scholarly journals Precision medicine: do not neglect the hurdles

2016 ◽  
Vol 1 (1) ◽  
Author(s):  
Lilla Landeck ◽  
Patricia Carrigan ◽  
Khusru Asadullah
Keyword(s):  
Precision Medicine ◽  
Targeted Therapies ◽  
Scientific Data ◽  
The Past

Tremendous expectations have been connected with precision medicine in the past years. Be-side the advantages that this type of therapy offers we should be aware of its challenges too. In this issue we will highlight on specific challenges that the pharmacological industry is opposed with when de-veloping new targeted therapies. In addition, we will discuss issues with the reproducibility of published scientific data.

Biological therapy of allergic diseases during the COVID-19 pandemic

2020 ◽  
Vol 17 (3) ◽  
pp. 115-120
Author(s):  
Elena S. Fedenko ◽  
Olga G. Elisyutina ◽  
Natalia I. Il`ina
Keyword(s):  
Biological Therapy ◽  
Treatment Approach ◽  
Allergic Diseases ◽  
Scientific Data ◽  
Biological Therapies ◽  
Active Infection ◽  
The Past ◽  
Type 2 Immune Response ◽  
Antiviral Medications

The outbreak of the SARS-CoV-2-induced Coronavirus Disease 2019 (COVID-19) pandemic started in December 2019 in Wuhan, China, continued to spread across the globe and spanned 188 countries. Under the new circumstances treatment approach for T2 allergic diseases such as asthma, chronic hives, atopic dermatitis, and sinusitis with polyps has been changed. In the past years, new biological therapies monoclonal antibodies for these diseases have been developed targeting different aspects of the type 2 immune response. New knowledge on the COVID-19 disease course raises many issues around the safety of biologicals in patients with active infection, as well as their interactions with antiviral medications. In Russia new biological therapies entered clinical practice but its effectiveness and safety still are not known. This newsletter is based on Considerations on Biologicals for Patients with allergic disease in times of the COVID-19 pandemic: an EAACI Statement and the latest scientific data.

Enzyme-Instructed Self-assembly in Biological Milieu for Theranostics Purpose

2019 ◽  
Vol 26 (8) ◽  
pp. 1351-1365 ◽  
Author(s):  
Zhentao Huang ◽  
Qingxin Yao ◽  
Simin Wei ◽  
Jiali Chen ◽  
Yuan Gao
Keyword(s):  
Small Molecules ◽  
Precision Medicine ◽  
Self Assembly ◽  
Public Healthcare ◽  
Enzymatic Conversion ◽  
Vaccine Adjuvants ◽  
New Paradigm ◽  
Cancer Treatments ◽  
The Past ◽  
Biological Milieu

Precision medicine is in an urgent need for public healthcare. Among the past several decades, the flourishing development in nanotechnology significantly advances the realization of precision nanomedicine. Comparing to well-documented nanoparticlebased strategy, in this review, we focus on the strategy using enzyme instructed selfassembly (EISA) in biological milieu for theranostics purpose. In principle, the design of small molecules for EISA requires two aspects: (1) the substrate of enzyme of interest; and (2) self-assembly potency after enzymatic conversion. This strategy has shown its irreplaceable advantages in nanomedicne, specifically for cancer treatments and Vaccine Adjuvants. Interestingly, all the reported examples rely on only one kind of enzymehydrolase. Therefore, we envision that the application of EISA strategy just begins and will lead to a new paradigm in nanomedicine.

scholarly journals SpheroidPicker for automated 3D cell culture manipulation using deep learning

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Istvan Grexa ◽  
Akos Diosdi ◽  
Maria Harmati ◽  
Andras Kriston ◽  
Nikita Moshkov ◽  
...  
Keyword(s):  
Cell Culture ◽  
Precision Medicine ◽  
High Throughput Screening ◽  
Standard Sample ◽  
Ex Vivo ◽  
Low Cost ◽  
3D Cell Culture ◽  
Syringe Pump ◽  
Experimental Conditions ◽  
The Past

AbstractRecent statistics report that more than 3.7 million new cases of cancer occur in Europe yearly, and the disease accounts for approximately 20% of all deaths. High-throughput screening of cancer cell cultures has dominated the search for novel, effective anticancer therapies in the past decades. Recently, functional assays with patient-derived ex vivo 3D cell culture have gained importance for drug discovery and precision medicine. We recently evaluated the major advancements and needs for the 3D cell culture screening, and concluded that strictly standardized and robust sample preparation is the most desired development. Here we propose an artificial intelligence-guided low-cost 3D cell culture delivery system. It consists of a light microscope, a micromanipulator, a syringe pump, and a controller computer. The system performs morphology-based feature analysis on spheroids and can select uniform sized or shaped spheroids to transfer them between various sample holders. It can select the samples from standard sample holders, including Petri dishes and microwell plates, and then transfer them to a variety of holders up to 384 well plates. The device performs reliable semi- and fully automated spheroid transfer. This results in highly controlled experimental conditions and eliminates non-trivial side effects of sample variability that is a key aspect towards next-generation precision medicine.

