Review
Pharmacology & Pharmacy
Rebeca Lapresa, Jesus Agulla, Juan P. Bolanos, Angeles Almeida
Summary: Alzheimer's disease is the main cause of dementia in the elderly and there are currently no effective treatment options. Synapse dysfunction and loss are early pathological features, and inactivation of the APC/C enzyme leads to neurodegeneration and cognitive decline. Phosphorylation reactions induced by Alzheimer's disease exacerbate the enzyme inactivation, leading to the accumulation of multiple targets in damaged areas, further impairing neurons.
FRONTIERS IN PHARMACOLOGY
(2022)
Review
Biochemistry & Molecular Biology
Theodora Ntetsika, Paraskevi-Evita Papathoma, Ioanna Markaki
Summary: Parkinson's disease is the second most common neurodegenerative disease globally, with treatment options currently limited to symptom relief without halting or reversing disease progression. Large biomarker development programs are being carried out to improve patient selection and clinical trial outcome assessment. Research on Parkinson's disease mechanisms and ongoing clinical trials are crucial for future perspectives in the field.
MOLECULAR MEDICINE
(2021)
Review
Endocrinology & Metabolism
Katharina Wang, Joakim Crona, Felix Beuschlein, Ashley B. Grossman, Karel Pacak, Svenja Noelting
Summary: Molecular targeted therapy plays an important role in the treatment of metastatic pheochromocytomas and paragangliomas. Some therapies are already in use with promising results, while others are still under evaluation in clinical trials. The development of molecular targeted therapies is of great significance for future tumor treatment.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2022)
Review
Gastroenterology & Hepatology
Tao Zhang, Guangqi Gao, Lai-Yu Kwok, Zhihong Sun
Summary: The advent of high-throughput 'omics' technologies has improved our understanding of the connection between gut microbiome and Alzheimer's disease (AD). Modulating the gut microbiota has emerged as a novel approach for clinical management of AD. This review provides an overview of the association between gut dysbiosis and AD, the mechanisms of gut microbiota in driving AD progression, and the success and challenges of gut microbiome-targeted therapies in AD studies.
Review
Biochemistry & Molecular Biology
Abygail G. Chapdelaine, Gongqin Sun
Summary: Triple negative breast cancer (TNBC) is a heterogeneous group of breast cancers characterized by their lack of estrogen receptors, progesterone receptors, and the HER2 receptor. Developing targeted therapies for TNBC has been a major challenge due to its heterogeneity, and its treatment still largely relies on surgery, radiation therapy, and chemotherapy. Accumulating evidence supports TNBCs as multi-driver cancers, and a strategy designed to generate mechanism-based combination targeted therapies for TNBC is discussed.
Review
Biochemistry & Molecular Biology
Francesco Bruno, Alessia Pellerino, Luca Bertero, Riccardo Soffietti, Roberta Ruda
Summary: This article reviews recent progress in the molecular understanding of and therapeutic options for rare brain tumors in both children and adults, showing how specific molecular treatments can improve neurological symptoms and quality of life.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Biochemistry & Molecular Biology
Ryota Tamura, Masahiro Toda
Summary: Vestibular schwannoma is a benign tumor that originates from Schwann cells. Gamma knife radiosurgery is an accepted treatment, while targeted therapy and immunotherapy show potential for refractory cases.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Review
Oncology
Rille Pihlak, Caroline Fong, Naureen Starling
Summary: Stomach cancer is an aggressive disease with poor survival outcomes, but in the past two decades, clinical research has made significant progress in understanding targets and potential targeted treatments for advanced stomach cancer. This article summarizes the recent advancements in targeted therapies and precision medicine in gastric cancer and discusses new potential treatments.
Editorial Material
Medicine, General & Internal
Zhijun Zhou, Min Li
Summary: Targeted therapy is crucial for improving overall survival and reducing side effects in cancer treatment, but each patient responds differently to treatment. The development of cutting-edge technologies, such as next-generation sequencing, has allowed for the identification of more actionable targets. This special issue of BMC Medicine presents a collection of studies on targeted therapies for various cancer types, aiming to bridge the gap between genomic testing and precision medicine and spark innovations in improving the efficacy of targeted therapies.
Review
Oncology
Akanksha Sharma, Lauren Singer, Priya Kumthekar
Summary: Metastatic disease to the central nervous system is a serious complication with high mortality rates. Advances in targeted therapy and immunotherapy have led to successful penetration and efficacy within the CNS, improving treatment options for patients. Identifying key targets in tumorigenesis pathway and upcoming trials continue to promise significant progress in managing CNS metastases.
Article
Clinical Neurology
Dylan Goehner, Sandeep Kandregula, Christopher P. Carroll, Mario Zuccarello, Bharat Guthikonda, Jennifer A. Kosty
Summary: Moyamoya disease, described in 1957 as an idiopathic disease, has proven difficult to treat due to its complex molecular and cellular mechanisms involving carotid artery narrowing and compensatory angiogenesis. This article reviews the literature on the pathophysiology of moyamoya disease with a focus on potential therapeutic targets.
NEUROSURGICAL FOCUS
(2021)
Review
Hematology
Sabine Kayser, Mark J. Levis
Summary: Research on the pathogenic mechanisms of AML has made remarkable advances in recent years, especially in the importance of cytogenetic and molecular aberrations. The development of new compounds targeting AML at a molecular level, based on increased understanding of AML pathogenesis facilitated by next-generation sequencing, has turned many hopeful predictions into therapeutic realities.
BRITISH JOURNAL OF HAEMATOLOGY
(2022)
Review
Oncology
Sarah L. Higginbottom, Eva Tomaskovic-Crook, Jeremy M. Crook
Summary: The recent molecular stratification of gliomas by the World Health Organisation in 2021 is expected to improve outcomes for patients by developing targeted treatments. However, current research is hindered by the lack of preclinical modelling platforms that can accurately replicate the heterogeneity and cellular phenotypes of gliomas in the human brain microenvironment. Recent attention has focused on utilizing pluripotent stem cell-based and tissue engineering techniques to improve traditional models and develop clinically relevant therapies.
CANCER AND METASTASIS REVIEWS
(2023)
Review
Clinical Neurology
Vincent Zimmern, Berge Minassian, Christian Korff
Summary: Genetic sequencing technologies have led to an increase in the identification and characterization of monogenic epilepsy syndromes, resulting in a strong interest in developing precision therapies based on the unique molecular genetics of each syndrome. This review evaluates the clinical validity of these therapies and discusses their future in each syndrome.
FRONTIERS IN NEUROLOGY
(2022)
Review
Oncology
Rossella Reddavid, Simona Dagatti, Caterina Franco, Lucia Puca, Mariano Tomatis, Simona Corso, Silvia Giordano, Maurizio Degiuli
Summary: Despite the addition of molecular therapy in the clinical treatment of advanced and metastatic gastric cancer, the survival benefits of these novel therapies remain rather unclear, possibly due to inaccurate patient selection.