Active Grants and Sponsored Research

Sponsor Grant ID: 5U01HL134812-04 Project Date: June 1, 2018 - May 31, 2023
Sponsoring Organization: NIH/NHLBI

Stem cells for therapeutics discovery in genetic blood disorders

Description of Major Goals
This multi-PI, multi-center component of the Progenitor Cell Translation Consortium exploits animal models of genetic blood disorders (zebrafish, murine, and human iPS cells) to screen and validate genes and small molecules as novel therapeutics and moves them into clinical trials. Co-PIs include Leonard Zon and Akiko Shimamura (BCH) and Adrianna Vlachos and Jeffrey Lipton (Hofstra-Northwell).
Sponsor Grant ID: 2P01HL048546-21A1 Project Date: September 1, 2016 - May 31, 2021
Sponsoring Organization: Oregon Health and Science University/NHLBI

Preclinical Studies of Marrow Failure and Clonal Evolution in Fanconi Anemia: Project 3

Description of Major Goals
Evaluate the effects of small molecule compounds, metformin and TGF-beta receptor kinase inhibitors that improve proliferation, hematopoiesis, and cell survival in Fanconia anemia models.
Sponsor Grant ID: 76777 Project Date: April 1, 2017 - February 28, 2021
Sponsoring Organization: Fanconi Anemia Fund, Inc.

Pilot Study of Metformin for Patients with Fanconi Anemia

Description of Major Goals
This pilot study explores whether Metformin is safe and efficacious in improving hematopoiesis in patients with Fanconi Anemia. We focus on pediatric and young adult patients with FA to determine safety, tolerability, and preliminary efficacy of Metformin in Fanconi Anemia patients. This study also integrates biological correlative studies to glean mechanistic insights which may inform future studies.
Sponsor Grant ID: 85147 Project Date: April 13, 2017 - April 12, 2020
Sponsoring Organization: V Foundation

Cell-Free Analysis of p53 and the Apoptosis Cascade

Description of Major Goals
This represents the remainder of the V Foundation grant received during in 1997. These are now unobligated funds. Investigate the regulation of apoptosis using a cell-free system. I designed the study and experiments, analyzed the data, and wrote the papers.
Sponsor Grant ID: R34 HL133384-01 Project Date: April 10, 2017 - March 31, 2020
Sponsoring Organization: NHLBI

Feasibility study randomizing IST vs URD BMT for children with aplastic anemia

Description of Major Goals
David Williams, PI
We propose a pilot study to determine the feasibility of randomizing children with acquired
aplastic anemia for either immunosuppression therapy (current standard of care) with highly
matched unrelated donor hematopoietic stem cell transplant.
Sponsor Grant ID: 5R01DK103854-02 Project Date: December 01, 2015 - November 30, 2019
Sponsoring Organization: FHCRC/National Institute of Diabetes and Digestive and Kidney Diseases

U2AF1 mutations in myelodysplastic syndromes: from mechanism to therapy

Description of Major Goals
The goal of this study is to determine the mechanistic and functional consequences of U2AF1 spliceosomal mutations in order to identify new therapeutic strategies. The specific aims are: 1) Determine the mechanistic basis and consequences of the observed genetic spectrum of U2AF1 mutations; 2) Determine how U2AF1 mutations dysregulate downstream molecular pathways, contributing to molecular features of dysplastic cells. 3) Identify potential therapeutic opportunities for targeting U2AF1-mutant cells.
Sponsor Grant ID: 5R24DK099808-05 Project Date: September 1, 2015 - August 31, 2019
Sponsoring Organization: FHCRC/National Institute of Diabetes and Digestive and Kidney Diseases

Translational Studies of Inherited Marrow Failure and Myelodysplastic Syndromes

Description of Major Goals
This is a resource development and discovery grant investigating inherited marrow failure and familial myelodysplastic syndromes with a focus on gene discovery.