Vaso-occlusion in sickle cell disease: pathophysiology and novel targeted therapies

Hematology ◽  
2013 ◽  
Vol 2013 (1) ◽  
pp. 362-369 ◽  
Author(s):  
Deepa Manwani ◽  
Paul S. Frenette
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Targeted Therapies ◽  
Therapeutic Intervention ◽  
Fetal Hemoglobin ◽  
Cell Disease ◽  
The Past ◽  
Symptomatic Management

Abstract Recurrent and unpredictable episodes of vaso-occlusion are the hallmark of sickle cell disease. Symptomatic management and prevention of these events using the fetal hemoglobin–reactivating agent hydroxyurea are currently the mainstay of treatment. Discoveries over the past 2 decades have highlighted the important contributions of various cellular and soluble participants in the vaso-occlusive cascade. The role of these elements and the opportunities for therapeutic intervention are summarized in this review.

The Steam Vaporizer

PEDIATRICS ◽  
1972 ◽  
Vol 50 (1) ◽  
pp. 168-169
Author(s):  
John A. Washington
Keyword(s):  
Scientific Data ◽  
The Past

The report "Steam Vaporizers—Therapy or Tragedy," in the January 1972 issue, points out that little scientific data substantiates the use of these appliances. It must be conceded that practitioners, who measure their successes partly on their avoidance of hospitalizations, tend to follow well worn routines wearing blinders. I share with Dr. Barich a dread of devices, common in the past, which heat to scalding temperatures a half gallon or more of water. Since the introduction of squat plastic vaporizers which emit steam from a core fed from an unheated reservoir, I have seen no accidents.

Skin as a Source of Nosocomial Infection: Directions for Future Research

1986 ◽  
Vol 7 (S2) ◽  
pp. 113-117 ◽  
Author(s):  
Dennis G. Maki
Keyword(s):  
Clinical Trials ◽  
Nosocomial Infection ◽  
Scientific Data ◽  
Control Measures ◽  
Future Research ◽  
Controlled Clinical Trials ◽  
Nosocomial Pathogens ◽  
The Past ◽  
Infection Control Measures ◽  
Per Se

Whereas infections of the skin per se comprise only a fraction of all institutionally-acquired infections, the skin has become one of the most important reservoirs of nosocomial pathogens in the hospital. Professor Noble has provided a scholarly review of the increasing importance of the major constituents of the cutaneous microflora as nosocomial pathogens and what we know of their epidemiology. Unfortunately, the empiricism and limited scientific data which underlie essential infection control measures in this area, particularly in regard to cutaneous antisepsis and handwashing, is almost incongruous in an era in which controlled clinical trials have dominated most other areas of medicine. The numerous outbreaks traced to contaminated antiseptics and disinfectants over the past two decades, stand as mute testimony to the inadequate investigative attention this area has received.

Precision medicine: beyond genomics to targeted therapies

2016 ◽  
Vol 13 (2) ◽  
pp. 97-100
Author(s):  
Ralph Snyderman ◽  
David Spellmeyer
Keyword(s):  
Precision Medicine ◽  
Targeted Therapies

Diagnostic Pathology in the Era of Precision Medicine

2021 ◽  
Vol 11 (03) ◽  
pp. 100-101
Author(s):  
Summaya Shawanna ◽  
◽  
Yasmeen Taj
Keyword(s):  
Gene Expression ◽  
Targeted Therapy ◽  
Precision Medicine ◽  
Cancer Patients ◽  
Expression Analysis ◽  
Gene Expression Analysis ◽  
Diagnostic Pathology ◽  
Neoplastic Diseases ◽  
The Past ◽  
The One

The ever-increasing importance of targeted therapy in the management of cancerous and non-neoplastic diseases calls for novel, advanced techniques of diagnostic pathology. The past decade has witnessed substantial changes in how cancer patients are managed, with a pronounced focus on precision medicine based on genomic profiles and gene expression analysis, thus discarding the “one-size-fits-all” approach1.

Impact of rapid multigene assays with short turnaround time (TAT) on the development of precision medicine for non-small cell lung cancer (NSCLC).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 9094-9094
Author(s):  
Shingo Matsumoto ◽  
Takaya Ikeda ◽  
Kiyotaka Yoh ◽  
Akira Sugimoto ◽  
Terufumi Kato ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Clinical Trials ◽  
Precision Medicine ◽  
Targeted Therapies ◽  
Advanced Nsclc ◽  
Genetic Alterations ◽  
Braf V600e ◽  
Gene Testing ◽  
Genomic Screening ◽  
Nsclc Patients

