In spring and fall 2019, NCATS issued two Therapeutic/Indication Pairing Projects awards. The funded preclinical projects are serving as “use cases” to demonstrate the utility of an independent crowdsourcing effort or a computational algorithm to predict new therapeutic uses of an existing drug or biologic.
- Network-Based Novel Therapeutics in Colorectal Cancers
- Preclinical Characterization of Saracatinib for Cystic Fibrosis Therapy
University of California, San Diego
Principal Investigators: Pradipta Ghosh, M.D., Soumita Das, Ph.D.
Grant Number: 1-UG3TR002938-01
The past 5 decades of treating cancer patients has resoundingly concluded that conventional approaches are not enough to cure most cancers because these approaches fail to ‘kill’ stem cells within tumors; these are the cells that escape by evolving and give rise to metastases and treatment-resistant disease. Differentiation therapy, which is aimed at re-activating differentiation programs in those stem cells has been shown to have ‘curative’ power in a certain type of blood cancer (leukemia), but such therapies are yet to emerge in cancers and remains an elusive Holy Grail and a grand challenge of our times. To tackle this challenge, this proposal seeks to repurpose one drug and validate one of the first differentiation therapies in cancers, and do so by using novel mathematical algorithms and a transdisciplinary approach that begins and ends at the patient’s bedside.
Cincinnati Children’s Hospital Medical Center
Principal Investigators: Anil Jegga, D.V.M., M.Res., John Paul Clancy, M.D., Anjaparavanda P. Naren, Ph.D.
Grant Number: 5-UG3TR002612-02
The pursuit of additional therapeutics for cystic fibrosis (CF) is needed due to several limitations of currently FDA-approved therapies. Our data suggest that src kinase inhibitors have a significant therapeutic benefit in CF primarily by impacting multiple CF pathophysiological processes. Saracatinib or AZD0530, a src kinase inhibitor from AstraZeneca, is well tolerated in humans on chronic administration. We therefore propose to test this asset as a novel CF disease modifying therapy.