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Myelodysplastic Syndromes (MDS) Workshop

Myelodysplastic Syndromes (MDS) Workshop, May 13 - 14, 2024 banner with a histological image of MDS

This workshop was held on May 13-14, 2024 and brought together senior leaders/experts,  junior investigators, and NCI staff who concentrate their research on Myelodysplastic Syndromes (MDS).

There are more than 10,000 new cases of MDS diagnosed in the U.S. every year. These blood cancers remain difficult to treat due to vast differences in the patient population including great genetic diversity, complicated clinical presentation with similarities to several overlap syndromes, and lack of highly effective and long-lasting treatments.

Presenters evaluated the current state of basic, preclinical and clinical sciences in this area, as well as identified key challenges and opportunities to foster MDS research and care.

Documents from the Workshop

Recordings from the Workshop

Introduction to MDS: Gaps and Needs

  • Welcoming remarks - Drs. Gosia Klauzinska, Dan Gallahan, and Peter Aplan (NCI)
  • Overview of MDS Advances, Gaps, and Needs - Dr. Guillermo Garcia-Manero (MD Anderson)

Session 1: Clinical Needs – Personalized Treatment Approaches for MDS/Studies Matching New Diagnostic Approaches with Improved Treatments

  • TP53 mutated MDS, will we make progress? - Dr. Amer Zeidan (Yale Cancer Center)
  • The epichaperome as a biomarker and therapeutic target - Dr. Monica Guzman  (Weill Cornell Medicine)
  • Mitochondrial heteroplasmy in the development of MDS - Dr. Lukasz Gondek (Johns Hopkins University)
  • Targeting unique metabolic vulnerabilities in MDS stem cells: Bench to bedside and back - Dr. Brett Stevens (University of Colorado Anschutz Medical Campus)
  • Predicting leukemia evolution and response to treatment by integrating transition state theory, machine learning, and the peripheral blood transcriptome - Dr. Russell Rockne (City of Hope)

Session 2: Genetic Predisposition to MDS

  • Germline predisposition to MDS in adults - Dr. Lucy Godley (Northwestern University)
  • Germline and somatic drivers of primary pediatric MDS - Dr. Marcin Wlodarski (St. Jude Children's Research Hospital)
  • Using large exome sequencing cohorts to evaluate germline MDS predisposition genes - Dr. Lisa McReynolds (NCI)
  • Clonal Hematopoiesis and CCUS: Mapping progression risk - Dr. Pinkal Desai (Weill Cornell Medicine)
  • DDX41 mutations: Splicing anomalies, cellular stress and MDS - Dr. Tim Chlon (Cincinnati Children's Hospital)

Session 3: Analysis of the Progression of CHIP to MDS to AML and Strategies to Intervene or Prevent Transformation

  • Inflammatory signaling in MDS - Dr. Daniel Starczynowski (Cincinnati Children's Hospital)
  • Pre-leukemic HSCs: Therapeutic targeting and clonal expansion - Dr. Ravi Majeti (Stanford University)
  • Building bone marrow - Dr. Brendan Harley (University of Illinois Urbana - Champaign)
  • Mechanisms by which the aging microenvironment drives progression of CH to MDS - Dr. Kira Young (Jackson Laboratory)
  • Biology and therapeutic targeting of cohesin-mutant myeloid malignancies - Dr. Zuzana Tothova (Dana-Farber Cancer Institute)

Session 4: Development of preclinical models for the discovery and development of new MDS therapies

  • Genetically Engineered Mouse (GEM) models for MDS - Dr. Peter Aplan (NCI)
  • Modeling niche-clone interactions in vivo: challenges and opportunities - Dr. Laura Calvi (University of Rochester School of Medicine and Dentistry)
  • Insights into MDS from human iPSCs - Dr. Eirini Papapetrou (Icahn School of Medicine at Mount Sinai)
  • RNA splicing factor mutations in MDS: Do RNA granule perturbations drive clonal advantage? - Dr. Stephanie Halen (Yale School of Medicine)
  • Altered RNA export sensitizes to nuclear export inhibition in SF3B1 mutant myelodysplastic syndromes - Dr. Justin Taylor (University of Miami Miller School of Medicine)

Summary and Final Discussion

  • Workshop summary - Dr. Peter Aplan (NCI)
  • Final discussion - Workshop Participants
  • Closing remarks - Dr. Gosia Klauzinska (NCI)

Social media posts related to the workshop can be found using #Research4MDS.

DCB Contact for the Workshop

For additional information about the MDS Workshop, please contact Dr. Margaret (Gosia) Klauzinska.

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