Genetic disorders: Repurposing metformin in FXS
Nature Reviews Drug Discovery 16, 456 (2017).
doi:10.1038/nrd.2017.119
Author: Sarah Crunkhorn
Fragile X syndrome (FXS) is caused by loss of the fragile X mental retardation 1 (FMR1) gene and is characterized by learning disabilities and cognitive impairment. Here, Gantois et al. have shown that injection of the type 2 diabetes therapeutic metformin for (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - June 16, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Cancer: Targeting MYC-driven translation
Nature Reviews Drug Discovery 16, 456 (2017).
doi:10.1038/nrd.2017.118
Author: Sarah Crunkhorn
Overactivity of the transcription factor MYC plays a central part in the progression of multiple myeloma through upregulation of ribosome synthesis and translation activity. Using a high-throughput screening approach, Manier et al. have identified a series of synthetic analogues of the rocaglate natural product (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - June 16, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Endocrinology: Reducing fat and building bone
Nature Reviews Drug Discovery 16, 454 (2017).
doi:10.1038/nrd.2017.121
Author: Sarah Crunkhorn
Menopause — characterized by an increase in circulating levels of follicle-stimulating hormone (FSH) and a decline in oestrogen due to ovarian failure — is associated with loss of bone mass and increased visceral adiposity. Although treatments for osteoporosis and obesity exist, their use may be (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - June 16, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Market watch: Upcoming market catalysts in Q3 2017
Nature Reviews Drug Discovery 16, 449 (2017).
doi:10.1038/nrd.2017.117
Author: Sonny Nghiem
Market catalysts in the third quarter of 2017 include top-line phase III trial results for several therapeutics: patisiran (developed by Alnylam Pharmaceuticals) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN); durvalumab (developed by AstraZeneca) for the first-line treatment of locally advanced or metastatic (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - June 16, 2017 Category: Drugs & Pharmacology Authors: Sonny Nghiem Tags: News and Analysis Source Type: research
Breaking barriers to novel analgesic drug development
Nature Reviews Drug Discovery 16, 545 (2017).
doi:10.1038/nrd.2017.87
Authors: Ajay S. Yekkirala, David P. Roberson, Bruce P. Bean & Clifford J. Woolf
Acute and chronic pain complaints, although common, are generally poorly served by existing therapies. This unmet clinical need reflects a failure to develop novel classes of analgesics with superior efficacy, diminished adverse effects and a lower abuse liability than those currently available. Reasons for this (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - June 9, 2017 Category: Drugs & Pharmacology Authors: Ajay S. Yekkirala David P. Roberson Bruce P. Bean Clifford J. Woolf Tags: Review Source Type: research
Cystic fibrosis: Thymosin α 1 rescues CFTR activity
Nature Reviews Drug Discovery 16, 386 (2017).
doi:10.1038/nrd.2017.99
Author: Sarah Crunkhorn
Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein and is characterized by chronic lung inflammation. Romani et al. show that the naturally occurring polypeptide thymosin α1 — clinically used as an immunotherapeutic agent — (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Ageing: FOXO4 inhibition eliminates senescent cells
Nature Reviews Drug Discovery 16, 386 (2017).
doi:10.1038/nrd.2017.98
Author: Sarah Crunkhorn
Senescent cells exhibit a pro-inflammatory phenotype, and are thought to accelerate ageing and the onset of age-related diseases. Baar et al. report a key role for forkhead box protein O4 (FOXO4) in maintaining senescent cell viability. In vitro, a FOXO4-derived peptide designed to (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Metabolic disease: PGC1 α inhibition ameliorates diabetes
Nature Reviews Drug Discovery 16, 386 (2017).
doi:10.1038/nrd.2017.97
Author: Sarah Crunkhorn
Peroxisome proliferator-activated receptor-γ co-activator 1α (PGC1α) plays a pivotal part in energy homeostasis and is involved in the regulation of gluconeogenesis. Using a cell-based high-throughput chemical screen, Sharabi et al. have identified a small molecule, SR-18292, that increases acetylation of PGC1α, which results in (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Infectious disease: Peroxin inhibitor treats Trypanosoma infection
Nature Reviews Drug Discovery 16, 386 (2017).
