The Diamond Blackfan Anemia Foundation (DBAF)  was pleased to host the 13th DBA International Consensus Conference (ICC). We are proud and honored to continue this important conference founded and previously hosted by the Daniella Maria Arturi Foundation. The goals of the ICC are to facilitate international exchange and to encourage cooperation in research investigations for DBA. Click to view the meeting agenda.

Summary of the ICC provided by the DBAF's Research Director, Steven R. Ellis, Ph.D.

The 13^th Diamond Blackfan Anemia International Consensus Conference was held March 8^th through the 10^th at the Ritz-Carlton Hotel in downtown Atlanta, Georgia.  This year's conference saw a record number of abstracts accepted for presentation as either short talks or posters.  This increased number of abstracts  submitted relative to previous years in part reflects an increasing number of investigators entering the DBA field.  This, in turn, is a reflection of the interest in DBA generated on many fronts by the remarkable progress in the field over the past 10-15 years.

The desire to give as many investigators as possible the opportunity to present their work within a very short time frame made for a very jam-packed agenda.  Fortunately, various session moderators and facilitators kept a tight reign over their different sessions and the meeting progressed without a hitch.  While I may seem a bit obsessed with the meeting's logistical issues, efficiently packing all this content into a day and a half was a major factor the organizers had to contend with in setting up the meeting's agenda.  As a way of limiting redundancies in meeting presentations, Jeff Lipton (New York) started the meeting off by giving an overview of DBA from both a clinical and scientific perspective.

Dr. Lipton's talk emphasized the limitations of current therapies for DBA (transfusions, steroids, and bone marrow transplants) and the need for improved therapies with fewer complications.  This emphasis tied in nicely with Dawn Baumgardner's (Buffalo) opening remarks stating that an 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. 

Current Clinical Trials (New Mechanistic Insights)

In this light, it is worth mentioning the 3 different clinical trials (Leucine, Lenalidomide, and Sotatercept) currently underway in DBA patients.  One of the more interesting aspects of this year's meeting concerns the drug Sotatercept.  This drug was initially developed by Acceleron Pharma as a potential treatment for osteoporosis, but in clinical trials for this condition, it was found that individuals receiving the drug showed a significant increase in red blood cell production.  This increase could be considered an adverse side effect in individuals with osteoporosis, but as a consequence of this serendipitous finding, Sotatercept is now being tested as a treatment for different types of anemia, including DBA.  A talk by Jingping Ge (Philadelphia) provided a potential mechanistic rationale for why this drug may be efficacious in DBA.  Her work on induced pluripotent stem cells (IPSC) from DBA patients revealed that the same signaling pathway targeted by Sotatercept appears to be dysregulated in DBA patients.  This observation provides a more solid scientific basis for the Sotatercept trail (in terms of full disclosure, I have no financial ties to either Acceleron Pharma or Celgene which is supporting the trial).  The Sotatercept-DBA relationship was further reinforced by studies from Shuo Lin (Los Angeles) showing that the generic form of this drug rescues the DBA-like phenotype in zebrafish.  While the Sotatercept clinical trial is still in early stages, these results certainly generated guarded optimism for a positive outcome.

The New Drug Pipeline (Turning the Spigot On)

In addition to the drugs currently in clinical trials for DBA, one of the most remarkable features of this year's meeting was the number of drugs entering the pipeline as potential therapies for DBA. Talks by Hsiang-Ying Lee and Harvey Lodish (working together in Boston) described screens for drugs that