Yale Scientists Discover a New Therapy for a Chronic Brain Disease

Diseased mice treated with mock (left) or angiopoietin-2-neutralizing antibody (right). The antibody blunts cerebral cavernous malformation progression.

Yale scientists have discovered a new and effective therapy for cerebral cavernous malformations.

A team of researchers led by Yale professor of pathology Wang Min have pinpointed a marker that contributes to a chronic condition affecting the brain. Known as cerebral cavernous malformations, the condition is characterized by a tangle of capillaries in the brain’s white matter, which can cause headaches, seizures, bleeding, and even death. No effective therapy exists. (It has been reported that Olympic runner Florence Griffith Joyner, who died suddenly in 1998, had this condition.)

Using an animal model of the disease, Min and his co-authors identified a growth factor (angiopoietin-2) that when blocked by an antibody, eliminates the malformations. “We have discovered a new and effective therapy for this potentially debilitating disorder,” he noted.

The study was published August 22 in Nature Medicine.

Reference: “Endothelial exocytosis of angiopoietin-2 resulting from CCM3 deficiency contributes to cerebral cavernous malformation” by Huanjiao Jenny Zhou, Lingfeng Qin, Haifeng Zhang, Wenwen Tang, Weidong Ji, Yun He, Xiaoling Liang, Zongren Wang, Qianying Yuan, Alexander Vortmeyer, Derek Toomre, Germaine Fuh, Minghong Yan, Martin S Kluger, Dianqing Wu and Wang Min, 22 August 2016, Nature Medicine.
DOI: 10.1038/nm.4169