Sidonio named PI on upcoming VWD trial
Robert Sidonio Jr., MD, Clinical Director of Hemostasis/Thrombosis with the Emory University School of Medicine, will be the principle investigator for the American Thrombosis and Hemostasis Network von Willebrand disease (VWD) trial called "A Real-World Safety and Efficacy Study of Symptomatic VWD." The study begins in the third quarter of 2017 and is funded by Shire, a global innovator in specialty biopharmaceuticals. Dr. Sidonio's PI is Angela Weyland, MD, from the University of Michigan.
This $2.5 million award will seek to characterize children and adults with VWD in the United States and focus on characterization of the bleeding symptoms of those VWD patients with symptomatic disease, requiring medical intervention on a regular basis. The study will focus on certain populations, such as adolescent girls and women with VWD, as they comprise nearly 80 percent of all medical claims, according to a recent medical claims study.
The study will also seek to further understand the different types of VWD and the severity of bleeding symptoms. It will provide the framework to answer future questions regarding why some patients have worse bleeding than others, while also documenting successful strategies in treatment, including factor replacement.
Dr. Sidonio hopes that this project will jump-start additional studies in VWD using the ATHNdataset, including but not limited to long-term effects of angiodysplasia in VWD, aging in VWD, cardiovascular effects from VWD and changes in levels of VWD over time.
VWD is the most common bleeding disorder in humans. It affects approximately 1 in 1,000 people in the United States. There have been more than 7,000 patients with VWD described as part of the ATHNdataset.
Wetmore named PI on new first-in-pediatrics study
Cynthia Wetmore, MD, PhD, Director of the Development Therapeutics Program at Aflac Cancer and Blood Disorders Center, is the principal investigator on a new first-in-pediatrics study involving abemaciclib, an inhibitor of cyclin dependent kinases 4 and 6 (Cdk 4/6) for children with newly diagnosed diffuse pontine glioma and for patients with recurrent or progressive solid tumors, including malignant brain tumors. Dr. Wetmore holds the Carter Samuel Martin endowed chair in developmental therapeutics and this new study is generously supported by the CURE Childhood Cancer Foundation and Eli Lilly Pharmaceuticals.
Abemaciclib is taken as a pill twice a day and is a small molecule inhibitor of Cdk 4/6, which is an important enzyme regulating progression through the cell cycle and cell division. Inhibition of this kinase is particularly damaging to tumor cells, which are often not able to recover from such a blockade. Unlike other inhibitors of Cdk 4/6, this agent is readily able to cross the blood brain barrier, making it a very attractive option for brain tumors that are resistant to other types of therapy. This class of inhibitors has been approved by the Federal Drug Administration (FDA) for use in certain types of breast cancer, but this is the first study to include pediatric patients.
In addition to developing our own approach, we are participating in the ongoing CTL-019 (Novartis) trial and are a referral center.