-
Fertility and Sterility Jun 2018There is a great deal of hype surrounding the concept of personalized medicine. Personalized medicine is rooted in the belief that since individuals possess nuanced and... (Review)
Review
There is a great deal of hype surrounding the concept of personalized medicine. Personalized medicine is rooted in the belief that since individuals possess nuanced and unique characteristics at the molecular, physiological, environmental exposure, and behavioral levels, they may need to have interventions provided to them for diseases they possess that are tailored to these nuanced and unique characteristics. This belief has been verified to some degree through the application of emerging technologies such as DNA sequencing, proteomics, imaging protocols, and wireless health monitoring devices, which have revealed great inter-individual variation in disease processes. In this review, we consider the motivation for personalized medicine, its historical precedents, the emerging technologies that are enabling it, some recent experiences including successes and setbacks, ways of vetting and deploying personalized medicines, and future directions, including potential ways of treating individuals with fertility and sterility issues. We also consider current limitations of personalized medicine. We ultimately argue that since aspects of personalized medicine are rooted in biological realities, personalized medicine practices in certain contexts are likely to be inevitable, especially as relevant assays and deployment strategies become more efficient and cost-effective.
Topics: Cost-Benefit Analysis; Humans; Motivation; Precision Medicine; Reproductive Techniques, Assisted; Research Design
PubMed: 29935653
DOI: 10.1016/j.fertnstert.2018.05.006 -
Genome Apr 2021Precision medicine is an emerging approach to clinical research and patient care that focuses on understanding and treating disease by integrating multi-modal or... (Review)
Review
Precision medicine is an emerging approach to clinical research and patient care that focuses on understanding and treating disease by integrating multi-modal or multi-omics data from an individual to make patient-tailored decisions. With the large and complex datasets generated using precision medicine diagnostic approaches, novel techniques to process and understand these complex data were needed. At the same time, computer science has progressed rapidly to develop techniques that enable the storage, processing, and analysis of these complex datasets, a feat that traditional statistics and early computing technologies could not accomplish. Machine learning, a branch of artificial intelligence, is a computer science methodology that aims to identify complex patterns in data that can be used to make predictions or classifications on new unseen data or for advanced exploratory data analysis. Machine learning analysis of precision medicine's multi-modal data allows for broad analysis of large datasets and ultimately a greater understanding of human health and disease. This review focuses on machine learning utilization for precision medicine's "big data", in the context of genetics, genomics, and beyond.
Topics: Artificial Intelligence; Genomics; Humans; Machine Learning; Precision Medicine
PubMed: 33091314
DOI: 10.1139/gen-2020-0131 -
Dialogues in Clinical Neuroscience 2019This article covers the renaissance of classical psychedelic drugs such as psilocybin and LSD plus 3,4-methylene dioxymethamphetamine (MDMA-ecstasy) in psychiatric... (Review)
Review
This article covers the renaissance of classical psychedelic drugs such as psilocybin and LSD plus 3,4-methylene dioxymethamphetamine (MDMA-ecstasy) in psychiatric research. These drugs were used quite extensively before they became prohibited. This ban had little impact on recreational use, but effectively stopped research and clinical treatments, which up to that point had looked very promising in several areas of psychiatry. In the past decade a number of groups have been working to re-evaluate the utility of these substances in medicine. So far highly promising preliminary data have been produced with psilocybin in anxiety, depression, smoking, alcoholism, and with MDMA for post-traumatic stress disorder (PTSD) and alcoholism. These findings have led to the European Medicines Agency approving psilocybin for a phase 3 study in treatment-resistant depression and the Food and Drug Administration for PTSD with MDMA. Both trials should read out in 2020, and if the results are positive we are likely to see these medicines approved for clinical practice soon afterwards. .
Topics: Hallucinogens; Humans; Ketamine; Lysergic Acid Diethylamide; Mental Disorders; N-Methyl-3,4-methylenedioxyamphetamine; Psilocybin; Psychiatry
PubMed: 31636488
DOI: 10.31887/DCNS.2019.21.2/dnutt -
British Journal of Clinical Pharmacology Mar 2015Safety and efficacy data on many medicines used in children are surprisingly scarce. As a result children are sometimes given ineffective medicines or medicines with... (Review)
Review
Safety and efficacy data on many medicines used in children are surprisingly scarce. As a result children are sometimes given ineffective medicines or medicines with unknown harmful side effects. Better and more relevant clinical trials in children are needed to increase our knowledge of the effects of medicines and to prevent the delayed or non-use of beneficial therapies. Clinical trials provide reliable evidence of treatment effects by rigorous controlled testing of interventions on human subjects. Paediatric trials are more challenging to conduct than trials in adults because of the paucity of funding, uniqueness of children and particular ethical concerns. Although current regulations and initiatives are improving the scope, quantity and quality of trials in children, there are still deficiencies that need to be addressed to accelerate radically equitable access to evidence-based therapies in children.
