Saturday, December 17, 2011

Hemophilia Funding & Research

HRSA is looking for applicants to develop the “National Hemophilia Program Coordinating Center” (NHPCC). The Center’s goal is to provide for an integrated and collaborative Regional Hemophilia Network (RTN) to help individuals with hemophilia and related bleeding disorders or clotting disorders such as thrombophilia.

The NHPCC will evaluate the activities of the RHN grantees, provide technical assistance to RHNs on standards, examine accessibility to care, resources available, and make information on genetics and public health as it relates to hemophilia available. Advances in genetic medicine and services will be included since they directly influence the health of individuals with hemophilia or other bleeding or clotting disorders.

About $800,000 is expected to be made available for FY 2012-2014. An annual cooperative agreement will fund one awardee with $300,000 specifically for projects to be undertaken with RHN grantees. Eligible applicants can include public and nonprofit entities, including faith-based and community-based organizations, Tribes, and tribal organizations

Applications for announcement (HRSA-12-135) to develop the NHPCC are due December 30, 2011. For more information, email Kathryn McLaughlin at or call (301) 443-6829. To view the announcement, released November 16th go to

In addition, NIH funding has been provided for gene therapy research to help patients with Hemophilia B. Hemophilia B is less common than hemophilia A with 1 in 5 hemophilia patients having hemophilia B while the other four have hemophilia A.

The problem is that Hemophilia B patients are unable to produce enough human clotting Factor IX also referred to as FIX, which is essential for normal blood clotting. Liver cells are the only cells in the body capable of producing a form of FIX that is active in the clotting process.

Researchers working on an experimental gene therapy technique have boosted the production of a vital blood clotting factor in six people with hemophilia B. Today, researchers are joining the FIX gene to a virus that targets liver cells.

Research teams from the University College London Cancer Institute and St. Jude Children’s Research Hospital in Memphis Tennessee have examined the effects of delivering the FIX gene to patients. Scientists inserted the FIX gene into a virus which served as the delivery vehicle for the gene therapy.

“Results from this study represent a promising step toward making gene therapy a viable treatment option for hemophilia B”, said Susan B. Shurin, M.D., Acting Director of the National, Heart, Lung, and Blood Institute (NHLBI) within NIH.

The research was partly funded by NHLBI, Howard Hughes Medical Institute, the ASSISI Foundation of Memphis, and the American Lebanese Syrian Associated Charities. Organizations in the United Kingdom such as the National Health Service, National Institute for Health Research, Department of Health, and the Royal Free Hospital are also providing funding for the research.

The research results are online in the New England Journal of Medicine and will appear in the December 22nd print edition.