Date Funded 12/18:

The DBAF and DBA Canada proudly increased DBA awareness among attendees at the 60th American Society of Hematology (ASH) Annual Meeting and Exposition held in San Diego. The event was attended by over 20,000 worldwide hematologists, researchers and other health-related professionals. Visitors to the DBA booths received information regarding DBA clinical care, patient opportunities, and research.

Date Funded 12/18:

The DBAF has awarded $54,754 with support from DBAC and from Friends of DBA to Dr. Scott C. Blanchard, PhD, from Weill Cornell Medicine for the project entitled “Identifying associations between DBA and genomic variation in ribosomal DNA”. The overall aims of the proposal are to sequence the ribosomal DNA (rDNA) repeat to assess whether sequence variation within this repeat contributes to the clinical features of DBA. This is a novel approach to understanding clinical variability among DBA patients.

Date Funded 3/18:

The 15th Diamond Blackfan Anemia International Consensus Conference was held March 10-12 at the Ritz-Carlton Hotel in downtown Atlanta, Georgia. The overriding goal of this and past ICC meetings has been to identify therapies and strategies to improve the quality of life for patients and families with DBA.

Date Funded 2/18:

Johan Flygare, M.D., Ph.D. of Lund University in Sweden was awarded $75,000, with support from DBA Canada and Friends of DBA, for his project entitled, “Targeted corticosteroid therapy for DBA.” The goal of the proposal is to enhance the delivery of steroids directly to erythroid progenitors in DBA to reduce the overall toxicity of steroids, which often limits their effectiveness as a treatment for DBA. The approach Dr. Flygare will use is to attach steroids to an antibody that specifically recognizes a protein expressed on erythroid progenitors. After binding to the erythroid progenitors, the antibody gets internalized, delivering the steroids specifically to only those cells that are recognized by the antibody. Using this approach, one could presumably lower the amounts of steroids used to get a beneficial effect, and since the delivery is specific, it would reduce the amounts of steroids hitting other tissues causing side effects.

Date Funded 1/18:

The DBAF awarded a grant for $15,000 to Dr. Steve Ellis, Ph.D. at the University of Louisville to continue to provide experimental services to clinicians and scientists worldwide in need of his laboratory’s expertise in studying different aspects of ribosome synthesis relevant to DBA. Not only is Steve the dedicated (and unpaid) DBAF Research Director, his laboratory provides valuable research support services to the wider DBA Community.

Date Funded 1/18:

Janis Abkowitz. M.D., from the University of Washington, Seattle, WA was awarded $63,120 to continue her project entitled, “The contribution of heme excess to macrocytic anemia in murine models of DBA.” They propose studying the relationships of heme synthesis, globin synthesis, ROS, P53 expression/activity, and ferroptosis (a recently described iron dependent, non-apoptotic death mechanism) in a new and more promising murine model of DBA, Rpl11 haploinsufficient mice, and in Rps19 deficient mice.