Topics: Child; Clinical Trials as Topic; Drug Evaluation; Drugs, Investigational; Ethics, Medical; Humans; Pediatrics; Research Design
PubMed: 24325152
DOI: 10.1111/bcp.12305 -
Infectious Diseases of Poverty Jan 2020Neglected tropical diseases (NTDs) have long been overlooked in the global health agenda. They are intimately related to poverty, cause important local burdens of... (Review)
Review
BACKGROUND
Neglected tropical diseases (NTDs) have long been overlooked in the global health agenda. They are intimately related to poverty, cause important local burdens of disease, but individually do not represent global priorities. Yet, NTDs were estimated to affect close to 2 billion people at the turn of the millennium, with a collective burden equivalent to HIV/AIDS, tuberculosis, or malaria. A global response was therefore warranted.
MAIN TEXT
The World Health Organization (WHO) conceived an innovative strategy in the early 2000s to combat NTDs as a group of diseases, based on a combination of five public health interventions. Access to essential NTD medicines has hugely improved thanks to strong public-private partnership involving the pharmaceutical sector. The combination of a WHO NTD roadmap with clear targets to be achieved by 2020 and game-changing partner commitments endorsed in the London Declaration on Neglected Tropical Diseases, have led to unprecedented progress in the implementation of large-scale preventive treatment, case management and care of NTDs. The coming decade will see as challenges the mainstreaming of these NTD interventions into Universal Health Coverage and the coordination with other sectors to get to the roots of poverty and scale up transmission-breaking interventions. Chinese expertise with the elimination of multiple NTDs, together with poverty reduction and intersectoral action piloted by municipalities and local governments, can serve as a model for the latter. The international community will also need to keep a specific focus on NTDs in order to further steer this global response, manage the scaling up and sustainment of NTD interventions globally, and develop novel products and implementation strategies for NTDs that are still lagging behind.
CONCLUSIONS
The year 2020 will be crucial for the future of the global response to NTDs. Progress against the 2020 roadmap targets will be assessed, a new 2021-2030 NTD roadmap will be launched, and the London Declaration commitments will need to be renewed. It is hoped that during the coming decade the global response will be able to further build on today's successes, align with the new global health and development frameworks, but also keep focused attention on NTDs and mobilize enough resources to see the effort effectively through to 2030.
Topics: Communicable Disease Control; Global Health; Humans; Neglected Diseases; Poverty; Tropical Medicine
PubMed: 31987053
DOI: 10.1186/s40249-020-0630-9 -
Annals of the Rheumatic Diseases Mar 2017To develop a Glucocorticoid Toxicity Index (GTI) to assess glucocorticoid (GC)-related morbidity and GC-sparing ability of other therapies.
OBJECTIVES
To develop a Glucocorticoid Toxicity Index (GTI) to assess glucocorticoid (GC)-related morbidity and GC-sparing ability of other therapies.
METHODS
Nineteen experts on GC use and outcome measures from 11 subspecialties participated. Ten experts were from the USA; nine from Canada, Europe or Australia. Group consensus methods and multicriteria decision analysis (MCDA) were used. A Composite GTI and Specific List comprise the overall GTI. The Composite GTI reflects toxicity likely to change during a clinical trial. The Composite GTI toxicities occur commonly, vary with GC exposure, and are weighted and scored. Relative weights for items in the Composite GTI were derived by group consensus and MCDA. The Specific List is designed to capture GC toxicity not included in the Composite GTI. The Composite GTI was evaluated by application to paper cases by the investigators and an external group of 17 subspecialists.
RESULTS
Thirty-one toxicity items were included in the Composite GTI and 23 in the Specific List. Composite GTI evaluation showed high inter-rater agreement (investigators κ 0.88, external raters κ 0.90). To assess the degree to which the Composite GTI corresponds to expert clinical judgement, participants ranked 15 cases by clinical judgement in order of highest to lowest GC toxicity. Expert rankings were then compared with case ranking by the Composite GTI, yielding excellent agreement (investigators weighted κ 0.87, external raters weighted κ 0.77).
CONCLUSIONS
We describe the development and initial evaluation of a comprehensive instrument for the assessment of GC toxicity.
Topics: Consensus; Decision Support Techniques; Dermatology; Glucocorticoids; Humans; Infectious Disease Medicine; Interdisciplinary Communication; Nephrology; Neurology; Observer Variation; Ophthalmology; Pediatrics; Psychiatry; Pulmonary Medicine; Reproducibility of Results; Rheumatology; Severity of Illness Index
PubMed: 27474764
DOI: 10.1136/annrheumdis-2016-210002 -
Archives of Medical Research Jan 2021Human Mesenchymal Stem Cells (hMSCs) are multipotent stem cells capable of renewing themselves and differentiation in vitro into different kinds of tissues. In vivo...
INTRODUCTION
Human Mesenchymal Stem Cells (hMSCs) are multipotent stem cells capable of renewing themselves and differentiation in vitro into different kinds of tissues. In vivo hMSCs are sources of trophic factors modulating the immune system and inducing intrinsic stem cells to repair damaged tissues. Currently, there are multiple clinical trials (CT) using hMSCs for therapeutic purposes in a large number of clinical settings.
