Johns Hopkins Researchers Awarded Dollar 15 Million to Develop Platform to Study Neurological Diseases and Screen Chemicals Using ‘New Approach Methodologies’

A team of Johns Hopkins researchers has been awarded a five-year $15 million grant from the National Institutes of Health to develop a platform to test potential new treatments for neurological diseases such as Alzheimer’s, and to screen for harmful chemicals.

The grant is through NIH’s Common Fund’s Complement Animal Research In Experimentation (Complement-ARIE) program which supports developing New Approach Methodologies (NAMs) that simulate human biology—complementing and, in some instances, replacing animal testing. This first round of funding will support a series of technology development centers—including one at Johns Hopkins—as well as a NAMs data hub and coordinating center. Total NIH funding for these initiatives is $150 million over five years, pending availability of funds.

The Drug Research Organoid Intelligence Development Platform (DROIDp) will use brain organoids—lab-grown neural tissues derived from human stem cells—advanced electrical sensors, and AI analytics to assess neural functions such as learning and memory in drug and chemical testing, and to develop integrated NAMs.

The Johns Hopkins team includes researchers with expertise in stem cell biology, neurodevelopmental disorders, bioengineering, sensors, machine learning and AI, data science, and neuroscience spanning the Johns Hopkins schools of public health, engineering, and medicine, as well as the Applied Physics Laboratory, the Berman Institute of Bioethics, and the Kennedy Krieger Institute.

The project leads are Lena Smirnova, PhD, assistant professor in the Department of Environmental Health and Engineering and program lead at the Center for Alternatives to Animal Testing at the Johns Hopkins Bloomberg School of Public Health, and Erik Johnson, PhD, senior research scientist at the Johns Hopkins Applied Physics Laboratory.

“The DROID platform is designed to help close one of the important gaps in in vitro neurobiology models: the ability to measure higher-order neural responses such as learning-related activity and memory in a human-relevant system,” says Smirnova. “Through the NIH Common Fund’s Complement-ARIE program, we have an opportunity to develop integrated NAMs into a practical framework that can reduce reliance on animal studies.”

The DROID platform will extend current in vitro approaches—test tubes and culture dishes—to modeling learning and memory using brain organoids, addressing a critical gap: Current in vitro assays cannot capture higher-order neural responses, and evaluations of neurotoxicity or drug efficacy still primarily rely on animal behavioral tests.

The researchers will also evaluate brain organoids derived from both healthy individuals and patients with Alzheimer’s disease and individuals with SYNGAP1-related disorders—a rare pediatric condition associated with intellectual disability, seizures, and autism—to test neural responses and sensitivity to pharmacological interventions.

By enabling researchers to assess complex neural responses that currently rely on animal behavioral tests, the DROIDp system aims to improve drug discovery and neurotoxicity testing. Ultimately, the goal of this platform is to provide a more predictive, human-relevant approach for studying neurological diseases and evaluating the safety of drugs and chemicals.