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The Future of Neuroscience Drug Discovery
How NAMs are changing the game
On November 17, 2025, at the Society for Neuroscience (SfN) Product Theatre, a panel of experts tackled one of the most pressing questions in drug development: How can we move beyond animal testing and embrace human-relevant models? The discussion, titled “Translational Science: Leveraging New Approach Methodologies for Neuroscience Innovation,” brought together leaders from research foundations, technology innovators, and service providers to explore the rise of New Approach Methodologies (NAMs).
Moderated by Sam Chuang (Executive Director, Scientific Advisory Services, Charles River), the panel featured Nicole Polinski (Director of Research Programs, The Michael J. Fox Foundation for Parkinson's Research) and Florian Larramendy (Chief Technology Officer and Co-Founder, NETRI). Their insights revealed a future where drug discovery is faster, safer, and more predictive.
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What are NAMs and why do they matter?
NAMs are tools that improve the predictability of the efficacy and/or safety of a therapeutic or chemical substance in humans and reduce and/or replace animal testing. They include monoculture and mixed culture of human-derived cells for use in in vitro assays, 3D organoids, organ-on-a-chip systems, and AI-driven computational models.
These methods aim to reduce reliance on animal models, which can sometimes fall short in predicting human outcomes, while accelerating development and cost efficiency. For neuroscience, where diseases like Parkinson’s disease are complex and multifaceted, NAMs provide an opportunity to initiate research and preclinical drug development earlier in a human context.
Regulatory Momentum: FDA Leads the Way
In April 2025, the FDA announced a pilot program to phase out animal testing for monoclonal antibodies (mAbs) and other drugs. This shift follows the FDA Modernization Act 2.0 (2022), which opened the door for non-animal methods in IND applications. The goal? Enhance safety prediction, expedite timelines, and minimize costs. Regulatory agencies in Europe, Japan, and the UK have since announced similar roadmaps, creating a global ripple effect.
Panel Insights: Opportunities and Challenges
Panelists shared both optimism and realism about NAMs. Nicole Polinski, PhD acknowledged initial concerns about the FDA's announcement given the complexity of age-related neurodegenerative diseases such as Parkinson's, but mentioned that as long as the goal is not to completely replace in vivo model systems, there is clear alignment with MJFF's mission to find patient-focused research. She noted that NAMs are most effective today for early-stage efficacy and neurotoxicity screening, though complex diseases like Parkinson’s still require integrated approaches.
Florian Larramendy of NETRI highlighted the innovation potential, describing efforts to combine microfluidics with Axion MEA platforms to create neuron-based sensors. He cautioned that regulatory acceptance hinges on large validation datasets, which could take years, but pointed to evidence where NAMs predicted toxicity missed by animal tests.
Sam Chuang of Charles River added that adoption is already underway, with most clients starting or looking into incorporating NAMs into strategies. He stressed a stepwise approach, augmenting rather than replacing traditional methods, and predicted the greatest near-term impact in safety and toxicology testing, particularly for cardiovascular and liver models.
Audience Q&A: Can NAMs Really Replace Animal Testing?
One attendee asked the question on everyone’s mind: “What evidence do we have that NAMs can truly replace animal testing?”
Initially, Mike Clements (SVP Scientific Partnerships & Strategy, Axion Biosystems) responded by referencing ongoing work in cardiotoxicity prediction, including studies aligned with the CiPA (Comprehensive in vitro Proarrhythmia Assay) initiative. He highlighted a recent publication: “In vitro cardiac New Approach Methodologies predict clinical cardiovascular repolarization risk comparable to nonclinical animal studies”.
In this paper, the authors from the FDA define human iPSC-derived cardiomyocytes (hiPSC-CMs), one of the simplest NAMs, as a “potential alternative to animal studies in regulatory assessments.” Notably, hiPSC-CMs predicted every instance of Grade 3 QT prolongation observed in the clinic and, in some cases, outperformed in vivo dog QT assessments. Using hiPSC-CM data in combination with other in vitro assays reduced nonclinical QT false negatives and provided predictive value comparable to multiple animal studies. This study demonstrates that NAM-based models can predict cardiac safety issues that are missed by animal tests, thereby reinforcing the case for human-relevant systems.
Nicole emphasized that context of use is critical: NAMs must be fit-for-purpose. While they offer powerful tools for early screening, they currently provide partial recapitulation of complex disease processes. Florian added that NETRI’s own studies demonstrated that NAMs can predict failures that animal models missed—strong evidence of their potential to de-risk development.
Serah Kang of Genentech, echoed this sentiment, stressing that NAMs aren’t universally applicable yet, but they enable earlier toxicity assessments, improving safety and focusing resources on promising programs.
Why This Matters
The shift to NAMs isn’t just about science —it’s about impact: faster drug development, lower costs, better patient outcomes, and reduced animal use. As Nicole put it, NAMs offer “piecemeal recapitulation” today, but they’re enabling technologies that will transform the earliest stages of drug discovery.
The Road Ahead
NAMs are not a silver bullet. Challenges remain, including validation and reproducibility, the appropriate context of use, global harmonization of standards, and investment in technology and training. Still, the trajectory is clear. NAMs will dominate early-stage drug discovery and gradually expand into safety testing.
NAMs are here to stay, and their adoption is accelerating worldwide. Regulatory momentum is global, with agencies driving change across regions. Success depends on rigorous validation and strong collaboration, and context remains critical: for now, NAMs complement rather than replace traditional models. Bottom line: the future of neuroscience drug discovery is human-relevant, data-driven, and collaborative. NAMs are leading the way, and the time to embrace them is now because every moment matters.