WHITE PAPER | A Platform for Screening Drugs to Treat Sickle Cell Disease and Other HemoglobinopathiesSCD wp-thumb


Here we describe the development of a serum-free assay platform where unaffected primary human bone marrow cells can be expanded in the presence of various cytokines in a two-step culture system. This serum-free assay platform offers a direct measurement of the HbF protein, using two methods in response to treatment with HU as shown, and additionally with other HbF inducers (data not shown). This differs from other approaches looking at increases in transcript numbers or other indirect measurements. This in vitro assay will facilitate the ability to examine new potential drug therapies, both alone and in combination with HU, or HbF induction. Although affected donor samples were not available in the current study, this assay may be used to assess HU responsiveness in some SCD patients and to identify other patient-specific drug options.