The Diamond Blackfan Anemia Foundation (DBAF) is proud to announce the funding of another important research project. Through the dedication, hard work, and generosity of our families, friends, and supporters, the DBAF was able to award $21,281 to Dr. Harvey Lodish’s project entitled, “High-throughput screening identifies many novel potential therapies for Diamond Blackfan Anemia.” The esteemed Dr. Lodish provided the following summary of his lab’s very exciting work and the significance of the Diamond Blackfan Anemia Foundation’s gap funding.
High-throughput screening identifies many novel potential therapies for Diamond-Blackfan Anemia
Sherry Lee, Xiaofei Gao, Lingbo Zhang, Lina Prak, Shilpa Hattangadi, and Harvey Lodish
Whitehead Institute and Departments of Biology and Biological Engineering, Massachusetts Institute of Technology, Cambridge MA 02142
Many Diamond Blackfan Anemia (DBA) patients respond to treatment with the steroid prednisone with increases in red cells, but continuous use of this and other corticosteroids causes severe side effects. We need additional drugs to treat this disease, and one way of proceeding is to screen existing drugs and related chemicals for their ability to stimulate red cell production.
Supported by generous grants from the U S Department of Defense, we screened a collection of over 2000 tested and approved therapeutic compounds for those that can stimulate mouse red cell production in culture. We obtained over 40 potential “hits” and then rescreened all of them in a new human cell culture system we developed. Ten drugs approved for human use stimulated human red cell production in this system at a developmental stage that suggests they may be useful in treating DBA. We have been screening these drugs one-by-one for their ability to stimulate red cell production in mice that are “models” for DBA and other bone marrow failure disorders. Encouragingly, we have already shown that one such drug, in clinical use to treat certain lipid (fat) disorders, works in this system! As this and the other new molecules have minimal side effects, several of these compounds have the potential to treat the anemia associated with DBA.
Because of problems with the Defense Department budget, our support from them for this work has temporarily lapsed. Fortunately, we just received a generous grant from the Diamond Blackfan Anemia Foundation that is allowing us to test this drug and others in several mouse models of DBA; our goal is to complete these “preclinical” experiments as soon as possible and then work with clinicians to initiate human trials.
The Diamond Blackfan Anemia Foundation is grateful to Dr. Lodish and his entire lab for their hard work and dedication. Thank you to all who made this work possible.