A potential Diamond-Blackfan anemia treatment swims into view

December 5, 2019

Zebrafish, besides being popular in aquariums, make good stand-ins for studying human diseases. They share about 70 percent of their genes with humans, and can be studied at a mass scale, enabling scientists to test hundreds, even thousands of drugs at a time simply by adding the drug to their water.

One such test came up with a new potential treatment for a rare blood disorder, Diamond-Blackfan anemia (DBA): a drug called trifluoperazine, normally used to treat psychotic disorders. It’s now in a clinical trial for DBA in adults, and Leonard Zon, MD, hematology/oncology researcher and director of the Stem Cell Research program at Boston Children’s Hospital, counts it as the fourth drug his team has identified in zebrafish to reach patients.

Read more on Boston Children’s Discoveries page