ReviewCurrent topics in medicinal chemistry2014
Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease.
Review in Current topics in medicinal chemistry, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 73 papers.
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The trial behind it
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Who cites it
73 citing papers in PubMed, 115 citations in OpenAlex.
- Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1).Human molecular genetics · 2014Trial
- Mito-TEMPO improves survival rates inScience advances · 2026Article
- Cross-sectoral and cross-agency team science: successful strategies from the National Center for Advancing Translational Sciences.Frontiers in psychology · 2026Article
- A novel HPβCD-Cu(DDC)Scientific reports · 2025Article
- Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment.Journal of inherited metabolic disease · 2025Review
- Cyclodextrins as multifunctional tools for advanced biomaterials in tissue repair and regeneration.Bioactive materials · 2025Review
- Microglia regulate myelin clearance and cholesterol metabolism after demyelination via interferon regulatory factor 5.Cellular and molecular life sciences : CMLS · 2025Article
- Cholesterol and COVID-19-therapeutic opportunities at the host/virus interface during cell entry.Life science alliance · 2024Review
- Cyclodextrin-Mediated Cholesterol Depletion Induces Adiponectin Secretion in 3T3-L1 Adipocytes.International journal of molecular sciences · 2023Article
- 2-Hydroxypropyl-β-cyclodextrin (HPβCD) as a Potential Therapeutic Agent for Breast Cancer.Cancers · 2023Article
- Effect of Substitution Degree and Homogeneity on Cyclodextrin-Ligand Complex Stability: Comparison of Fenbufen and Fenoprofen Using CD and NMR Spectroscopy.International journal of molecular sciences · 2023Article
- Lysosomal Targeting of β-Cyclodextrin.Chemistry (Weinheim an der Bergstrasse, Germany) · 2023Article
- Alterations in Cholesterol and Phosphoinositides Levels in the Intracellular Cholesterol Trafficking Disorder NPC.Advances in experimental medicine and biology · 2023Article
- Article
- Hydroxypropyl-beta-cyclodextrin (HP-BCD) inhibits SARS-CoV-2 replication and virus-induced inflammatory cytokines.Antiviral research · 2022Article
- Experimental animal models of drug-induced sensorineural hearing loss: a narrative review.Annals of translational medicine · 2021Review
- Clinically translatable formulation strategies for systemic administration of nerve-specific probes.Advanced therapeutics · 2021Article
- Free sialic acid storage disorder: Progress and promise.Neuroscience letters · 2021Review
- Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts.ACS pharmacology & translational science · 2021Article
- Combinations of Piperine with Hydroxypropyl-β-Cyclodextrin as a Multifunctional System.International journal of molecular sciences · 2021Article
13 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
25 authors at 4 institutions in 2 countries.
Funding
Abstract
In 2010, the National Institutes of Health (NIH) established the Therapeutics for Rare and Neglected Diseases (TRND) program within the National Center for Advancing Translational Sciences (NCATS), which was created to stimulate drug discovery and development for rare and neglected tropical diseases through a collaborative model between the NIH, academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies. This paper describes one of the first TRND programs, the development of 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) for the treatment of Niemann-Pick disease type C1 (NPC1). NPC is a neurodegenerative, autosomal recessive rare disease caused by a mutation in either the NPC1 (about 95% of cases) or the NPC2 gene (about 5% of cases). These mutations affect the intracellular trafficking of cholesterol and other lipids, which leads to a progressive accumulation of unesterified cholesterol and glycosphingolipids in the CNS and visceral organs. Affected individuals typically exhibit ataxia, swallowing problems, seizures, and progressive impairment of motor and intellectual function in early childhood, and usually die in adolescence. There is no disease modifying therapy currently approved for NPC1 in the US. A collaborative drug development program has been established between TRND, public and private partners that has completed the pre-clinical development of HP-β-CD through IND filing for the current Phase I clinical trial that is underway. Here we discuss how this collaborative effort helped to overcome scientific, clinical and financial challenges facing the development of new drug treatments for rare and neglected diseases, and how it will incentivize the commercialization of HP-β-CD for the benefit of the NPC patient community.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.