Article
Multidisciplinary Sciences
Hugo Garcia, Alice S. Serafin, Flora Silbermann, Esther Poree, Amandine Viau, Clementine Mahaut, Katy Billot, Eleonore Birgy, Meriem Garfa-Traore, Stephanie Roy, Salome Ceccarelli, Manon Mehraz, Pamela C. Rodriguez, Berangere Deleglise, Laetitia Furio, Fabienne Jabot-Hanin, Nicolas Cagnard, Elaine Del Nery, Marc Fila, Soraya Sin-Monnot, Corinne Antignac, Stanislas Lyonnet, Pauline Krug, Remi Salomon, Jean-Philippe Annereau, Alexandre Benmerah, Marion Delous, Luis Briseno-Roa, Sophie Saunier
Summary: Nephronophthisis (NPH) is a common genetic kidney disease in children with no current curative treatment available. However, research has shown that prostaglandin E-2 receptor agonists can ameliorate the pleotropic phenotypes associated with the loss of NPHP1, potentially serving as a therapeutic option for NPH-related ciliopathies in juveniles.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2022)
Article
Biochemistry & Molecular Biology
Mariusz Sandomierski, Katarzyna Adamska, Maria Ratajczak, Adam Voelkel
Summary: Chitosan scaffolds have great potential in biomedical applications, particularly in bone tissue engineering. This study successfully achieved controlled drug release in chitosan scaffolds by incorporating drug carriers, specifically for the osteoporosis drug risedronate. The addition of zeolite to the chitosan scaffold resulted in better drug retention compared to hydroxyapatite, demonstrating promising results for the potential use of this material in bone tissue engineering.
INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES
(2022)
Article
Pharmacology & Pharmacy
Justyna Magdalena Hermanowicz, Bartlomiej Kalaska, Krystyna Pawlak, Beata Sieklucka, Joanna Miklosz, Mariusz Mojzych, Dariusz Pawlak
Summary: MM-129, a novel inhibitor targeting BTK/PI3K/AKT/mTOR and PD-L1, demonstrates antitumor activity against colon cancer with favorable safety profile in zebrafish and rodent models. Pharmacokinetic studies on Wistar rats show quick absorption and high bioavailability of MM-129 after intraperitoneal administration, indicating its potential as a safe and well-tolerated anticancer formulation for future treatment of patients with colon cancer.
Article
Spectroscopy
Mariusz Sandomierski, Marcel Jakubowski, Maria Ratajczak, Adam Voelkel
Summary: This study explores a new method for treating osteoporosis, using biocompatible zinc titanate coated titanium implants as potential new carriers for drugs, and confirms the effective release and attachment of the drug.
SPECTROCHIMICA ACTA PART A-MOLECULAR AND BIOMOLECULAR SPECTROSCOPY
(2022)
Review
Pharmacology & Pharmacy
Kate H. Kennedy, Krisstel Gomez, Natalie J. Thovmasian, Dennis C. Chang
Summary: There is a narrative that large pharma relies on biotech and academia for drug innovation. However, our research shows that while biotechs and academia do originate more drugs, large pharma remains important in shepherding drugs through clinical development and approval, and in originating high-impact novel therapies. Our analysis of FDA-approved first-in-class oncology drugs from 2010 through 2020 indicates that large pharma was the sole originator of only 14% of these drugs, while small biotechs originated 46%, and academic labs 14%. Despite this, large pharma companies launched or were involved in launching 76% of these drugs, including three of the five top-selling ones.
DRUG DISCOVERY TODAY
(2023)
Review
Oncology
Vivek Subbiah, Howard A. Burris III, Razelle Kurzrock
Summary: Advances in next-generation sequencing, targeted therapies, and immunotherapies have transformed cancer therapy. Basket trials, an effective clinical trial design, evaluate the efficacy of therapies based on specific molecular alterations across multiple cancer types, offering potential for personalized treatments and revolutionizing cancer care.
Review
Geriatrics & Gerontology
B. A. Cedeno-Veloz, J. Erviti Lopez, M. Gutierrez-Valencia, L. Leache Alegria, L. C. Saiz, A. M. Rodriguez Garcia, M. Sanchez Latorre, R. Ramirez Velez, M. Izquierdo, N. Martinez-Velilla
Summary: Antiresorptive drugs have a statistically significant effect on preventing hip fractures in older adults, but with moderate quality of evidence and a high number needed to treat. There is no reduction in the risk of hip fractures in individuals aged 75 years and older. More randomized controlled trials are needed in very old osteoporotic adults.
JOURNAL OF NUTRITION HEALTH & AGING
(2022)
Article
Chemistry, Physical
Mariusz Sandomierski, Wiktoria Stachowicz, Adam Patalas, Karol Grochalski, Wieslaw Grabon, Adam Voelkel
Summary: Osteoporosis is a common metabolic disease of the skeletal system, characterized by impaired bone strength and increased risk of low-energy fractures. Tissue engineering with surface modification and bisphosphonates may be a new method for preventing the deterioration of osteoporosis. Titanium implants coated with magnesium or zinc zeolite have been prepared and successfully released risedronate at a low level, indicating the potential for endoprostheses in patients with bone diseases.
