Abstract
While mortality is low for intraocular retinoblastoma patients in the developed world who receive aggressive multimodal therapy, partial or full loss of vision occurs in approximately 50% of patients with advanced bilateral retinoblastoma. Therapies that preserve vision and reduce late effects are needed. Because clinical trials for retinoblastoma are difficult due to the young age of the patient population and relative rarity of the disease, robust preclinical testing of new therapies is critical. The last decade has seen advances towards identifying new therapies including the development of animal models of retinoblastoma for preclinical testing, progress in local drug delivery to reach intraocular targets, and improved understanding of the underlying biological mechanisms that give rise to retinoblastoma. This review discusses advances in these areas, with a focus on discovery and development of small molecules for the treatment of retinoblastoma, including novel targeted therapeutics such as inhibitors of the MDMX-p53 interaction (nutlin-3a), histone deacetylase (HDAC) inhibitors, and spleen tyrosine kinase (SYK) inhibitors.
Keywords: Chemoreduction, Ocular drug delivery, Retinoblastoma.
Mini-Reviews in Medicinal Chemistry
Title:Progress in Small Molecule Therapeutics for the Treatment of Retinoblastoma
Volume: 16 Issue: 6
Author(s): Eleanor M. Pritchard, Michael A. Dyer and R. Kiplin Guy
Affiliation:
Keywords: Chemoreduction, Ocular drug delivery, Retinoblastoma.
Abstract: While mortality is low for intraocular retinoblastoma patients in the developed world who receive aggressive multimodal therapy, partial or full loss of vision occurs in approximately 50% of patients with advanced bilateral retinoblastoma. Therapies that preserve vision and reduce late effects are needed. Because clinical trials for retinoblastoma are difficult due to the young age of the patient population and relative rarity of the disease, robust preclinical testing of new therapies is critical. The last decade has seen advances towards identifying new therapies including the development of animal models of retinoblastoma for preclinical testing, progress in local drug delivery to reach intraocular targets, and improved understanding of the underlying biological mechanisms that give rise to retinoblastoma. This review discusses advances in these areas, with a focus on discovery and development of small molecules for the treatment of retinoblastoma, including novel targeted therapeutics such as inhibitors of the MDMX-p53 interaction (nutlin-3a), histone deacetylase (HDAC) inhibitors, and spleen tyrosine kinase (SYK) inhibitors.
Export Options
About this article
Cite this article as:
Pritchard M. Eleanor, Dyer A. Michael and Guy Kiplin R., Progress in Small Molecule Therapeutics for the Treatment of Retinoblastoma, Mini-Reviews in Medicinal Chemistry 2016; 16 (6) . https://dx.doi.org/10.2174/1389557515666150722100610
DOI https://dx.doi.org/10.2174/1389557515666150722100610 |
Print ISSN 1389-5575 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5607 |
Call for Papers in Thematic Issues
Bioprospecting of Natural Products as Sources of New Multitarget Therapies
According to the Convention on Biological Diversity, bioprospecting is the exploration of biodiversity and indigenous knowledge to develop commercially valuable products for pharmaceutical and other applications. Bioprospecting involves searching for useful organic compounds in plants, fungi, marine organisms, and microorganisms. Natural products traditionally constituted the primary source of more than ...read more
Computational Frontiers in Medicinal Chemistry
The thematic issue "Computational Frontiers in Medicinal Chemistry" provides a robust platform for delving into state-of-the-art computational methodologies and technologies that significantly propel advancements in medicinal chemistry. This edition seeks to amalgamate top-tier reviews spotlighting the latest trends and breakthroughs in the fusion of computational approaches, including artificial intelligence (AI) ...read more
Natural Products and Dietary Supplements in Alleviation of Metabolic, Cardiovascular, and Neurological Disorders
Metabolic disorders like diabetes, obesity, inflammation, oxidative stress, cancer etc, cardiovascular disorders like angina, myocardial infarction, congestive heart failure etc as well as neurological disorders like Alzheimer?s, Parkinson?s, Epilepsy, Depression, etc are the global burden. They covered the major segment of the diseases and disorders from which the human community ...read more
Natural Products in Drug Discovery
Natural products have always been one of the important ways of drug discovery due to their novel skeleton and diverse functional group characteristics. According to statistics, between 1981 and 2019, the FDA approved a total of 1,394 small molecule drugs for marketing, of which 930 marketed drugs originated from the ...read more
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
Related Articles
-
Testicular Germ Cell Tumors: A Paradigm for the Successful Treatment of Solid Tumor Stem Cells
Current Cancer Therapy Reviews Targeting Nodal and Cripto-1: Perspectives Inside Dual Potential Theranostic Cancer Biomarkers
Current Medicinal Chemistry Potassium Channels: Novel Emerging Biomarkers and Targets for Therapy in Cancer
Recent Patents on Anti-Cancer Drug Discovery Molecular and Genetic Profiling of Prostate Cancer: Implications for Future Therapy
Current Cancer Therapy Reviews Beneficial and Adverse Effects of Molecularly Targeted Therapies for Acute Promyelocytic Leukemia in Central Nervous System
CNS & Neurological Disorders - Drug Targets Antineovascular Agents in the Treatment of Eye Diseases
Current Pharmaceutical Design Stem Cell Fate Decisions: The Role of Heparan Sulfate in the Control of Autocrine and Paracrine Signals
Current Stem Cell Research & Therapy Liver X Receptors: Potential Novel Targets in Cardiovascular Diseases
Current Drug Targets - Cardiovascular & Hematological Disorders Peptides or Small Molecules? Different Approaches to Develop More Effective CDK Inhibitors
Current Medicinal Chemistry Retraction Note: Low Doses of CPS49 and Flavopiridol Combination as Potential Treatment for Advanced Prostate Cancer
Current Pharmaceutical Biotechnology MicroRNAs in Human Diseases
Recent Patents on DNA & Gene Sequences Regulators of the G1 Phase of the Cell Cycle and Neurogenesis
Central Nervous System Agents in Medicinal Chemistry Sirolimus and its Analogs and its Effects on Vascular Diseases
Current Pharmaceutical Design Interleukin-6/interleukin-6 Receptor Pathway as a New Therapy Target in Epithelial Ovarian Cancer
Current Pharmaceutical Design CD95 Signaling in Cancer Treatment
Current Pharmaceutical Design The Role of Stress Proteins in Prostate Cancer
Current Genomics Progress of HDAC Inhibitor Panobinostat in the Treatment of Cancer
Current Drug Targets Ultrasound Therapeutics– A Review
Current Medical Imaging Rb Function in the Apoptosis and Senescence of Non-Neuronal and Neuronal Cells: Role in Oncogenesis
Current Molecular Medicine Biochemical Mechanisms of Cisplatin Cytotoxicity
Anti-Cancer Agents in Medicinal Chemistry