Special Issue "Scientific Highlights in the First European Paediatric Translational Research Infrastructure Open Meeting"

A special issue of Pharmaceutics (ISSN 1999-4923).

Deadline for manuscript submissions: 31 December 2021.

Special Issue Editors

Dr. Rosa Maria Iacobazzi
E-Mail Website
Guest Editor
Experimental Pharmacology Laboratory, IRCCS Istituto Tumori Giovanni Paolo II, Bari, Italy
Interests: targeted anticancer drug delivery; nanotechnology applied tumor imaging and therapy

Special Issue Information

Dear Colleagues,

It is a great pleasure to announce the opening of a Special Issue dedicated to highlight the European Paediatric Translational Research Infrastructure (EPTRI) open meeting (https://www.cvbf.net/eptri/).

EPTRI is an EU-funded project aimed to design the framework for a new infrastructure dedicated to paediatric research that will work to accelerate paediatric drug development, from the discovery of drugs and the definition of biomarkers and biosamples to developmental pharmacology and age-tailored formulations and medical devices. The final goal is to facilitate the translation of new knowledge and scientific innovations into paediatric research and activities underpinning paediatric clinical studies.  

EPTRI involves partners from EU and non-EU countries, including non-profit research organisations, top-level universities, scientific and clinical centers of excellence. According to the survey conducted to map paediatric research facilities and competences, more than 300 research units declared their availability to provide technologies, services and paediatric expertise and have been grouped in the following Thematic Research Platforms: Paediatric Medicines Discovery, Paediatric Biosamples and Biomarkers, Developmental Pharmacology, Paediatric Medicines Formulations and Medical Devices.

This Special Issue is addressed to all researchers and partners involved in the development of paediatric medicines.

We welcome original research articles, reviews, opinion papers, editorials or short communications on the following topics:

  • paediatric medicines drug targets and methods of identification;
  • advanced cellular/in vitro models for paediatric drug targeting;
  • paediatric formulations and toxicology of drugs;
  • drug delivery design in paediatrics;
  • translational research.

Prof. Dr. Nunzio Denora
Dr. Rosa Maria Iacobazzi
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2200 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • paediatric formulations
  • drug targets
  • drug delivery
  • translational research

Published Papers (4 papers)

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Research

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Open AccessArticle
Breath-Triggered Drug Release System for Preterm Neonates
Pharmaceutics 2021, 13(5), 657; https://doi.org/10.3390/pharmaceutics13050657 - 04 May 2021
Viewed by 155
Abstract
A major disadvantage of inhalation therapy with continuous drug delivery is the loss of medication during expiration. Developing a breath-triggered drug release system can highly decrease this loss. However, there is currently no breath-triggered drug release directly inside the patient interface (nasal prong) [...] Read more.
A major disadvantage of inhalation therapy with continuous drug delivery is the loss of medication during expiration. Developing a breath-triggered drug release system can highly decrease this loss. However, there is currently no breath-triggered drug release directly inside the patient interface (nasal prong) for preterm neonates available due to their high breathing frequency, short inspiration time and low tidal volume. Therefore, a nasal prong with an integrated valve releasing aerosol directly inside the patient interface increasing inhaled aerosol efficiency is desirable. We integrated a miniaturized aerosol valve into a nasal prong, controlled by a double-stroke cylinder. Breathing was simulated using a test lung for preterm neonates on CPAP respiratory support. The inhalation flow served as a trigger signal for the valve, releasing humidified surfactant. Particle detection was performed gravimetrically (filter) and optically (light extinction). The integrated miniaturized aerosol valve enabled breath-triggered drug release inside the patient interface with an aerosol valve response time of <25 ms. By breath-triggered release of the pharmaceutical aerosol as a bolus during inhalation, the inhaled aerosol efficiency was increased by a factor of >4 compared to non-triggered release. This novel nasal prong with integrated valve allows breath-triggered drug release directly inside the nasal prong with short response time. Full article
Open AccessArticle
Anticonvulsant Action of GluN2A-Preferring Antagonist PEAQX in Developing Rats
Pharmaceutics 2021, 13(3), 415; https://doi.org/10.3390/pharmaceutics13030415 - 19 Mar 2021
Viewed by 382
Abstract
The GluN2A subunit of N-methyl-D-aspartate (NMDA) receptors becomes dominant during postnatal development, overgrowing the originally dominant GluN2B subunit. The aim of our study was to show changes of anticonvulsant action of the GluN2A subunit-preferring antagonist during postnatal development of rats. Possible anticonvulsant action [...] Read more.
The GluN2A subunit of N-methyl-D-aspartate (NMDA) receptors becomes dominant during postnatal development, overgrowing the originally dominant GluN2B subunit. The aim of our study was to show changes of anticonvulsant action of the GluN2A subunit-preferring antagonist during postnatal development of rats. Possible anticonvulsant action of GluN2A-preferring antagonist of NMDA receptors P = [[[(1S)-1-(4-bromophenyl)ethyl]amino](1,2,3,4-tetrahydro-2,3-dioxo-5-quinoxalinyl)methyl]phosphonic acid tetrasodium salt (PEAQX) (5, 10, 20 mg/kg s.c.) was tested in 12-, 18-, and 25-day-old rats in three models of convulsive seizures. Pentylenetetrazol-induced generalized seizures with a loss of righting reflexes generated in the brainstem were suppressed in all three age groups in a dose-dependent manner. Minimal clonic seizures with preserved righting ability exhibited only moderately prolonged latency after the highest dose of PEAQX. Anticonvulsant action of all three doses of PEAQX against cortical epileptic afterdischarges (generated in the forebrain) was found in the 25-day-old animals. The highest dose (20 mg/kg) was efficient also in the two younger groups, which might be due to lower specificity of PEAQX and its partial affinity to the GluN2B subunit. Our results are in agreement with the postero-anterior maturation gradient of subunit composition of NMDA receptors (i.e., an increase of GluN2A representation). In spite of the lower selectivity of PEAQX, our data demonstrate, for the first time, developmental differences in comparison with an antagonist of NMDA receptors with a dominant GluN2B subunit. Full article
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Review

