The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance, using Bone Marrow Failure as a Model
Annual rept. 28 Sep 2010-27 Sep 2011
FEINSTEIN INST FOR MEDICAL RESEARCH MANHASSET NY
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Diamond Blackfan anemia DBA is a rare inherited red cell aplasia. Mutations have been described in ribosomal protein genes. Currently standard therapy includes corticosteroids, red cell transfusions or stem cell transplantation however all are fraught with many side effects. Leucine is one of the branched chain amino acids and has been shown to upregulate protein translation. This is a pilot study to test the feasibility of administering leucine to 50 patients with DBA, monitoring for clinical hematologic response and side effects. The study has not yet opened due to some delays. It has also required multiple revisions in the site-specific and master protocols. In addition the manufacturing company has a shortage of Leucine due to a factory issue since May 2011. At present we have procured adequate product and it is being set into capsular form. Since the protocol has not yet opened there is no scientific progress for this study at this time.
- Anatomy and Physiology
- Medicine and Medical Research
- Organic Chemistry