Review
Pharmacology & Pharmacy
Barbara Costa, Nuno Vale
Summary: The COVID-19 pandemic has posed an unprecedented challenge in finding effective drugs, with anticancer drugs showing potential for repurposing. Clinical management needs further improvement to effectively treat this infection.
Article
Oncology
Richard D. Baird, Constanza Linossi, Mark Middleton, Simon Lord, Adrian Harris, Jordi Rodon, Christof Zitt, Ulrike Fiedler, Keith M. Dawson, Nicolas Leupin, Michael T. Stumpp, Andreas Harstrick, Analia Azaro, Stefanie Fischer, Aurelius Omlin
Summary: MP0250 is a first-in-class DARPin drug candidate with suitable tolerability and appropriate pharmacokinetic properties for further development in combination with other anticancer therapies. Signs of single-agent antitumor activity were observed.
JOURNAL OF CLINICAL ONCOLOGY
(2021)
Article
Biochemistry & Molecular Biology
Chien-Ching Lee, Chao-Ming Hung, Chung-Hwan Chen, Yi-Chiang Hsu, Yuan-Pin Huang, Tsung-Bin Huang, Mon-Juan Lee
Summary: A novel aptamer-based competitive drug screening platform was developed for osteoporosis, facilitating high-throughput screening for potential small-molecule sclerostin inhibitors and offering a reliable strategy for target-specific new drug discovery. The potential sclerostin inhibitors obtained in this study showed promising effects on decreasing sclerostin levels and increasing alkaline phosphatase activity in osteocyte and mesenchymal stem cells, suggesting their potential as new therapeutics for osteoporosis.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Clinical Neurology
Samantha E. Stilley, Akshay S. Naraine, Krishna P. Yadavalli, Samantha L. Maki, Elyse M. Jutte, Jared M. Kahn, Alexis A. Surtel, Salvatore D. Lepore, Ken Dawson-Scully
Summary: In this study, a new class of small-molecule synthetic compounds that can ameliorate seizure-like behavior was described. The electroshock assay in Caenorhabditis elegans was used to examine seizure duration and determine that these compounds, called resveramorphs, act through an irreversible binding mechanism.
Review
Gastroenterology & Hepatology
Mathieu Uzzan, Yoram Bouhnik, Maria Abreu, Harris A. Ahmad, Shashi Adsul, Hilde Carlier, Marla Dubinsky, Matthew Germinaro, Vipul Jairath, Irene Modesto, Eric Mortensen, Neeraj Narula, Ezequiel Neimark, Alessandra Oortwijn, Marijana Protic, David T. Rubin, Young S. Oh, Jolanta Wichary, Laurent Peyrin-Biroulet, Walter Reinisch
Summary: The enrolment rates of clinical trials in inflammatory bowel disease (IBD) have decreased dramatically in recent years, causing delays, increased costs, and failures to develop novel treatments. This article aims to describe the current bottlenecks of IBD clinical trial enrolment and propose solutions. A taskforce of experienced IBD clinical trialists explored the reasons for declining enrolment rates at different levels and generated potential solutions. It is recommended that this joint effort serves as the basis for clinical trial transformation to improve recruitment.
JOURNAL OF CROHNS & COLITIS
(2023)
Article
Oncology
David G. DeNardo, Anna Galkin, Jakob Dupont, Lei Zhou, Johanna Bendell
Summary: Resistance to immune checkpoint inhibitors and other anticancer therapies is often associated with the accumulation of myeloid-derived suppressor cells and tumor-associated macrophages in the tumor microenvironment. Targeting MDSC recruitment or function, particularly through the activation of CD11b, shows promise in enhancing antitumor immune responses and improving the effectiveness of immunotherapy.
JOURNAL FOR IMMUNOTHERAPY OF CANCER
(2021)
Article
Oncology
Xiaoli Li, Yuyan Liu, Minhui Zhu, Cuixia He, Yuanyuan Xu, Jiaxiang Ding, Ying Wang, Rongfang Shan, Bingyan Liu, Yuzhou Ding, Jing Xie, Huan Zhou, Zhiqiang Wang, Yuanyuan Liu
Summary: SH-1028 is a third-generation EGFR tyrosine kinase inhibitor used to treat locally advanced or metastatic non-small cell lung cancer (NSCLC). This study investigated the drug-drug interaction (DDI) potential of SH-1028 through in vitro experiments and clinical trials. In vitro studies showed that SH-1028 was primarily metabolized by CYP3A4 and had slight inhibitory effects on the activities of CYP1A2, 2B6, 2C19, 2D6, and 3A4 enzymes. The phase I clinical trial demonstrated that co-administration of rifampicin (a CYP3A4 inducer) and itraconazole (a CYP3A4 inhibitor) affected the pharmacokinetics of SH-1028. While SH-1028 did not significantly induce or inhibit human CYPs at the clinically expected dose, interactions with CYP3A4-affecting drugs or foods should be closely monitored during the clinical use of SH-1028. (Clinical trial registration number: CTR20210558)
INVESTIGATIONAL NEW DRUGS
(2023)