ArticleGenes & development2018
DPM-1001 decreased copper levels and ameliorated deficits in a mouse model of Wilson's disease.
Article in Genes & development, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed, 70 citations in OpenAlex.
- Cuproptosis and the bioinorganic basis of copper-induced cell death.Journal of biological inorganic chemistry : JBIC : a publication of the Society of Biological Inorganic Chemistry · 2026Review
- PTP1B inhibition promotes microglial phagocytosis in Alzheimer's disease models by enhancing SYK signaling.Proceedings of the National Academy of Sciences of the United States of America · 2026Article
- Rapid transcellular hepatic copper depletion by ARBM-101 rescues severe liver damage in Wilson disease rodents.Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie · 2025Article
- N,N'-bis(2-mercaptoethyl)isophthalamide (NBMI) as a novel chelator for Wilson's disease.Free radical biology & medicine · 2025Article
- Natural Copper Ion Scavenger: Investigation of the Hepatoprotective Effects of Green Tea Extract in Toxic-Milk Mice with Wilson's Disease Model.Foods (Basel, Switzerland) · 2025Article
- Current Management of Neurological Wilson's Disease.Tremor and other hyperkinetic movements (New York, N.Y.) · 2025Review
- Review
- Therapeutic potential of targeting protein tyrosine phosphatases in liver diseases.Acta pharmaceutica Sinica. B · 2024Review
- Protein tyrosine phosphatase 1B in metabolic and cardiovascular diseases: from mechanisms to therapeutics.Frontiers in cardiovascular medicine · 2024Review
- A small molecule inhibitor of PTP1B and PTPN2 enhances T cell anti-tumor immunity.Nature communications · 2023Article
- Can Allostery Be a Key Strategy for Targeting PTP1B in Drug Discovery? A Lesson from Trodusquemine.International journal of molecular sciences · 2023Review
- Protein Tyrosine Phosphatases: Mighty oaks from little acorns grow.IUBMB life · 2023Article
- Copper in cancer: from limiting nutrient to therapeutic target.Frontiers in oncology · 2023Review
- The Role of Copper Homeostasis in Brain Disease.International journal of molecular sciences · 2022Review
- Pan-cancer analysis of cuproptosis regulation patterns and identification of mTOR-target responder in clear cell renal cell carcinoma.Biology direct · 2022Article
- Review
- Human Protein Tyrosine Phosphatase 1B (PTP1B): From Structure to Clinical Inhibitor Perspectives.International journal of molecular sciences · 2022Review
- Connecting copper and cancer: from transition metal signalling to metalloplasia.Nature reviews. Cancer · 2022Review
- Early Diagnosis of Wilson's Disease in Children in Southern China by Using Common Parameters.Frontiers in genetics · 2022Article
- The cuproptosis-related signature predicts prognosis and indicates immune microenvironment in breast cancer.Frontiers in genetics · 2022Article
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
Abstract
The levels of copper, which is an essential element in living organisms, are under tight homeostatic control. Inactivating mutations in ATP7B, a P-type Cu-ATPase that functions in copper excretion, promote aberrant accumulation of the metal, primarily the in liver and brain. This condition underlies Wilson's disease, a severe autosomal recessive disorder characterized by profound hepatic and neurological deficits. Current treatment regimens rely on the use of broad specificity metal chelators as "decoppering" agents; however, there are side effects that limit their effectiveness. Here, we present the characterization of DPM-1001 {methyl 4-[7-hydroxy-10,13-dimethyl-3-({4-[(pyridin-2-ylmethyl)amino]butyl}amino)hexadecahydro-1H-cyclopenta[a]phenanthren-17-yl] pentanoate} as a potent and highly selective chelator of copper that is orally bioavailable. Treatment of cell models, including fibroblasts derived from Wilson's disease patients, eliminated adverse effects associated with copper accumulation. Furthermore, treatment of the toxic milk mouse model of Wilson's disease with DPM-1001 lowered the levels of copper in the liver and brain, removing excess copper by excretion in the feces while ameliorating symptoms associated with the disease. These data suggest that it may be worthwhile to investigate DPM-1001 further as a new therapeutic agent for the treatment of Wilson's disease, with potential for application in other indications associated with elevated copper, including cancer and neurodegenerative diseases.
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