Emicizumab (ACE910) Shows Promising Results for Treatment of Hemophilia A

This essay was featured in the BloodStream Pod.

    Promising results have been reported in early clinical trials of Emicizumab, otherwise known as ACE910. This subcutaneous therapy is being developed for individuals with hemophilia A, with and without inhibitors. ACE910 has been on a fast-track approval process since September 2015 when it was given breakthrough therapy status by the U.S. Food and Drug Administration. Subsequent study is currently being conducted by the makers of the drug, Tokyo-based Chugai Pharmaceutical Co. Ltd. Its hope is that ACE910 will soon offer a new prophylactic treatment option to individuals with hemophilia A.

 

Shire/Baxalta Merger

This essay was featured in the BloodStream Pod.

   On June 3, 2016, Shire announced its combination with Baxalta in a $32 million merger that will notably expand its global reach. The combination positions Shire as the, “global leader in rare diseases.”  In addition to the significant anticipated revenue, the announcement also signals substantial development and innovation of next generation treatment therapies.  Read more about the combination here.

Spark Therapeutics Reports Promising Gene Therapy Results

This essay was featured in the BloodStream Pod.

   After decades of study, gene therapy is showing exciting possibility for the treatment of hemophilia B patients. Researchers at Spark Therapeutics have reported promising results in a study of four hemophilia B patients who were injected with viruses containing the gene that codes for factor IX. Factor IX protein which clots blood is otherwise absent in the bodies of hemophilia B patients. While the small study size is keeping scientists from declaring an overall cure, the consistently positive results observed in each of these four patients is being received by scientists as quite encouraging for future one-time treatment possibilities for hemophilia B. In addition to the optimism regarding a potential cure for segments of the hemophilia population, these findings also have encouraging implications for the treatment of several other rare diseases.

 

Precision BioLogic Donates Coagulation Diagnostic Products Through the WFH Humanitarian Aid Program

This essay was featured in the BloodStream Pod.

   Last week Precision BioLogic announced its donation of coagulation diagnostic products through the World Federation of Hemophilia (WFH) Humanitarian Aid Program. The donation delivered products to the University Teaching Hospital in Yanoudé, Cameroon to aid in the diagnosis and treatment of individuals with bleeding disorders. Executive Vice President of Precision BioLogic, Paul Empey says, “We’re excited to make this donation and directly support WFH’s efforts to close the gap in care between those with bleeding disorders who receive proper diagnosis and treatment and those who do not.” The WFH Humanitarian Aid Program seeks to ensure treatment for all patients with bleeding disorders despite current limitations in access to care in developing countries throughout the world. Learn more about the WFH Humanitarian Aid Program here.

 

Let's Talk Period

This essay was featured in the BloodStream Pod.

   Queens University researcher, Dr. Paula James, has developed a self-administered bleeding assessment tool (self-BAT) to aid women in determining whether they may have a bleeding disorder. Bleeding disorders in women frequently go undiagnosed as individuals are often unfamiliar with the symptoms and/or mistake them for normal. The website LetsTalkPeriod.ca which houses self-BAT, “aims to increase awareness of the signs and symptoms of bleeding disorders.” Through increased understanding of what constitutes abnormal bleeding, women who suffer from bleeding disorders can better access treatment to limit unnecessary health complications.