Collection 01 February 2017

Key Advances in Medicine 2017

In 2016, numerous discoveries set the world of clinical and biomedical research abuzz with possibilities for the future. The Key Advances in Medicine eBook provides a hive of information for students, physicians and clinical researchers wanting to keep up to date with these clinical and biomedical advances, as well as developments to look out for in 2017.

This eBook is free to access and download for registered users, and contains 45 articles written by international experts who have identified the top groundbreaking papers published within their specialty. Altogether, the authors provide summaries and critiques of >200 key papers, published in journals such as The New England Journal of Medicine and The Lancet.

Key Advances in Medicine, a product from the clinical Nature Reviews journals, is a vital resource for time-poor students, physicians and clinical researchers who want expert opinion on the latest groundbreaking developments in 2016.

Download/View the Key Advances in Medicine 2017 PDF (10 MB).

Content

Advances in new therapeutic targets for atherosclerosis

In 2016, advances in atherosclerosis research were focused on the discovery and validation of new targets with genetic and mechanistic links to atherothrombotic heart disease. Novel targets include proteins involved in glycoprotein recognition and clearance, regulators of triglyceride-rich particle metabolism, inflammatory pathways that impair efferocytosis, and the gut microbiome.
- W. H. Wilson Tang
- Stanley L. Hazen
Year in Review17 Jan 2017 Nature Reviews Cardiology
Common and rare genetic variants and risk of CHD

Much of the progress in cardiovascular genetics in 2016 has been driven by next-generation sequencing studies, and the clinical utility of knowing an individual's genotype for predicting their risk of cardiovascular disease is gaining credibility, both for monogenic and polygenic disorders. Additionally, phenotype data are increasingly abundant, although databases linking genotype with clinically relevant phenotypes require optimization.
- Daniel I. Swerdlow
- Steve E. Humphries
Year in Review5 Jan 2017 Nature Reviews Cardiology
Arrhythmia treatment — evidence catching up with technology

Clinical cardiac electrophysiology has evolved rapidly over the past 2 decades. Although the fast pace of technical and therapeutic advances has occasionally outpaced evidence supporting widespread effectiveness, the highlights of electrophysiology research in 2016 illustrate the emergence of robust evidence for implementation of several important therapies.
- Benjamin A. Steinberg
- Jonathan P. Piccini
Year in Review17 Jan 2017 Nature Reviews Cardiology
Assessing strategies to improve patient management

The leading studies in 2016 on acute coronary syndromes focused on strategies of acute coronary management, from the influence of revascularization timing on outcomes, to selection of second-generation antiplatelet therapy and the utility of monitoring platelet function in patients at high risk of coronary artery disease.
- Ron Waksman
Year in Review5 Jan 2017 Nature Reviews Cardiology
Fresh perspectives on treatment and moments of clarity

Data obtained in the past year underscored the benefit of a triplet regimen comprising bortezomib, lenalidomide, and dexamethasone for patients with newly-diagnosed multiple myeloma, and have provided high-level evidence supporting the safety of adding daratumumab to standard-of-care doublets for those with relapsed and/or refractory disease. As a result, achieving minimal residual disease-negativity at any stage of myeloma is now a realistic possibility.
- Prashant Kapoor
- S. Vincent Rajkumar
Year in Review10 Jan 2017 Nature Reviews Clinical Oncology
Shining light on novel targets and therapies

In 2016, the pace of biological insights into small-cell lung cancer (SCLC) was reflected in new treatment approaches that have suggested meaningful clinical benefit to patients. We focus on three highlights of 2016: preclinical studies defining NFIB as a putative driver of metastasis, and two clinical studies; one that assessed the efficacy of an agent targeting the Notch ligand DLL3, and the other that explored T-cell checkpoint-blockade therapies targeting PD-1 and CTLA-4.
- Charles M. Rudin
- John T. Poirier
Year in Review13 Dec 2016 Nature Reviews Clinical Oncology
A watershed year for improvements in treatment?

