Award Will Support Novel Hemophilia Therapy Investigation

The Children’s Hospital of Philadelphia’s Lacramioara Ivanciu, PhD, was one of five investigators who recently received funding through the Bayer Hemophilia Awards Program (BHAP). A “unique initiative dedicated to supporting innovative research and educational initiatives that benefit people with hemophilia,” the BHAP is administered by Bayer Healthcare, a subsidiary of the German pharmaceutical giant Bayer AG, and awards grants to hemophilia investigators and professionals.

An investigator in the Children’s Hospital’s Division of Hematology, Dr. Ivanciu’s research is focused on the design of novel bypass agents for the treatment of hemophilia. An inherited bleeding disorder, hemophilia is a lifelong disease that can require chronic management According to the CDC, each year in the U.S. approximately 400 babies with hemophilia are born.

Dr. Ivanciu’s research deals with the blood coagulation response, and in particular the coagulation factor IX (FIX). A key part of the coagulation system, FIX deficiency results in Hemophilia B, which is most often treated by replacement FIX therapy. However, FIX replacement therapy requires multiple injections and high doses, as FIX has a short half-life.

“My research focus on bioengineering coagulation factor IX as a potential alternative strategy for hemophilia B therapy,” said Dr. Ivanciu. “The novel FIX variants are expected to have prolonged half-life and efficacy and thus, be more efficient at lower doses. This could greatly benefit the patients with hemophilia by reducing the therapeutic dose, an important goal in the replacement therapy,” said Dr. Ivanciu.

In addition, Dr. Ivanciu recently co-authored a paper published in Blood examining recombinant activated human Factor VII, and was the first author another paper also published in Blood that focused on two other coagulation factors, factor Xa and factor Va.

Saying she was “very pleased” to receive the BHAP Early Careeer Investigator award, Dr. Ivanciu noted that its support will allow her “to advance the understanding of bleeding disorders by developing and applying new systems models and therapeutics to these problems.”

“I am in a unique position to make meaningful contribution to the field and possibly advance new therapies for the treatment of hemophilia,” she added.

“BHAP, now in its twelfth year, continues to attract high-quality applicants who are committed to answering important scientific and medical questions. Through our support of their research, this year's winners will contribute to a better understanding of hemophilia and bleeding disorders and ways to improve treatment and optimize patient outcomes,” said Bayer Healthcare’s Prasad Mathew, MD.

For more information hematology research and care at The Children’s Hospital of Philadelphia, see the Division of Hematology’s website.