Editor’s Choice Article | Trends of Clinical Trials for Drug Development in Rare Diseases

Author(s): Ryuichi Sakate, Akiko Fukagawa, Yuri Takagaki, Hanayuki Okura, Akifumi Matsuyama*.

Graphical Abstract:


Background: Drug development for rare diseases is challenging because it is difficult to obtain relevant data from very few patients. It must be informative to grasp current status of clinical trials for drug development in rare diseases.

Objective: Clinical trials in rare diseases are to be outlined and compared among the US, EU and Japan.

Method: ClinicalTrials.gov (NCT, National Clinical Trial), EU Clinical Trials Register (EUCTR) and the Japan Primary Registries Network (JPRN) were analyzed. Clinical trials involving information on rare diseases and drugs were extracted by text-mining, based on the diseases and drugs derived from Orphanet and DrugBank, respectively.

Results: In total, 28,526 clinical trials were extracted, which studied 1,535 rare diseases and 1,539 drugs. NCT had the largest number of trials, involving 1,252 diseases and 1,332 drugs. EUCTR and JPRN also had registry-specific diseases (250 and 22, respectively) and drugs (172 and 29, respectively) that should not be missed. Among the 1,535 rare diseases, most diseases were studied in only a limited number of trials; 70% of diseases were studied in fewer than 10 trials, and 28% were studied in only one. Additionally, most studied rare diseases were cancer-related ones.

Conclusion: This study has revealed the characteristics of the clinical trials in rare diseases among the US, EU and Japan. The number of trials for rare diseases was limited especially for non-cancerrelated ones. This information could contribute to drug development such as drug-repositioning in rare diseases.



Read out more at: http://www.eurekaselect.com/162697/article

Open Access Articles – Oncolytic Virotherapy for Breast Cancer Treatment

Journal Name: Current Gene Therapy

Author(s): Samia M. O`Bryan, J. Michael Mathis*.


Breast cancer continues to be a leading cause of mortality among women. While at an early stage, localized breast cancer is easily treated; however, advanced stages of disease continue to carry a high mortality rate. The discrepancy in treatment success highlights that current treatments are insufficient to treat advanced-stage breast cancer. As new and improved treatments have been sought, one therapeutic approach has gained considerable attention. Oncolytic viruses are uniquely capable of targeting cancer cells through intrinsic or engineered means. They come in many forms, mainly from four major virus groups as defined by the Baltimore classification system. These vectors can target and kill cancer cells, and even stimulate immunotherapeutic effects in patients. This review discusses not only individual oncolytic viruses pursued in the context of breast cancer treatment but also the emergence of combination therapies with current or new therapies, which has become a particularly promising strategy for treatment of breast cancer. Overall, oncolytic virotherapy is a promising strategy for increased treatment efficacy for advanced breast cancer and consequently provides a unique platform for personalized treatments in patients.

For more details, please visit: http://www.eurekaselect.com/165254

MOST ACCESSED ARTICLE – Targeted Nanosystems for Cancer Therapy – Current Cancer Therapy Reviews

Journal: Current Cancer Therapy Reviews

Author(s): Ruhee Jain, Tahseen Khan, Sourabh Jain, Ashutosh Pal Jain, Aakanchha Jain

Graphical Abstract:


Background: Significant shortcomings have been displayed in conventional chemotherapeutics delivery which possesses some genuine side effects including harm of the immunity and different organs with quickly multiplying cells because of nonparticular focus on the absence of dissolvability and powerlessness to enter the tumor core bringing about debilitated treatment with diminished dosage and with low survival rate. Rapid development has adapted nanocarriers as distinct therapeutics which can directly access the tumor cells specifically with expanded medication limitation and cell take-up for cancer treatment.

Methodology: This review focuses on core objective of drug targeting to the cancerous cells by demonstrating the advantages of the young medical field, “nanocarriers” including liposomes, polymer based nanoparticles, metal based nanoparticles, dendrimers, protein linked systems, co-polymers and fullerenes, which have been proven remarkably promising in enhancing drug distribution and bioavailability, increasing half life and achieving targeted drug delivery, thus reducing toxicity.

Conclusion: Here we provide an update on the recent clinical trials in nanocarrier based therapy of colon rectal cancer, food and drug administration (FDA) approved nanomedicines for cancer and those in nanoplatforms which have reached an advanced stage of clinical development.
To access the article, please visit: http://www.eurekaselect.com/153540/article


Testimonial by Yuanyuan Wang!

Yuanyuan Wang

Contributed Article: “Chinese Herbal Medicine for the Treatment of Depression: Applications, Efficacies and Mechanisms


Shuangqing Zhai


Contributed Article: “Chinese Herbal Medicine for the Treatment of Depression: Applications, Efficacies and Mechanisms

Testimonial by Chiara Ambrogio!

Chiara Ambrogio.jpg

Contributed Article: “Back to the bench? MEK and ERK inhibitors for the treatment of KRAS mutant lung adenocarcinoma”

Editor’s Choice – “Use of Recursive Partitioning Analysis in Clinical Trials and Meta-Analysis of Randomized Clinical Trials, 1990-2016”

Journal: Reviews on Recent Clinical Trials

Author(s): Martha Maria Fors, Carmen Elena Viada, Paloma Gonzalez

Graphical Abstract:



Background: Recursive Partitioning Analysis (RPA) is a very flexible non parametric algorithm that allows classification of individuals according to certain criteria, particularly in clinical trials, the method is used to predict response to treatment or classify individuals according to prognostic factors.

Objectives: In this paper we examine how often RPA is used in clinical trials and in meta-analysis.

Methods: We reviewed abstracts published between 1990 and 2016, and extracted data regarding clinical trial phase, year of publication, type of treatment, medical indication and main evaluated endpoints.

Results: One hundred and eighty three studies were identified; of these 43 were meta-analyses and 23 were clinical trials. Most of the studies were published between 2011 and 2016, for both clinical trials and meta-analyses of randomized clinical trials. The prediction of overall survival and progression free survival were the outcomes most evaluated, at 43.5% and 51.2% respectively. Regarding the use of RPA in clinical trials, the brain was the most common site studied, while for meta-analytic studies, other cancer sites were also studied. The combination of chemotherapy and radiation was seen frequently in clinical trials.

Conclusion: Recursive partitioning analysis is a very easy technique to use, and it could be a very powerful tool to predict response in different subgroups of patients, although it is not widely used in clinical trials.

Read more here: http://www.eurekaselect.com/145588 


Highlighted Article Flyer for the journal “Reviews on Recent Clinical Trials”



A very Happy Birthday to Alberto Zanoboni!


Alberto Zanoboni

Editor-in-Chief: Reviews on Recent Clinical Trials

Alberto Zanoboni Oncologia Medica

Fondazione Poliambulanza Brescia Italy

Highlighted Article Flyer for the journal “Current Women’s Health Reviews”



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