MATERIAL AND METHODS
The search strategy on clinicaltrials.gov has focused on the key term "Mesenchymal Stem Cells", and the inclusion and exclusion criteria were separated into two stages. Stage 1, CT on phases 1-4: location, the field of application, phase, and status. For stage 2, CT that have published outcome results: field of application, treatment, intervention model, source, preparation methods, and results.
RESULTS
By July 2020, there were a total of 1,138 registered CT. Most studies belong to either phase 2 (61.0%) or phase 1 (30.8%); most of them focused in the fields of traumatology, neurology, cardiology, and immunology. Only 18 clinical trials had published results: the most common source of isolation was bone marrow; the treatment varied from 1-200 M hMSCs; all of them have similar preparation methods; all of them have positive results with no serious adverse effects.
CONCLUSIONS
There appears to be a broad potential for the clinical use of hMSCs with no reported serious adverse events. There are many trials in progress, their future results will help to explore the therapeutic potential of these promising cellular sources of medicinal signals.
Topics: Cell Differentiation; Clinical Trials as Topic; Humans; Medicine; Mesenchymal Stem Cell Transplantation; Mesenchymal Stem Cells; Regenerative Medicine
PubMed: 32977984
DOI: 10.1016/j.arcmed.2020.08.006 -
Ugeskrift For Laeger Sep 2023Polygenic risk scores (PRS) identify at-risk individuals for many common diseases. A discussion of strengths and limitations is carried out in this review. PRS... (Review)
Review
Polygenic risk scores (PRS) identify at-risk individuals for many common diseases. A discussion of strengths and limitations is carried out in this review. PRS complement traditional genetic testing and have shown utility in establishing a proper diagnosis and guiding primary and secondary prevention. Some individuals with high PRS have risks similar to those with monogenic predisposition. Limitations include potential misinterpretations, problems with application across ancestries, and limited usefulness in low-heritability traits. Despite its shortcomings PRS are predicted to play major roles in the future of personal medicine and genetic testing.
Topics: Humans; Risk Factors; Genetic Testing; Medicine; Secondary Prevention; Genetic Predisposition to Disease; Genome-Wide Association Study
PubMed: 37873989
DOI: No ID Found -
Clinical Pharmacology and Therapeutics Jan 2023Real-world data/evidence (RWD/RWE) may provide insightful information on medicines' clinical effects to guide regulatory decisions. While its contribution has been...
Real-world data/evidence (RWD/RWE) may provide insightful information on medicines' clinical effects to guide regulatory decisions. While its contribution has been recognized for safety monitoring and disease epidemiology across medicines' life cycles, using RWD/RWE to demonstrate efficacy requires further evaluation. This study aimed to (i) characterize RWD/RWE presented by applicants to support claims on medicines' efficacy within initial marketing authorization applications (MAAs) and extension of indication applications (EoIs), and (ii) analyze the contribution of RWD/RWE to regulatory decisions on medicines' benefit-risk profile. RWD/RWE was included to support efficacy in 32 MAAs and 14 EoIs submitted 2018-2019. Of these, RWD/RWE was part of the preauthorization package of 16 MAAs and 10 EoIs, and was (i) considered supporting the regulatory decision in 10 applications (five MAAs, five EoIs), (ii) considered not supporting the regulatory decision in 11 (seven MAAs, four EoIs), and (iii) not addressed at all in the evaluation of 5 applications (four MAAs, one EoI). Common limitations of submitted RWD/RWE included missing data, lack of representativeness of populations, small sample size, absence of an adequate or prespecified analysis plan, and risk of several types of bias. The suitability of RWD/RWE in a given application still requires a case-by-case analysis considering its purpose of use, implying reflection on the data source, together with its assets and limitations, study objectives and designs, and the overall data package issued. Early interactions and continuous dialogues with regulators and relevant stakeholders is key to optimize fit-for-purpose RWE generation, enabling its broader use in medicines development.
Topics: Humans; Decision Making; Europe; Government Regulation; Medicine
PubMed: 36254408
DOI: 10.1002/cpt.2766 -
Ugeskrift For Laeger Mar 2023Play is a non-invasive, safe, and inexpensive intervention that can help children and adolescents better manage difficult aspects of hospitalisation. Play has existed in... (Review)
Review
Play is a non-invasive, safe, and inexpensive intervention that can help children and adolescents better manage difficult aspects of hospitalisation. Play has existed in hospitals for decades but is emerging as an interdisciplinary scientific field. The field concerns all medical specialties and healthcare professionals working with children. In this review, we describe play within different clinical contexts and recommend that directed and non-directed play activities should be prioritised in future paediatric departments. We also emphasise the need for professionalisation and research in the area.
Topics: Child; Adolescent; Humans; Hospitals; Health Personnel; Hospital Departments; Medicine
PubMed: 36896603
DOI: No ID Found