ACC is a rare but lethal malignancy. There is currently a lack of effective therapies for advanced and metastatic ACC patients. In fact, most patients develop a recurrence even after complete surgical resection. The emerging field of drug repositioning provides new possibilities for ACC treatment with advantages such as existing pharmacological and toxicity data to help accelerate therapy development. For rare cancers, such as ACC, drug repositioning can play an essential role in finding a treatment for a disease that may otherwise be neglected due to high costs.
Researchers at the National Cancer Institute, Endocrine Oncology Branch developed a method of treating adrenocortical cancer (ACC) using niclosamide. In collaboration with investigators at the National Center for Advancing Translational Sciences, the researchers identified niclosamide as an active compound with anticancer affects. Niclosamide is an anti-parasitic drug that targets the WNT/β-catenin pathway in ACC and induces apoptosis and cell cycle arrest. Preclinical studies confirmed the inhibition of cellular proliferation by niclosamide in both time- and dose-dependent studies. Thus, niclosamide may prove to be an effective and novel strategy for ACC treatment.
- Niclosamide as a therapeutic for ACC
- Therapeutic strategy for ACC patients who do not respond to standard therapies
- Niclosamide is an FDA-approved drug with a good safety profile
- Targets the WNT/β-catenin pathway which is important for adrenocortical cancer tumorigenesis
- Existing pharmacological and toxicity data reduces high costs of developing new treatments
Electron Kebebew, Lisa J. Zhang (NCI), Ya-Qin N. Zhang (NCATS), Min Shen (NCATS)
K. Satoh et al. [PMID: 26873959]
- U.S. Provisional: U.S. Provisional Patent Application Number 62/280,521, Filed 19 Jan 2016
- Foreign Filed: Foreign Filed - Patent Application PCT/US2017/013873, Filed 18 Jan 2017