In the past year, clinical trials have provided important information on strategies to decrease treatment-associated toxicities in patients with head and neck cancer. In addition, the FDA approved the first immunotherapeutic agents for patients with recurrent and/or metastatic disease, based on the observation of durable responses to pembrolizumab in a phase Ib trial, and demonstration of improved survival and quality of life with the use of nivolumab versus chemotherapy in a phase III trial.
- Alain P. Algazi
- Jennifer R. Grandis
Year in Review6 Dec 2016 Nature Reviews Clinical Oncology
Evolving biological understanding and treatment of sarcomas

In 2016, novel findings on the role of predisposing gene variants in sarcoma oncogenesis were published, as well as studies addressing novel molecular classifications and results from randomized controlled trials highlighting successful new treatments. Herein, we discuss these meaningful advances.
- Jean-Yves Blay
- Isabelle Ray-Coquard
Year in Review13 Dec 2016 Nature Reviews Clinical Oncology
Defining rules for increasingly personalized treatments

In 2016, results of an extensive trial broadened the range of malignancies that can be treated with everolimus to include neuroendocrine tumours (NETs) of the lung and gastrointestinal tract. Furthermore, studies aimed at identifying biomarkers with increased specificity, and at better defining high-grade NETs have enabled substantial progress towards delivering effective targeted treatments to patients with NETs.
- Massimo Falconi
- Stefano Partelli
Year in Review6 Dec 2016 Nature Reviews Clinical Oncology
Advances in treatment — jostling for pole position

In 2016, two major trials provided conflicting evidence regarding the role of 1 year of adjuvant therapy with sunitinib for patients with high-risk renal-cell carcinoma. In the second-line metastatic setting, updated data from key trials showed that cabozantinib improved overall survival over everolimus, and nivolumab was associated with a better quality of life compared with everolimus. Finally, a phase II study in previously untreated patients showed cabozantinib to be superior to sunitinib.
- Laurence Albiges
- Toni K. Choueiri
Year in Review10 Jan 2017 Nature Reviews Clinical Oncology
Neuroendocrine control of metabolism and reproduction

Neuroendocrine networks were previously perceived mainly as transcriptionally controlled, neural regulatory pathways that are centred at the hypothalamus. However, multisystemic circuits encompassing the brain and peripheral tissues have now been uncovered that involve nonneuronal cells and nontranscriptional regulatory mechanisms, with previously unidentified functions, such as reward and behaviour. Several developments in 2016 have helped to consolidate these new advances.
- Manuel Tena-Sempere
Year in Review6 Jan 2017 Nature Reviews Endocrinology
Advances in the understanding of adipose tissue biology

Adipose tissues have a central role in energy homeostasis, as they secrete adipokines and regulate energy storage and dissipation. Novel adipokines from white, brown and beige adipocytes have been identified in 2016. Identifying the specific receptors for each adipokine is pivotal for developing greater insights into the fat-derived signalling pathways that regulate energy homeostasis.
- Shingo Kajimura
Year in Review4 Jan 2017 Nature Reviews Endocrinology
Biological and translational insights from T2DM genetics

Type 2 diabetes mellitus (T2DM) is a major global health challenge. Development of more effective strategies for prevention and therapy depends on an improved understanding of its pathogenetic mechanisms. 2016 ends a period during which large-scale discovery of risk alleles for T2DM became routine and heralds a shift in research focus towards their exploitation to fuel mechanistic insights.
- Mark I. McCarthy
Year in Review4 Jan 2017 Nature Reviews Endocrinology
Health benefits of exercise — more than meets the eye!

Although regular physical activity can prevent or reduce the risk of many age-related diseases, the molecular mechanisms underpinning the protective effects of exercise are largely unknown. In 2016, a series of studies demonstrated that crosstalk between tissues during exercise can protect against metabolic disease, cancer, retinal degeneration and memory loss. These studies provide a molecular basis for the concept of 'exercise as medicine'.
- Mark A. Febbraio
Year in Review4 Jan 2017 Nature Reviews Endocrinology
A new frontier in FGF21 biology

In 2016, four studies were published that provided crucial new information on the endocrine actions of the hormone fibroblast growth factor 21 (FGF21). These studies provide a framework for the nutritional stimuli that regulate FGF21 expression and demonstrate a major role for FGF21 in primates and humans in regulating food intake, macronutrient preference and central reward pathways.
- Matthew J. Potthoff
Year in Review16 Dec 2016 Nature Reviews Endocrinology
Obesity in geroscience — is cellular senescence the culprit?

