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Therapeutic RSK1 Targeting in Myeloid Malignancies

Project Term
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Headshot - Oh_Stephen SQ.jpg

Stephen Oh, MD, PhD

Washington University in St. Louis

St. Louis, MO
United States


Project Summary

The objective of this study is to evaluate RSK1 as a therapeutic target in myeloid malignancies. We will utilize PMD-026, a first-in-class, orally administered, small molecule inhibitor of RSK kinases to treat patients with myelofibrosis and other advanced myeloid neoplasms.

Lay Abstract

This study investigates PMD-026, a first-in-class, orally administered, small molecule inhibitor of RSK kinases to treat patients with myelofibrosis and other advanced myeloid neoplasms.

The clinical trial is a Phase I dose escalation study where participants will receive different doses of the study drug to determine safety and tolerability in this patient population.

Additional evaluations to assess response to the treatment will include volumetric imaging to evaluate response as well as symptom response via standardized symptom assessment forms. Laboratory assessments will include evaluation of blood counts and evaluation of liver and kidney function.

Samples for correlative analyses will also be obtained, to understand how the study treatment is working on various pathways and cell types in the blood and bone marrow.

The overall objective of this study is to evaluate this novel treatment approach to improve outcomes for patients with these diseases.


Program

Academic Clinical Trials Program (ACT)

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