2013 NHF/Novo Nordisk Career Development Award
Annette von Drygalski, MD, PharmD
Assistant Clinical Professor of Medicine, University of California at San Diego
“Anti-fibrinolytic Strategies to Decrease Bleeding in Hemophilic Arthropathy”
Dr. von Drygalski’s CDA project is “Anti-fibrinolytic Strategies to Decrease Bleeding in Hemophilic Arthropathy”. Her research focuses on better understanding the mechanisms operating the anti-fibrinolytic system and how this process works in patients with hemophilia and specifically with joint bleeding. Her mentor will be John H. Griffin, PhD, distinguished researcher at UCSD.
Accelerated fibrinolysis and clot instability are becoming increasingly recognized as contributing factors to bleeding in hemophilia. One important molecule that prevents fibrinolysis is called TAFI (Thrombin Activatable Fibrinolysis Inhibitor). TAFI has low activity in hemophilia since it requires activation by thrombin which is not sufficiently generated in hemophilia. Interestingly, active TAFI not only prevents clot resolution, it has been recently discovered to have anti-inflammatory properties in rheumatoid arthritis and osteoarthritis. Dr. von Drygalski will be studying the dual anti-fibrinolytic and anti-inflammatory functions of TAFI in hemophilia arthropathy and develop a therapeutic TAFI-based approach to improving the efficacy of FVIIa based bypassing strategies for acute bleeding and joint protection. Her studies will be utilizing specifically engineered long-acting TAFI molecules in the plasma of patients with hemophilia as well as in vivo using mouse models.
Von Drygalski received a MD from the Universities of Erlangen/Nurnberg and Munich in 1995 and her PharmD from the University of Munich in 1988. She joined the faculty in July 2011 as Assistant Clinical Professor at UCSD and Director of the Adult Hemophilia and Thrombosis Treatment Center (HTC) in the Division of Hematology/Oncology, Department of Medicine. She also has an appointment as Adjunct Assistant Professor at the Scripps Research Institute (TSRI). Dr. von Drygalski has published several first author publications in peer-reviewed journals and was a recipient of the Bayer Early Career Investigator Award in 2012. Her long-term career goal is to continue building her career as a physician-scientist by developing a strong basic and translational research program for hemophilia while continuing to treat adult patients with hemophilia and other bleeding disorders.
Through the CDA, von Drygalski will receive $70,000 per year for up to three years. This award was selected through a process of peer review conducted by NHF’s Research Review Committee. This volunteer committee is made up of highly experienced and respected physicians and researchers working in the field of hematology. NHF wishes to thank them as well as Novo Nordisk, Inc. for their very generous support of this research award.
The funding for this award is made possible thanks to a generous grant from Novo Nordisk to NHF’s Research Catalyst Fund.
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Letter of Intent Deadline: January 11, 2013
Application Deadline: March 22, 2013
Award Start Date: July 1, 2013
The National Hemophilia Foundation (NHF), a nonprofit organization founded in 1948, is dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy, and research. NHF seeks research grant applications from established investigators, preferably at the assistant professor level or above, who have demonstrated a commitment to bleeding disorders research. We especially encourage innovative projects that promote the development of novel technologies and/or therapies to advance the field of bleeding disorders research. The funding for this award has been made possible by a generous grant from Novo Nordisk, Inc.
NHF/Novo Nordisk Career Development Award (CDA) candidates must hold an MD, PhD, or equivalent degree, with no more than six years of postdoctoral years of experience or greater than six years since completing their medical training. United States citizenship is not required, but applicants must be affiliated with or be faculty members of domestic organizations such as universities, colleges, hospitals or laboratories. Only non-commercial institutions and investigators associated with a non-commercial institution are eligible.
The CDA award recipient will be expected to spend at least 90% of their working time on the funded research project. The CDA recipient and his/her mentor are expected to remain at their institution for the duration of the project. Letters of support from appropriate institutional officials (department chair and financial grants officer) indicating the institution’s commitment to allow the candidate to focus his/her efforts on the stated research project will be required for the application.
Scope of Research
NHF is interested in funding innovative research studies to be carried out at the sub cellular, cellular, animal or human (patient) levels.
Studies to evaluate the genetic and immunologic bases for inhibitor development may lead to strategies to circumvent this potential complication of gene transfer. This research could provide the opportunity to determine the prognostic indicators that place patients at risk of inhibitor development. Inhibitor research may also lead to the development of synthetic replacement factor and gene therapy products with decreased immunogenicity. This includes research on genotype/phenotype relationships and susceptibility to inhibitors.
Better functional assays for clotting factor proteins are also needed. Reproducible, low-volume measure of factor VIII and factor IX activity in different animal models would greatly facilitate validation of in vivo gene therapy studies as well as innovative alternative treatment strategies. Structural studies of clotting factor proteins are needed in order to design more efficacious, longer-lived clotting factor proteins. Development of both small, biologically active, orally delivered molecules and improved protein delivery systems represent strategies that could lead to advances in our understanding of bleeding disorders.
While gene therapy has been an approach which has shown much promise, no one method has been demonstrated to be superior. Multiple experimental strategies may be undertaken to help find a “cure” for bleeding disorders. Research on the nature, propagation, and use of hematopoietic, liver, or other stem cells as an approach for gene transfer for hemophilia and other bleeding disorders may provide novel approaches for the treatment of these disorders.
All interested candidates need to submit a ‘Letter of Intent’ by no later than Friday, January 11, 2013. Letters of intent will be evaluated by members of NHF’s Research Review Committee who will collectively decide upon the candidates invited to submit a CDA application. If selected, candidates will have until March 22, 2013 to submit their full CDA application to NHF. All grant applications are subjected to a rigorous peer review process. Applications will be reviewed on scientific merit and scored in terms of significance, approach, innovation, investigator and environment.
LOI for the 2013 NHF/Novo Nordisk Career Development Award should be sent to:
Director, Research & Medical Information
National Hemophilia Foundation
116 West 32nd Street, 11th Floor
New York, NY 10001-3212
CDA letters of intent need to be on institutional letterhead and will not be considered for review unless signed by the applicant. Letters should be no more than 2 pages in length and include a brief introduction of the candidate-researcher, his/her mentor, institution and a description of the proposed research project. Letters must also include a CV (NIH-style biosketch) for both the candidate as well as the mentor.
All general questions regarding the Career Development Award program can be directed to Angelina Wang at email@example.com or by calling 800-424-2634, ext. 3727.