9094 Background: A variety of oncogene drivers have been identified in NSCLC and molecularly-stratified precision medicine has led to improved survival in advanced NSCLC. Next-generation sequencing (NGS)-based testing is utilized to detect actionable gene alterations; however, the TAT of NGS is often too long to translate into clinical decision making. Thus, rapid multi-gene testing alternatives are needed. Methods: A lung cancer genomic screening project (LC-SCRUM-Asia) capturing clinical outcome was established in 2013 to identify patients with oncogene drivers and to support the development of novel targeted therapies. Since February 2013 to May 2019 (LC-SCRUM-Asia 1st-phase), single gene testing and/or a targeted NGS assay, Oncomine Comprehensive Assay (OCA), were used for the genomic screening. Since June 2019 to December 2020 (2nd-phase), a multi-gene PCR assay (Amoy 9-in-1 test) and a rapid NGS assay (Genexus/Oncomine Precision Assay [OPA]) were also implemented as rapid multi-gene testing. Results: A total of 10667 Japanese NSCLC patients, including 6826 in the 1st-phase and 3841 in the 2nd-phase, were enrolled in the LC-SCRUM-Asia. Success rate for OCA: 93%, for 9-in-1 test: 98%, for Genexus/OPA: 96%. Median TAT for OCA: 21 days, for 9-in-1 test: 3 days, for Genexus/OPA: 4 days. The frequencies of genetic alterations detected in the 1st-/2nd-phase were EGFR: 17/24%, KRAS: 15/16%, HER2 ex20ins: 4/3%, ALK fusions: 3/3%, RET fusions: 3/2%, ROS1 fusions: 3/2%, MET ex14skip: 2/2%, BRAF V600E: 1/1%, NRG1 fusions: 0/0.2% and NTRK3 fusions: 0.05/0.04%. Overall percent agreement of 9-in-1 test compared with OCA for EGFR/KRAS/HER2/BRAF/MET/ALK/ROS1/RET/NTRK3 alterations was 98%, and that of OPA compared with OCA was 95%. The rate of patients who received targeted therapies as 1st-line treatment was significantly elevated in the 2nd-phase compared with the 1st-phase (510/3841 [13%] vs. 567/6826 [8%], p < 0.001). Through the genomic screening, 1410 (37%) and 1269 (18%) candidate patients for clinical trials of KRAS, HER2, BRAF, MET, ALK, ROS1, RET or TRK-targeted drugs were identified in the 2nd-phase and in the 1st-phase, respectively. The rate of patients who were actually enrolled into the genotype-matched clinical trials were also significantly higher in the 2nd-phase than in the 1st-phase (222 [6%] vs. 186 [3%], p < 0.001). In 1st-line treatments for advanced NSCLC patients, the median progression-free survival was 8.5 months (95% CI, 7.7−9.4) in the 2nd-phase (n = 1839) versus 6.1 months (95% CI, 5.9−6.3) in the 1st-phase (n = 4262) (p < 0.001). Conclusions: Both the 9-in-1 test and Genexus/OPA had short TATs (3−4 days), high success rates (96−98%) and good concordance (95−98%) compared with another NGS assay (OCA). These rapid multi-gene assays highly contributed to enabling precision medicine and the development of targeted therapies for advanced NSCLC.

scholarly journals Targeted Therapies and Immunotherapies in the Treatment of Esophageal Cancers

2019 ◽  
Vol 7 (10) ◽  
pp. 100 ◽  
Author(s):  
Adam Barsouk ◽  
Prashanth Rawla ◽  
Andreas V. Hadjinicolaou ◽  
John Sukumar Aluru ◽  
Alexander Barsouk
Keyword(s):  
Targeted Therapies ◽  
Treatment Options ◽  
Survival Rates ◽  
Line Treatment ◽  
Her2 Positive ◽  
Specific Patient ◽  
The Past ◽  
Fda Approval ◽  
The Us ◽  
Third Line

Esophageal cancer (EC) is among the most frequent and deadly cancers around the world. While esophageal adenocarcinoma (EAC) has one of the fastest-growing incidences amongst cancers in the US, it also has one of the lowest survival rates due to the limited effective treatment options. Fortunately, in the past decade, two targeted therapies and an immunotherapy agent have been approved by the FDA for metastatic EAC and esophageal squamous cell carcinoma (ESCC), with several more currently being considered for approval. In terms of immunotherapies, in July 2019, the FDA approved the PD1 inhibitor pembrolizumab for second-line treatment of PDL1-positive, advanced or metastatic ESCC. Two years before, pembrolizumab had been approved for the third-line treatment of PDL1-positive EAC. The PD1 inhibitor nivolumab, which was found in one study to outperform chemotherapy irrespective of PDL1 status, has yet to secure FDA approval. In terms of targeted therapies, although as many as 90% of EC cases show upregulated EGFR, anti-EGFR therapy has not been shown to improve survival. Ramucirumab, an antibody targeting both VEGF and HER2/neu receptors, has been approved for the treatment of refractory EAC, while the anti-HER2 monoclonal antibody (mAb) trastuzumab has been approved as front-line treatment for HER2-positive cases which account for approximately 20% of ECs. Although these targeted therapies and immunotherapies have resulted in significant improvements in survival for specific patient populations that are positive for certain biomarkers, such as PDL1 and HER2/neu, the survival rates remain low for a large proportion of the metastatic EC patient population, necessitating the development of further targeted treatment options.

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