doi:10.1038/nrd.2017.93
Author: Sarah Crunkhorn
Insect-borne Trypanosoma spp. parasites transmitted predominantly by the tsetse fly infect humans as well as livestock, which causes devastating diseases, such as human African trypanosomiasis (HAT) and Chagas disease. Current treatments for trypanosomiases are limited, can cause serious side effects and require long treatment (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Inflammation: Oncostatin M blockade attenuates colitis
Nature Reviews Drug Discovery 16, 386 (2017).
doi:10.1038/nrd.2017.100
Author: Sarah Crunkhorn
Anti-tumour necrosis factor (TNF) antibodies represent established therapies for inflammatory bowel disease (IBD), but up to 40% of patients exhibit primary nonresponsiveness to anti-TNF agents, and resistance can develop. West et al. observe high levels of the cytokine oncostatin M (OSM) and its receptor (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Sarah Crunkhorn Tags: Research Highlight Source Type: research
Ageing: A youthful reminder
Nature Reviews Drug Discovery 16, 384 (2017).
doi:10.1038/nrd.2017.112
Author: Darran Yates
In rodents, the introduction of blood from young to old animals can reverse some of the negative effects that ageing exerts on CNS function, including detrimental changes in hippocampal function. However, it is not known which molecules found in young blood exert such effects or (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Darran Yates Tags: Research Highlight Source Type: research
Birgitte Volck
Nature Reviews Drug Discovery 16, 378 (2017).
doi:10.1038/nrd.2017.105
Small patient populations are big business. In 2000, worldwide sales of orphan drugs for rare diseases accounted for just 6% of global prescription drug sales. Last year they accounted for US$114 billion in revenue and nearly 16% of total prescription drug sales, and they are on track to account for 21% of sales by 2022. For Birgitte Volck, head of rare disease R&D at GlaxoSmithKline, the explosion of this space has tracked with the arrival of a new era in rare disease drug discovery and development. She told Asher Mullard about how new ...
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Tags: News and Analysis Source Type: research
Deal watch: Neurokinin 3 receptor antagonist revival heats up with Astellas acquisition
Nature Reviews Drug Discovery 16, 377 (2017).
doi:10.1038/nrd.2017.102
Author: Megan Cully
Japan's Astellas Pharma has announced that it will acquire the Belgian biotech Ogeda for up to [euro]800 million, including milestone payments (Fig. 1). Ogeda's sole clinical asset, fezolinetant, is a small-molecule inhibitor of the neurokinin 3 receptor (NK3R), for which the company reported (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Megan Cully Tags: News and Analysis Source Type: research
FDA approves first targeted drug for acute myelogenous leukaemia
Nature Reviews Drug Discovery 16, 375 (2017).
doi:10.1038/nrd.2017.110
Author: Asher Mullard
The FDA approved Novartis's FLT3 inhibitor midostaurin for acute myeloid leukaemia (AML). The multikinase inhibitor is the first targeted treatment for AML, and the first new FDA approval for AML in decades.The FLT3 kinase is mutated in around one-third of AML patients, and (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Asher Mullard Tags: News and Analysis Source Type: research
FDA approves first new ALS drug in over 20 years
Nature Reviews Drug Discovery 16, 375 (2017).
doi:10.1038/nrd.2017.109
Author: Asher Mullard
The FDA approved Mitsubishi Tanabe Pharma's edaravone for amyotrophic lateral sclerosis (ALS), the first US approval for the deadly neurodegenerative disease since riluzole in 1995.ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles, affecting the ability (Source: Nature Reviews Drug Discovery)
Source: Nature Reviews Drug Discovery - May 31, 2017 Category: Drugs & Pharmacology Authors: Asher Mullard Tags: News and Analysis Source Type: research