Obesity and ageing are major worldwide health challenges associated with lifestyle changes and an increase in age-related diseases, characterized by chronic inflammation dubbed metaflammation and inflammaging. However, the mechanistic link between these inflammatory processes is still unknown. New findings in 2016 shed light on these issues and indicate common targets for intervention.
- Claudio Franceschi
Year in Review6 Jan 2017 Nature Reviews Endocrinology
Brain–gut–microbiota axis — mood, metabolism and behaviour

In 2016, key studies have increased our understanding of the part played by the brain–gut–microbiota axis in disorders as diverse as depression, obesity and autism spectrum disorder. The data indicate that alterations in gut-microbial composition can substantially affect central physiology, and that transplantation of the gut microbiota can transfer a behavioural or physiological phenotype.
- Timothy G. Dinan
- John F. Cryan
Year in Review5 Jan 2017 Nature Reviews Gastroenterology & Hepatology
Global and immunotherapeutic insights into hepatitis B

The burden of HBV infection remains high and new strategies to improve HBV vaccination and therapy are needed. Key research in 2016 highlights the efficacy of current approaches and proposes new concepts for some of the immunological defects that need to be overcome for HBV functional cure.
- Mala K. Maini
- Antonio Bertoletti
Year in Review5 Jan 2017 Nature Reviews Gastroenterology & Hepatology
Circulating tumour cells and cell-free DNA in gastrointestinal cancer

The challenge to obtain needle biopsy samples from patients with cancer has steered the development of new blood-based diagnostics called 'liquid biopsy'. In 2016, major advances have been made in the use of circulating tumour cells and cell-free DNA for monitoring tumour evolution in patients with cancer of the gastrointestinal tract, with a focus on colorectal cancer.
- Klaus Pantel
- Catherine Alix-Panabières
Year in Review18 Jan 2017 Nature Reviews Gastroenterology & Hepatology
Biologicals and biosimilars in IBD — the road to personalized treatment

In 2016, personalized medicine for IBD has been evolving. Increasing comfort with biosimilar infliximab was achieved with 'real-life' data. Drugs with alternative modes of action confirmed substantial benefit, even in patients failing anti-TNF agents. Adipose-derived mesenchymal stem cells yielded a new treatment option for perianal fistulas.
- Krisztina B. Gecse
- Péter L. Lakatos
Year in Review11 Jan 2017 Nature Reviews Gastroenterology & Hepatology
High-definition PBC: biology, models and therapeutic advances

In 2016, obeticholic acid became the first new licensed therapy for primary biliary cholangitis in >20 years. This therapeutic came at a time of improved disease understanding from biliary and immunological mechanistic insights.
- Gwilym J. Webb
- Gideon M. Hirschfield
Year in Review11 Jan 2017 Nature Reviews Gastroenterology & Hepatology
A banner year for gut microbiota research

Fascination about the gut microbiota shows no signs of slowing down. The launch of the US National Microbiome Initiative in 2016, and similar efforts across the globe, underscore the continued enthusiasm for microbiome studies in the USA and beyond. Indeed, 2016 has been yet another notable year for gut microbiota research.
- Wendy S. Garrett
Year in Review11 Jan 2017 Nature Reviews Gastroenterology & Hepatology
New advances in the treatment of glomerular disease

Studies published in 2016 provide insights that bring us closer to achieving the goal of personalized therapy for primary glomerular diseases. Moreover, promising renal outcome data with new classes of glucose-lowering agents — SGLT2 inhibitors and GLP-1 agonists — offer new hope for patients with diabetic nephropathy.
- Rutger J. Maas
- Jack F. Wetzels
Year in Review19 Jan 2017 Nature Reviews Nephrology
Ingenious tactics to unravel complex kidney disease genetics

The genetic background of many kidney diseases is complex and involves multiple genes, genetic variants and molecular pathways. Here, we look at how researchers tackled this challenging topic in 2016, focusing on studies that used ingenious data-integration tactics, which led to new insights into kidney disease aetiology and renal disease progression.
- Kirsten Y. Renkema
- Nine V.A.M. Knoers
Year in Review19 Jan 2017 Nature Reviews Nephrology
The evolution of anti-angiogenic therapy for kidney cancer

Tyrosine kinase inhibitors that target pro-angiogenic pathways improve progression-free and overall survival in patients with metastatic kidney cancer and were thus tested in the adjuvant setting in studies published this past year. 2016 also saw the emergence of new inhibitors of pro-angiogenic pathways that might represent the next step in kidney cancer therapy.
- Chung-Han Lee
- Robert J. Motzer
Year in Review19 Jan 2017 Nature Reviews Nephrology
Managing organ dysfunction in critical care

