Experience and expertise of
~200
neuroscientists

Charles River worked on
95%
of new FDA-approved CNS drugs in the last 5 years

CNS Scientific achievement
80
publications and patents

Neurotoxicity Testing

We understand how important it is to know if your test material adversely affects the chemistry, structure, or function of the nervous system. With this need in mind we offer GLP-compliant neurotoxicity testing to assess the effects of pharmaceuticals, biopharmaceuticals, biologics, or chemicals in both rodent and non-rodent species. With over two decades of experience, we have valuable historical control databases.

Our Neurotoxicity Testing Capabilities

  • Neurobehavior/ behavior testing, including assays of locomotor activity, rotarod forced activity, water maze learning and memory testing, auditory startle habituation, and functional observational batteries.
  • Abuse liability testing with any drug that crosses the blood-brain barrier regardless of therapeutic indication.
  • Neurite outgrowth assays, method to examine the potential effects of exogenous compounds on neuronal activity and development, providing valuable information on the potential of therapeutic compounds to either enhance or inhibit neuritogenesis.
  • Ototoxicity testing, GLP-compliant and measuring auditory function, cytocochleogram, otic histology, and evaluation of potential recovery.
  • Neurochemistry, including cholinesterase assays in red blood cell, plasma, and brain samples, as well as quantification of endogenous levels of neurotransmitters.
  • Neuropathology, including in situ perfusion, morphometry, stereology, and qualitative neuropathological evaluations.

Neurotoxicity Testing Study Designs

Dose Routes of Neurotoxicity Assessments

Choosing the correct administration route is essential in neurotoxicity studies, especially for CNS-targeted therapies. From systemic delivery to direct CNS administration, our experts help identify the most effective approach for your small or large molecule. With the rise of gene therapies, oligonucleotides, and antibodies, overcoming the blood-brain barrier has become a key challenge in neuroscience drug development. Our team brings deep experience across delivery strategies to support safety and efficacy goals. Explore how we support CNS discovery programs through optimized delivery methods.

Illustration of neurons

Exploratory Toxicology for Neuroscience Drug Discovery
This eBook describes strategies across the early stages of drug discovery to support confidence in your lead small molecule candidate and to ensure you proceed through the drug development process with the most promising candidate.
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Standard and Specialized Parenteral Routes in Neurotoxicity Assays

These are commonly used in both routine and advanced neurotoxicity evaluations:

These routes vary in invasiveness and distribution potential, with several designed to bypass the Blood-Brain Barrier (BBB) or deliver compounds directly into cerebrospinal fluid or CNS tissues.

10 Regulatory Truths for Biotech Founders
Do not leave your breakthrough to a chance; download this complimentary infographic as a concise checklist to help you stay on track and reach your milestones.
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Emerging Route in Neurotoxicity Studies: MRgFUS (Magnetic Resonance–guided Focused Ultrasound)

MRgFUS is a non-invasive, image-guided delivery method that opens the BBB temporarily and precisely, allowing therapeutic agents to reach targeted brain regions. This innovative route offers several key advantages in neurotoxicity studies:

  • Targeted Delivery: Reduces off-target exposure and systemic toxicity
  • Enhanced Safety Profiling: Enables real-time monitoring during BBB opening for earlier detection of adverse effects
  • Improved Translational Relevance: Facilitates the development of more realistic CNS disease models and supports safer clinical translation

By integrating MRgFUS into neurotoxicity screening programs, you will access a cutting-edge tool that enhances precision and safety in your CNS drug delivery.

This partnership will not only expand the application of our low-frequency focused ultrasound technology, but also accelerate the development of groundbreaking therapies for neurological conditions.By combining our expertise, we aim to revolutionize the way next generation therapeutics are delivered and actuated in the brain, ultimately improving the lives of millions globally.”

Arjun Desai, M.D., Chief Strategic Innovation Officer of Insightec

 

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Frequently Asked Questions (FAQs) About Neurotoxicity Testing Services

  • What is neurotoxicity, the fundamental purpose of neurotoxicity testing?

    Neurotoxicity testing studies are used to evaluate adverse effects of biological, chemical, or physical agents on the structure or function of the central and/or peripheral nervous system. The level and the intensity of exposure to neurotoxins often alters the normal activity of the nervous system. This can eventually disrupt or even kill neurons, key cells that transmit, and process signals in the brain and other parts of the nervous system.

  • What are key regulatory agencies and guidelines that govern neurotoxicity testing?

    Since 1984 the U.S. Environmental Protection Agency (EPA)  set forth Guidelines for Neurotoxicity Risk Assessment, to guide EPA scientists in evaluating environmental contaminants that may pose neurotoxic risks, and inform Agency decision makers and the public about these procedures. The National Center for Toxicological Research (NCTR) is an important research component of the U.S. Food and Drug Administration (FDA) that conducts FDA research with the goal of developing a scientifically sound basis for regulatory decisions and reduce risks associated with FDA-regulated products. NCTR’s the division of neurotoxicology focuses on increasing FDA’s understanding of the processes associated with neurotoxic outcomes—harmful effects associated with the brain and nervous system. In the European Union, so far, the systematic testing for Developmental Neurotoxicity Testing (DNT), is not a mandatory regulatory requirement for chemical safety assessment purposes. Significant research work carried out over the last three decades has built awareness about in vivo developmental neurotoxicity testing guidelines by US EPA and OECD.

  • What is neurotoxicity from chemotherapy?

    Neurotoxicity Models in Oncology Studies: Neurotoxicity is a common side effect of chemotherapy and of emerging modalities such as CAR-T cells. Charles River’s neurotoxicity models can act as a stand-alone test of therapeutic agents targeting these side effects, or as a model to test the neurotoxic effect of a novel or combination therapy. Charles River has two in vivo models of the neurotoxic effects associated with chemotherapy and many other cancer treatments: peripheral neurotoxicity model and the neuropathic pain model.

  • How is MRgFUS being used today, and what types of therapies can benefit from it?

    MRgFUS is showing strong promise across a range of neuroscience applications. It supports the targeted, noninvasive delivery of genetic medicines like viral vector gene therapies, siRNA, ASOs, monoclonal antibodies, and biologics, whether single or repeat dosing. The U.S. Food and Drug Administration (FDA) has approved the ExAblate® MRgFUS system for four indications: treatment of uterine fibroids (leiomyomata), palliation of pain associated with tumors metastatic to bone, medication-refractory essential tremor, and tremor-dominant Parkinson’s disease.

  • How is MRgFUS transforming nonclinical neurotoxicity studies in CNS drug development?

    MRgFUS is breaking new ground in nonclinical neurotoxicity by safely opening and modulating the blood-brain barrier (BBB) to enable precise delivery of CNS therapies. This translatable focused ultrasound approach enhances the accuracy of therapeutic delivery to the brain and has the potential to reduce off-target side effects, ensuring the efficacy and safety of your novel therapeutic.