Franklin Biolabs Achieves CLIA Certification for AAV NAb Testing
Learn more

Functional Recovery Assessment in a Spinal Cord Injury NHP Model Following MSC Therapy

PROVEN INTELLIGENCE ACCELERATING NEXT-GENERATION THERAPIES

Functional Recovery Assessment in a Spinal Cord Injury NHP Model Following MSC Therapy

Functional Recovery Assessment in Spinal Cord Injury NHP Models

CELL & GENE | RNA | BIOLOGICS

    What functional endpoints are most relevant for assessing MSC therapy efficacy in an SCI NHP model?

    Key endpoints include validated motor function scales (e.g., BMS), quantitative sensory testing for multiple modalities, and electrophysiological measurements such as motor evoked potentials (MEPs) and somatosensory evoked potentials (SSEPs). This multi-faceted approach provides a comprehensive view of neurological recovery.

    How do you ensure the translational relevance of the NHP model for human spinal cord injury?

    Translational relevance is established through precise surgical induction of the injury at a clinically relevant spinal level, mimicking the pathophysiology of human SCI. We characterize the model to ensure it aligns with the expected clinical presentation, which is foundational for evaluating therapeutic candidates.

    What is the typical study duration for an NHP SCI functional recovery study?

    Study duration is program-dependent but is designed to be sufficient for observing and quantifying meaningful, long-term functional improvements. The timeline must also allow for comprehensive terminal histological analysis of neural tissue repair to correlate function with mechanism.

    How is the biodistribution of mesenchymal stem cells (MSCs) tracked in these models?

    We employ a multi-modal strategy under GxP standards. This includes qPCR for cell-specific genetic markers in all relevant tissues, advanced in-life imaging where applicable, and definitive confirmation through terminal histology to assess cell engraftment, persistence, and non-target tissue biodistribution.

Non-human primate (NHP) models of spinal cord injury (SCI) provide the most translationally relevant in vivo data for assessing functional recovery following administration of Mesenchymal Stem Cell (MSC) therapies. A robust program design integrates quantitative behavioral scoring, electrophysiology, and advanced imaging, culminating in terminal histology to correlate functional gains with structural neural repair. This rigorous approach generates the definitive data package required to de-risk clinical development and support an accelerated 18-24 month IND timeline.

A stylized rendering of a DNA double helix on the left side of a light blue gradient background.

Defining Translational Endpoints in NHP Models

A comprehensive evaluation of a cell therapy for SCI focuses on quantifying neurological function to supplement safety and viability data. To generate clinically relevant data from the primate central nervous system, a multi-modal battery of assessments is employed.

Our study designs incorporate a matrix of endpoints to build a comprehensive profile of therapeutic effect:

  • Behavioral and Motor Function: Longitudinal assessment using established scoring systems to track recovery of limb movement, coordination, and locomotion.

  • Sensory Pathway Assessment: Quantitative testing to measure the recovery of sensory responses, a key aspect of patient quality of life.

  • Electrophysiology: Direct measurement of nerve conduction velocity and pathway integrity using MEPs and SSEPs provides objective data on neural circuit restoration.

  • Advanced Imaging: In-life MRI can be used to monitor lesion volume, edema, and gross morphological changes within the spinal cord over time.

Program Design for Cell Therapy Evaluation

A successful preclinical program hinges on a meticulously planned study design executed in a facility equipped for long-term NHP care and complex surgical procedures. Our >100,000 sq ft facility is designed to support these demanding, long-duration studies. Optimizing the operational aspects of these programs is key to managing development timelines and resources effectively.

A close-up, blue-toned image of scientific glassware, featuring vials placed in a dish filled with clear, spherical beads, suggesting a laboratory or research setting.

A scientist in a modern lab analyzes colorful DNA sequencing data on a tablet.

De-risking Clinical Development with Predictive Models

The primary objective of any preclinical study is to generate a data package that accurately predicts clinical outcomes. As demonstrated in complex biologic development programs (PMID: 27386755), the use of a well-characterized, translationally relevant large animal model is fundamental to establishing efficacy and determining an effective dose before advancing to first-in-human trials. This principle of using a robust model to enable accurate evaluation is directly applicable to assessing cell therapies in NHP SCI models. This rigorous, data-driven approach has supported our clients in achieving a 100% IND success rate since 2019. (The Franklin Biolabs brand launched in 2024).

Integrated Histology and Biodistribution Analysis

Functional data must be supported by physical evidence of cellular activity and tissue repair. Our programs conclude with a comprehensive GxP-compliant analysis to provide this mechanistic link.

  • Histology: Detailed microscopic examination of spinal cord tissue to identify and quantify axonal sprouting, myelination, glial scar reduction, and immune cell infiltration.

  • Biodistribution: A full analysis of non-target tissue biodistribution is performed to confirm the safety profile and ensure the cell therapy remains localized or behaves as expected systemically.

All in vivo research is conducted with the highest commitment to ethical standards. We execute these studies through our strategic partner, The Bioculture Group, whose animal welfare program is accredited by AAALAC and registered with the USDA. Their program is built upon the principles of the 3Rs (Replacement, Reduction, and Refinement), ensuring both the ethical treatment of animals and the integrity of the scientific data generated.

Scientific Process Diagram

This content is for informational purposes. For guidance specific to your therapeutic program, please contact our team for a consultation.

Explore More Capabilities