DBAF and DBAC Fund Continuing Drug Development Research

The Diamond Blackfan Anemia Foundation (DBAF) and Diamond Blackfan Anemia Canada (DBAC) are joining forces once again to fund Johan Flygare, M.D., Ph.D.’s project entitled, “A novel group of kinase inhibitors for treatment of Diamond Blackfan Anemia.” The goal of this $35,000 project is to develop safer and more potent drugs to treat DBA. Ideally, these drugs would act by correcting part of the underlying problem causing the anemia. In previously funded research, Dr. Flygare’s lab utilized cells from their DBA mouse to test more than 12,000 chemical substances and they identified a group of molecules that had a therapeutic effect on the formation of red blood cells. Additionally, the preliminary research identified 12 potential drug targets. Based on this solid preliminary data, Dr Flygare’s project plans to continue researching these compounds for further drug development and a completely novel strategy for treating Diamond Blackfan Anemia.

Dr. Flygare stated,

 To me it makes a very big difference if funding for a project is supported by tax payers or, like in this case, by people who worked hard to raise this money and choose to donate it specifically to our project. That you believe in us is an energy boost for the whole team! When I started my own lab in Lund, Sweden, my first goal was to start a project with the aim to identify a new candidate DBA drug. During the last three years, Kavitha Siva in my group has screened more than 12,000 chemical compounds in collaboration with two chemical biology groups in Sweden. We have now identified several new and promising compounds that rescue production of red blood cells in our RPS19-deficient mouse model for DBA. We are now at the stage where we are trying to figure out how these compounds work. The experiments required to identify the drug target and explain the therapeutic mechanism are expensive and I am truly grateful that the DBA Foundation and DBA Canada support these studies with this grant.

The DBAF and DBAC thank Dr. Flygare, Kavitha Siva and the entire team at Lund for their continued hard work and commitment to this very exciting project. A big thank you to all our supporters! Your donations and fund raisers are appreciated and make funding research possible.