Approaches to effectively prevent and manage organ dysfunction in critically ill patients remain elusive. Key studies in 2016 highlighted the challenges in finding effective treatments for renal failure in sepsis and assessed the optimal timing of renal replacement therapy initiation in critically ill patients with acute kidney injury.
- Ravindra L. Mehta
Year in Review19 Jan 2017 Nature Reviews Nephrology
Novel approaches to improve recipient and allograft outcomes

Kidney transplantation was the focus of numerous publications in 2016. Key studies demonstrated a survival advantage of HLA-incompatible kidney transplantation and suggested that novel approaches such as co-stimulation blockade using belatacept and treatment of antibody-mediated rejection using a C1 esterase inhibitor might prove to be future game changers.
- Paolo Malvezzi
- Lionel Rostaing
Year in Review19 Jan 2017 Nature Reviews Nephrology
Blood pressure goals, variability and SGLT2 blockade in CKD

Blood pressure (BP) goals and the management of BP in patients with chronic kidney disease (CKD) remain controversial topics. Key articles in the past year have addressed BP goals in CKD, the use of new agents to slow CKD progression and the effects of visit-to-visit variability in systolic BP on cardiovascular events and renal progression in patients with CKD.
- Debbie L. Cohen
- Raymond R. Townsend
Year in Review28 Dec 2016 Nature Reviews Nephrology
2016, the year of Zika virus

In 2016, the literature on neurological infections was, understandably, dominated by Zika virus. However, we should not overlook important publications on the treatment of cryptococcal and bacterial meningitis.
- Diederik van de Beek
- Matthijs C. Brouwer
Year in Review13 Jan 2017 Nature Reviews Neurology
Advances in brain tumour classification and therapy

Brain tumours encompass a heterogeneous collection of neoplasms, traditionally classified by histopathological criteria. In 2016, the WHO published an updated classification that, for the first time, defines brain tumour types according to integrated histological and molecular parameters. Furthermore, clinical trial results were reported that inform therapeutic decision-making in diffuse gliomas.
- Matthias Preusser
- Christine Marosi
Year in Review20 Jan 2017 Nature Reviews Neurology
Immune-directed therapies in MS — efficacy and limitations

In 2016, new highly active treatment options for relapsing–remitting multiple sclerosis (MS) emerged. At the same time, large clinical trials in progressive MS highlighted the limitations of immune-directed therapies, and called for new strategies to treat disease progression in MS.
- Bernhard Hemmer
- Mark Mühlau
Year in Review20 Jan 2017 Nature Reviews Neurology
Putting AD treatments and biomarkers to the test

Investigational treatments to impede the progression of Alzheimer disease (AD) are being evaluated in clinical trials, and biomarkers to detect and track the disease are being developed and deployed. Recent findings underscore the importance of ongoing clinical trials and biomarker developments in the understanding, treatment and prevention of AD.
- Eric M. Reiman
Year in Review13 Jan 2017 Nature Reviews Neurology
Progress in Parkinson disease and other movement disorders

In the field of movement disorders, areas that have seen important advances in 2016 include the pathogenesis of Parkinson disease involving extra-CNS α-synuclein pathology, treatment of hyperkinetic disorders with novel dopamine-depleting drugs, and MRI-guided ultrasound surgery for the treatment of essential tremor.
- Joseph Jankovic
Year in Review13 Jan 2017 Nature Reviews Neurology
Stroke is treatable, but prevention is the key

The past 2 years have seen major breakthroughs in endovascular treatment for acute ischaemic stroke. As highlighted in 2016, we now need to refine the logistics for delivery of this treatment, including patient selection. We should not forget, however, that it is better to prevent strokes in the first place.
- Ale Algra
- Marieke J. H. Wermer
Year in Review20 Jan 2017 Nature Reviews Neurology
Important milestones on the way to clinical translation

Regenerative medicine can be viewed as 'tissue engineering V2.0'. Discoveries and novel applications of technology advanced the field considerably in 2016, with the use of new biomaterials, stem cells and biologically active molecules.
- Daniel A. Grande
Year in Review5 Jan 2017 Nature Reviews Rheumatology
Gene expression profiling comes closer to the clinic

