Many individuals suffer from difficult-to-treat, life-threatening diseases, including specific types of cancers, neurological disorders, and pediatric and rare diseases. For these individuals, clinical trials are an important strategy to finding effective new and repurposed therapies. Unfortunately, around 85 percent of late-stage clinical trials of investigational drugs fail because of safety problems or ineffectiveness, despite promising preclinical test results in conventional models, such as 2D cell culture systems, and animal models, such as mice. Moreover, certain diseases, such as rare disorders or pediatric conditions, are not adequately represented in clinical trials or do not have the resources to conduct them.

To improve the rate of success of new therapeutics in drug development, NCATS has awarded 10 inaugural grants to support researchers’ efforts in creating microphysiological, bioengineered models of human tissues and organ systems to inform clinical trial design for both common and rare diseases. In addition to helping inform clinical trial design, these projects also will support the planning and execution of clinical trials, assist in patient stratification, help identify reliable clinical trial endpoints, and ultimately develop tools for more informative and efficient clinical trials for both common and rare diseases.

The awards are administered through a new program, “Clinical Trials” on a Chip, which is led by NCATS, in conjunction with several other NIH Institutes and Centers.

These awards were made in response to RFA-TR-19-014.

View the project details below:

Boston Children’s Hospital

Tissue Chips for Precision Treatment of Catecholaminergic Polymorphic Ventricular Tachycardia

Principal Investigators: William Pu, M.D. (Harvard Medical School), and Kevin Kit Parker, Ph.D. (Harvard University) 
Grant Number: 1-UG3-TR-003279-01

*Funded by NCATS with additional funding from the Eunice Kennedy Shriver National Institute of Child Health and Human Development.

Brigham and Women’s Hospital

“Clinical Trials” on a Premature Vascular Aging-on-a-Chip Model

Principal Investigator: Yu-Shrike Zhang, Ph.D. (Harvard Medical School) 
Grant Number: 1-UG3-TR-003274-01

*Funded by NCATS.

Cedars-Sinai

A Microphysiological Multicellular Organ-on-Chip to Inform Clinical Trials in FTD/ALS

Principal Investigator: Clive Niels Svendsen, Ph.D. 
Grant Number: 1-UG3-TR-003264-01

*Funded by NCATS.

Columbia University

Clinical Trials in a Dish Using a Personalized Multi-Tissue Platform for Atopic Dermatitis*

Principal Investigator: Angela M. Christiano, Ph.D. 
Grant Number: 1-UG3-AR-079297-01

*Funded by NCATS, with additional funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

Johns Hopkins University

Engineering Clinical Trials on a Chip for Dystrophin-Deficient Muscular Dystrophy*

Principal Investigators: Deok-Ho Kim, Ph.D., and David Alan Kass, M.D. 
Grant Number: 1-UG3-TR-003271-01

*Funded by NCATS with additional funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

Texas A&M Engineering Experiment Station

Developing Extracellular Vesicle–Based Therapeutics Against Preterm Birth Through the Use of Maternal-Fetal Interface on a Chip*

Principal Investigators: Arum Han, Ph.D. (Texas A&M University), and Ramkumar Menon, Ph.D. (The University of Texas) 
Grant Number: 1-UG3-TR-003283-01

*Funded by NCATS with additional funding from the Eunice Kennedy Shriver National Institute of Child Health and Human Development.

University of Pittsburgh

A Vascularized, Patient-Derived iPSC Liver Acinus Microphysiological System as an Innovative Precision Medicine Platform for Optimizing Clinical Trial Design for Nonalcoholic Fatty Liver Disease

Principal Investigators: D. Lansing Taylor, Ph.D., Jaideep Behari, M.D., Ph.D., and Alejandro Soto-Gutierrez, M.D., Ph.D. 
Grant Number: I-UG3-TR-003289-01

*Funded by NCATS.

University of Rochester

A Microphysiological System of Tendon Inflammation and Fibrosis for Drug Screening and Efficacy Testing*

Principal Investigators: Hani A. Awad, Ph.D., James L. McGrath, Ph.D., and Benjamin L. Miller, Ph.D. 
Grant Number: 1-UG3-TR-003281-01

*Funded by NCATS with additional funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

University of Washington

Safety and Efficacy of Human Clinical Trials Using Kidney-on-a-Chip Microphysiological Systems

Principal Investigators: Jonathan Himmelfarb, M.D. (University of Washington), and Matthias Kretzler, M.D. (University of Michigan) 
Grant Number:1-UG3-TR-003288-01

*Funded by NCATS.

University of Wisconsin–Madison

Mechanisms of Microenvironment-Mediated Resistance to Cancer Cell–Surface Targeted Therapeutics*

Principal Investigators: David J. Beebe, Ph.D., and Joshua Michael Lang, M.D.  
Grant Number: 1-UG3-TR-003280-01

*Funded by the National Cancer Institute with additional funding from NCATS.