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Open AccessReview
Patient-Derived Induced Pluripotent Stem Cells (iPSCs) and Cerebral Organoids for Drug Screening and Development in Autism Spectrum Disorder: Opportunities and Challenges
Pharmaceutics 2021, 13(2), 280; https://doi.org/10.3390/pharmaceutics13020280 - 19 Feb 2021
Viewed by 735
Abstract
Autism spectrum disorder (ASD) represents a group of neurodevelopmental diseases characterized by persistent deficits in social communication, interaction, and repetitive patterns of behaviors, interests, and activities. The etiopathogenesis is multifactorial with complex interactions between genetic and environmental factors. The clinical heterogeneity and complex [...] Read more.
Autism spectrum disorder (ASD) represents a group of neurodevelopmental diseases characterized by persistent deficits in social communication, interaction, and repetitive patterns of behaviors, interests, and activities. The etiopathogenesis is multifactorial with complex interactions between genetic and environmental factors. The clinical heterogeneity and complex etiology of this pediatric disorder have limited the development of pharmacological therapies. The major limit to ASD research remains a lack of relevant human disease models which can faithfully recapitulate key features of the human pathology and represent its genetic heterogeneity. Recent advances in induced pluripotent stem cells (iPSCs), reprogrammed from somatic cells of patients into all types of patient-specific neural cells, have provided a promising cellular tool for disease modeling and development of novel drug treatments. The iPSCs technology allowed not only a better investigation of the disease etiopathogenesis but also opened up the potential for personalized therapies and offered new opportunities for drug discovery, pharmacological screening, and toxicity assessment. Moreover, iPSCs can be differentiated and organized into three-dimensional (3D) organoids, providing a model which mimics the complexity of the brain’s architecture and more accurately recapitulates tissue- and organ-level disease pathophysiology. The aims of this review were to describe the current state of the art of the use of human patient-derived iPSCs and brain organoids in modeling ASD and developing novel therapeutic strategies and to discuss the opportunities and major challenges in this rapidly moving field. Full article
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Open AccessReview
The Neonatal and Juvenile Pig in Pediatric Drug Discovery and Development
Pharmaceutics 2021, 13(1), 44; https://doi.org/10.3390/pharmaceutics13010044 - 30 Dec 2020
Cited by 1 | Viewed by 810
Abstract
Pharmacotherapy in pediatric patients is challenging in view of the maturation of organ systems and processes that affect pharmacokinetics and pharmacodynamics. Especially for the youngest age groups and for pediatric-only indications, neonatal and juvenile animal models can be useful to assess drug safety [...] Read more.
Pharmacotherapy in pediatric patients is challenging in view of the maturation of organ systems and processes that affect pharmacokinetics and pharmacodynamics. Especially for the youngest age groups and for pediatric-only indications, neonatal and juvenile animal models can be useful to assess drug safety and to better understand the mechanisms of diseases or conditions. In this respect, the use of neonatal and juvenile pigs in the field of pediatric drug discovery and development is promising, although still limited at this point. This review summarizes the comparative postnatal development of pigs and humans and discusses the advantages of the juvenile pig in view of developmental pharmacology, pediatric diseases, drug discovery and drug safety testing. Furthermore, limitations and unexplored aspects of this large animal model are covered. At this point in time, the potential of the neonatal and juvenile pig as nonclinical safety models for pediatric drug development is underexplored. Full article
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