Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)
Grant Award Details
Grant Type:
Grant Number:
DISC2-13428
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$1,244,160
Status:
Closed
Progress Reports
Reporting Period:
Year 2
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
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.