Gene expression profiling has been used for the first time to stratify patients with systemic lupus erythematosus (SLE) into potentially useful clinical groups, and also to further understand differences in the cell-specificity and nature of the interferon signature typical of SLE and other autoimmune diseases.
- Guillermo Barturen
- Marta E. Alarcón-Riquelme
Year in Review5 Jan 2017 Nature Reviews Rheumatology
Dermal white adipose tissue implicated in SSc pathogenesis

Several strands of new research indicate that skin-specific adipocyte progenitor cells regulate myofibroblasts and skin fibrosis in scleroderma.
- John Varga
- Roberta G. Marangoni
Year in Review12 Jan 2017 Nature Reviews Rheumatology
T follicular helper cells and the gut microbiome in arthritis

Rheumatoid arthritis is associated with an expansion of certain gut commensals, although the underlying mechanism remains unknown. In 2016, studies using experimental models of arthritis have begun to unravel the links between the gut microbiota, T follicular helper cells and arthritis.
- Veena Taneja
Year in Review12 Jan 2017 Nature Reviews Rheumatology
New tools for diagnosis and therapy

In 2016, there have been several major scientific achievements related to myositis, including the discovery of a novel autoantibody and the relationship between autoantibodies and distinct clinical phenotypes. Advances in the way clinical trials are conducted have also led to breakthroughs in treatment strategies.
- Ingrid E. Lundberg
Year in Review25 Jan 2017 Nature Reviews Rheumatology
Anti-NGF treatments for pain — two steps forward, one step back?

Inhibitors of β-nerve growth factor (NGF) have impressive effects in reducing musculoskeletal pain, but have also been associated with adverse events of unclear aetiology. Several studies in the past year have sought to clarify the relative benefits and risks of anti-NGF treatment.
- Nancy E. Lane
- Maripat Corr
Year in Review25 Jan 2017 Nature Reviews Rheumatology
New insights into interstitial cystitis and chronic pelvic pain syndromes

In 2016, immunohistochemical evidence revealed major differences in the inflammatory characteristics of Hunner and non-Hunner interstitial cystitis/bladder pain syndrome (IC/BPS). Evidence has also emerged that an isomer of testosterone, etio-S, might be a urinary biomarker of IC/BPS. Intravesical botulinum toxin injections became a standard treatment of IC/BPS. Furthermore, the International Continence Society has published a new Standard for Terminology for Chronic Pelvic Pain Syndromes.
- Jia-Fong Jhang
- Hann-Chorng Kuo
Year in Review29 Nov 2016 Nature Reviews Urology
Improved outcomes and precision medicine come within reach

2016 was an important year for prostate cancer research. New clinical data highlight the need for personalized treatment across clinical disease states and have changed clinical practice for men with metastatic disease. Molecular studies have characterized tumour heterogeneity and informed biomarker development for advanced disease and research into mechanisms of treatment resistance.
- Cora N. Sternberg
- Himisha Beltran
Year in Review4 Jan 2017 Nature Reviews Urology
Associating infection and incontinence with the female urinary microbiota

The discovery and confirmation of the female urinary microbiota in 2012 provided opportunities to improve insight into lower urinary tract disorders in women, including UTI and urgency urinary incontinence. Now, research in 2016 has shown that expanded culture techniques enable improved uropathogen detection and confirm that bacteria detected by culture-independent methods are alive.
- Linda Brubaker
- Alan J. Wolfe
Year in Review4 Jan 2017 Nature Reviews Urology
News in diagnosis, treatment, and risk group assessment

2016 has been a good year for research in non-muscle-invasive bladder cancer. Topics to see progress include risk assessment of patients treated with BCG maintenance, the role of repeat transurethral resection of the bladder (TURB), a prospective study of white-light versus narrow-band imaging, a meta-analysis regarding single instillation chemotherapy, and the effect of the use of fluorescence-guided TURB on progression.
- Richard Zigeuner
Year in Review4 Jan 2017 Nature Reviews Urology
RCC — advances in targeted therapeutics and genomics

Recent advances have been exciting in the genomics of and targeted therapeutics for renal cell carcinoma (RCC). New agents have been approved for advanced RCC, a novel agent targeting hypoxia-inducible factor 2α has shown considerable promise and molecular characterization of papillary RCC provides the foundation for development of targeted therapeutic approaches for this disease.
- W. Marston Linehan
- Christopher J. Ricketts
Year in Review29 Dec 2016 Nature Reviews Urology

Key Advances in Medicine 2017

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