Perspectives on drug development in chronic myelomonocytic leukemia: changing the paradigm
Drug development for chronic myelomonocytic leukemia (CMML) has failed to parallel the recent success observed in related myeloid neoplasms. To address these shortcomings, the US Food and Drug Administration (FDA) held a "Mini-symposium on CMML: Current State of the Art and Trial Design" in September 2023. This symposium brought together a panel of key FDA regulators and academic experts in CMML drug development to discuss challenges and provide perspectives on future drug development for this disease. The panel explored unique challenges that underlie the lack of therapeutic advances in CMML to date and discussed relevant topics such as clinical trial design, study end points, and key regulatory considerations. This article summarizes the key points of discussion from this symposium to facilitate advancements in the field.
Authors
- Raphaël Itzykson (ORCID: https://orcid.org/0000-0003-2139-6262)
- Yanxia Li (ORCID: https://orcid.org/0000-0001-7889-6124)
- R. Angelo de Claro (ORCID: https://orcid.org/0000-0001-8362-1977)
- Anthony Michael Hunter (ORCID: https://orcid.org/0000-0002-9819-4151)
- Ruben A. Mesa (ORCID: https://orcid.org/0000-0001-5880-7972)
- Eric Padron (ORCID: https://orcid.org/0000-0002-4707-7916)
- Kelly J. Norsworthy (ORCID: https://orcid.org/0000-0002-8462-9994)
- Marc Robert Theoret (ORCID: https://orcid.org/0000-0003-4973-5297)
- Mrinal M. Patnaik (ORCID: https://orcid.org/0000-0001-6998-662X)
- Chatchada Karanes
- Elizabeth Pulte
Institutions
- Atrium Health Wake Forest Baptist (US)
- United States Food and Drug Administration (US)
- Mayo Clinic (US)
- Center for Drug Evaluation and Research (US)
- Emory University (US)
- Inserm (FR)
- Université Paris Cité (FR)
- WinnMed (US)
- Moffitt Cancer Center (US)
- Assistance Publique – Hôpitaux de Paris (FR)
- Hôpital Saint-Louis (FR)
- Wake Forest Baptist Comprehensive Cancer Center
Publication Details
- Journal
- Blood
- Published
- 2024-11-07
- DOI
- https://doi.org/10.1182/blood.2024025648
- Citations
- 9
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 4.09