FDA rejects first-in-class osteoporosis drug
Nature Reviews Drug Discovery 16, 593 (2017).
doi:10.1038/nrd.2017.176
Author: Asher Mullard
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - September 1, 2017 Category: Drugs & Pharmacology Authors: Asher Mullard Tags: News and Analysis Source Type: research
Diabetes drug shows promise in Parkinson disease
Nature Reviews Drug Discovery 16, 593 (2017).
doi:10.1038/nrd.2017.175
Author: Asher Mullard
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - September 1, 2017 Category: Drugs & Pharmacology Authors: Asher Mullard Tags: News and Analysis Source Type: research
FDA approves first-in-class cancer metabolism drug
Nature Reviews Drug Discovery 16, 593 (2017).
doi:10.1038/nrd.2017.174
Author: Asher Mullard
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - September 1, 2017 Category: Drugs & Pharmacology Authors: Asher Mullard Tags: News and Analysis Source Type: research
FDA OKs first in vitro route to expanded approval
Nature Reviews Drug Discovery 16, 591 (2017).
doi:10.1038/nrd.2017.140
Author: Katie Kingwell
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. (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - September 1, 2017 Category: Drugs & Pharmacology Authors: Katie Kingwell Tags: News and Analysis Source Type: research
Synthetic lethality screens point the way to new cancer drug targets
Nature Reviews Drug Discovery 16, 589 (2017).
doi:10.1038/nrd.2017.165
Author: Asher Mullard
A resurgence in synthetic lethality screening, enabled by CRISPR gene editing, is unlocking targeted drugs for patients with cancerous loss-of-function mutations. (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - September 1, 2017 Category: Drugs & Pharmacology Authors: Asher Mullard Tags: News and Analysis Source Type: research
Pulling away from the pack in drug launches
Nature Reviews Drug Discovery 16, 749 (2017).
doi:10.1038/nrd.2017.122
Authors: Nicholas Donoghoe, Jon Duane, Jin Kim & George Xu
This analysis of US drug launches from the past decade indicates that commercial success has become more difficult to achieve, owing to factors such as increased competition, greater challenges in achieving therapeutic differentiation and payer pressures. (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 18, 2017 Category: Drugs & Pharmacology Authors: Nicholas Donoghoe Jon Duane Jin Kim George Xu Tags: News and Analysis Source Type: research
Inflammatory diseases: An IL-9 solution to inflammation resolution
Nature Reviews Drug Discovery 16, 600 (2017).
doi:10.1038/nrd.2017.164
Author: Megan Cully
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 18, 2017 Category: Drugs & Pharmacology Authors: Megan Cully Tags: Research Highlight Source Type: research
Cancer: Inhibiting ROCK in neuroblastoma
Nature Reviews Drug Discovery 16, 602 (2017).
doi:10.1038/nrd.2017.160
Author: Crunkhorn Sarah
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 11, 2017 Category: Drugs & Pharmacology Authors: CrunkhornSarah Tags: Research Highlight Source Type: research
Systems medicine: Understanding wellness and disease
Nature Reviews Drug Discovery 16, 602 (2017).
doi:10.1038/nrd.2017.159
Author: Crunkhorn Sarah
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 11, 2017 Category: Drugs & Pharmacology Authors: CrunkhornSarah Tags: Research Highlight Source Type: research
Immunotherapy: CAR T cells in glioblastoma
Nature Reviews Drug Discovery 16, 602 (2017).
doi:10.1038/nrd.2017.158
Author: Crunkhorn Sarah
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. (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 11, 2017 Category: Drugs & Pharmacology Authors: CrunkhornSarah Tags: Research Highlight Source Type: research
Anticancer agents: Fighting resistance
Nature Reviews Drug Discovery 16, 602 (2017).
doi:10.1038/nrd.2017.157
Author: Crunkhorn Sarah
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 11, 2017 Category: Drugs & Pharmacology Authors: CrunkhornSarah Tags: Research Highlight Source Type: research
Antibacterial agents: New routes to tuberculosis treatment
Nature Reviews Drug Discovery 16, 600 (2017).
doi:10.1038/nrd.2017.156
Author: Sarah Crunkhorn
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 11, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Cancer immunotherapy: Searching in the immune checkpoint black box
Nature Reviews Drug Discovery 16, 599 (2017).
doi:10.1038/nrd.2017.163
Author: M. Teresa Villanueva
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 11, 2017 Category: Drugs & Pharmacology Authors: M. Teresa Villanueva Tags: Research Highlight Source Type: research
Regulatory watch: Structural and procedural characteristics of international regulatory authorities
Nature Reviews Drug Discovery 16, 594 (2017).
doi:10.1038/nrd.2017.135
Authors: Anand B. Jain, Annette Mollet & Thomas D. Szucs
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 (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - August 11, 2017 Category: Drugs & Pharmacology Authors: Anand B. Jain Annette Mollet Thomas D. Szucs Tags: News and Analysis Source Type: research
Cancer: mRNA-encoded bispecific antibodies eliminate tumours
Nature Reviews Drug Discovery 16, 530 (2017).
doi:10.1038/nrd.2017.145
Author: Sarah Crunkhorn
The development of therapeutic bispecific T cell-engaging antibodies, which recruit cytotoxic T cells to tumour cells, has been hampered by manufacturing challenges as well as their short serum half-life. To circumvent these issues, Stadler et al. generated 1-methylpseudouridine-containing mRNAs encoding bispecific antibodies directed against (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - July 31, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
