Grant Award Details

Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)
Grant Number: 
DISC2-13428
Investigator: 
Type: 
PI
Disease Focus: 
Blood Disorders
Human Stem Cell Use: 
Adult Stem Cell
Award Value: 
$1,244,160
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)
Public Abstract: 

Research Objective

This proposal will deliver a small molecule therapeutic candidate for the treatment of Myelodysplastic Syndromes and will act by inducing differentiation on mutated hematopoietic stem cells.

Impact

This application will enable development of a therapeutic candidate for the treatment of Myelodysplastic Syndromes, a preneoplastic hematological condition of HSCs.

Major Proposed Activities

  • Determine the MDS subtypes and mutational signatures responsive to differentiation induced by the lead series (Q1-Q2, YEAR 1).
  • Establish in vivo proof of concept differentiation with oral compound administration (Q1-Q2, YEAR 1).
  • Establish an ideal preclinical candidate with requisite pharmacology, safety, and efficacy (Q1-Q8, YEARS 1-2).
  • Further define the mechanism by which the lead series promotes differentiation in MDS (Q1-Q6; YEAR 1-2).
Statement of Benefit to California: 

This work will generate a novel therapeutic candidate for the treatment of Myelodysplastic Syndromes (MDS), a life threatening hematological condition that is rapidly becoming more common as our population ages. In contrast to bone marrow transplants, which are at present the only curative treatment for MDS, this application will deliver a more equitable small molecule therapy with greater access for underserved communities, for whom the risk factors for developing MDS are more common.