This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Endocrine/metabolic disease

Glycosphingolipid storage disorders

Adenosine A2A receptor (ADORA2A)

In vitro studies suggest ADORA2A agonists could help treat Niemann-Pick disease type C1 (NPC1). ADORA2A plays a role in regulating lysosomal pH and calcium ion (Ca2+) levels, which are downregulated in NPC1. In primary fibroblasts from patients with NPC1, the ADORA2A agonist CGS21680 increased lysosomal Ca2+ levels and decreased cholesterol accumulation to levels comparable to those of fibroblasts from healthy controls.
Next steps could include testing ADORA2A agonists in animal models of NPC1.
Adenosine Therapeutics LLC has the ADORA2A agonist Stedivaze apadenoson in Phase III testing for use as a pharmacological stress agent in myocardial perfusion imaging (MPI).
Swedish Orphan Biovitrum AB, CBT Development Ltd. and Ergomed Clinical Research Ltd. have the ADORA2A agonist BVT.115959 in Phase II testing to treat pain.
Forest Laboratories Inc. and Zalicus Inc. have the ADORA2A agonist ALT313 in preclinical testing to treat cancer. Forest also has ALT313 in preclinical testing to treat pain.
CGS21680 is a research reagent.

SciBX 6(40); doi:10.1038/scibx.2013.1129
Published online Oct. 17, 2013

Patent and licensing status unavailable

Visentin, S. et al. J. Neurosci.; published online Sept. 25, 2013;
doi:10.1523/JNEUROSCI.0558-13.2013
Contact: Patrizia Popoli, National Institute of Health, Rome, Italy
e-mail:

patrizia.popoli@iss.it