilly's acquisition of co-development rights to Nektar's NKTR-358, the acquisition
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Nigel Blackburn ()
In 2007, the charity Cancer Research UK (CRUK) launched its Clinical Development Partnerships (CDP) initiative to run trials of deprioritized anticancer candidates that still offered glimmers of promise. 10 years on, these reprioritization efforts have yielded mixed results. Despite generating some promising clinical data, none of their biopharma partners has as yet fully reprioritized any candidates. There is still hope for these drugs, although likely with new sponsors. In the case of an Aurora kinase inhibitor that was initially discovered by GlaxoSmithKline (GSK), for example, Nemucore has since licensed CRUK's data and further development rights. Now, however, smaller biotech firms are increasingly turning to the CDP for help with clinical trials of lead drug candidates. Nigel Blackburn, CRUK's Director of drug development, discusses the CDP's changing strategy with Asher Mullard
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What is the right amount to spend on biopharma R&D? ()
This analysis investigates whether some companies may be negatively affecting their overall commercial success by aiming to maintain steady earnings per share growth and/or a target ratio of R&D spending to sales.
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Cancer immunotherapy: Searching in the immune checkpoint black box ()
Checkpoint blockade through inhibition of programmed cell death protein 1 (PD1), which enables tumours to evade the immune system, has radically changed the treatment and prognosis of many cancers, especially melanoma. However, a majority of patients do not respond to PD1 inhibition, and great effort
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Antibacterial agents: New routes to tuberculosis treatment ()
Treatment of drug-resistant strains of Mycobacterium tuberculosis is lengthy, involving the use of costly and less effective second-line drugs that exhibit substantial side effects. Two new papers now report the identification of novel agents that inhibit previously untargeted essential M. tuberculosis enzymes and
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Inflammatory diseases: An IL-9 solution to inflammation resolution ()
Drugs for inflammation-driven diseases, such as rheumatoid arthritis, often aim to reduce the severity of the inflammatory response. Rauber et al. suggest an alternative strategy: promoting the resolution of inflammation. They found that IL-9 is needed for regulatory T (Treg) cells, which
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Biotechnology: CRISPR–Cas becoming more human ()
In the past few years, the CRISPR–Cas technology has been developed to precisely edit genomic DNA; so far, these approaches have been used mainly in cultured cells or in animal embryo models, but only a few studies have reported their use in human embryos. Ma
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Anticancer agents: Fighting resistance ()
RAF–MEK–ERK pathway inhibitors are used to treat cancers exhibiting BRAFV600E mutations, but drug resistance commonly develops. Here, analysis of patient-derived xenograft (PDX) of BRAFV600E-mutant cancers and single-cell DNA sequencing showed that following drug treatment, parallel evolutionary tracts enable the selection and
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Immunotherapy: CAR T cells in glioblastoma ()
Chimeric antigen receptor-modified T (CAR T) cells have shown efficacy in leukaemia and lymphoma. Now, O'Rourke et al. report on the first clinical trial of CAR T cells targeted to the epidermal growth factor receptor variant III (EGFRvIII) in 10 patients with recurrent glioblastoma.
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Systems medicine: Understanding wellness and disease ()
Systems medicine aims to understand the basis of wellness and disease. Here, Price et al. report findings from the Pioneer 100 Wellness Project, a collection of data from 108 individuals over 9 months - including whole genome sequences, clinical tests, metabolomes, proteomes, microbiomes and
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Cancer: Inhibiting ROCK in neuroblastoma ()
Neuroblastoma is a childhood tumour originating from the neural crest, for which treatment options are limited. Dyberg et al. report a high frequency of mutations in genes associated with RHO–RAC signalling in human neuroblastoma samples. Increased expression of the downstream RHO-activating kinase ROCK2 was
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Cancer: Ironing it out ()
Cancer cells with mesenchymal-like properties are more likely to become resistant to a wide range of therapies. A new study suggests that this mesenchymal-like state of cancer cells is vulnerable to inhibition of the lipid peroxidase pathway, leading to an iron-dependent form of cell death,
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The antifungal pipeline: a reality check ()
Invasive fungal infections continue to appear in record numbers as the immunocompromised population of the world increases, owing partially to the increased number of individuals who are infected with HIV and partially to the successful treatment of serious underlying diseases. The effectiveness of current antifungal
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Remyelination therapies: a new direction and challenge in multiple sclerosis ()
Multiple sclerosis is characterized by inflammatory activity that results in destruction of the myelin sheaths that enwrap axons. The currently available medications for multiple sclerosis are predominantly immune-modulating and do not directly promote repair. White matter regeneration, or remyelination, is a new and exciting potential
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Therapeutic targeting of the angiopoietin–TIE pathway ()
The endothelial angiopoietin (ANG)–TIE growth factor receptor pathway regulates vascular permeability and pathological vascular remodelling during inflammation, tumour angiogenesis and metastasis. Drugs that target the ANG–TIE pathway are in clinical development for oncological and ophthalmological applications. The aim is to complement current vascular endothelial growth
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Opportunities for therapeutic antibodies directed at G-protein-coupled receptors ()
Nature Reviews Drug Discovery16 (201710.1038/nrd.2017.91)In the article, the clinical trial for the biparatopic CXCR2 nanobody was described as being for cancer instead of inflammation. This error has been corrected in the online version.
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