Expert Interview
A Third Look: Discussing Fulcrum's pociredir and other oral HbF inducers for sickle cell disease (SCD)
Ticker(s): FULCInstitution: University of Alabama
- Director of the UAB Adult Sickle Cell Clinic and associate professor in the Division of Hematology and Oncology
- Treats 800 SCD patients, 10 with beta-thal
- Clinical and research interests in sickle cell disease include improving access to care for affected individuals, and identifying and developing novel therapies for sickle cell disease, as well as enhancing horizontal care for patients with sickle cell disease.
Of the different mechanisms being investigated, which of them seems to have the most promise moving forward?
Added By:Can you provide a broad overview of fetal hemoglobin (HbF) inducers and pyruvate kinase activators in development for sickle cell disease (SCD), including their mechanisms and available clinical data?
Added By:What are your thoughts on the efficacy of Fulcrum’s pociredir, particularly regarding its HbF increases at different dosage levels, and what level of increase would be needed to reduce fatigue and vaso-occlusive crises (VOCs)?
Added By:What are the safety concerns associated with HbF inducers like pociredir, particularly regarding potential cancer risks and other adverse events such as neutropenia and GI toxicity?
Added By:How do you compare the potential of HbF inducers versus pyruvate kinase activators for treating SCD, and do you foresee one class being more successful than the other?
Added By:Regarding the kinetics of response, do you expect HbF induction to plateau within the 12-week treatment period, and could there be compensatory mechanisms that might reduce long-term efficacy?
Added By:Are You Interested In These Questions?
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