Intrathecal catheter drug delivery platform for central nervous system disorders
A novel intrathecal drug delivery approach targeting the cerebrospinal fluid to treat multiple sclerosis and other CNS disorders. Building on validated riluzole delivery data showing 17-fold increased spinal cord drug concentration with reduced variability, this platform evaluates masitinib delivery via a steerable catheter designed to reach basal cisterns, potentially overcoming blood-brain barrier limitations.
Overview
This research addresses a fundamental challenge in treating central nervous system (CNS) disorders: delivering therapeutic drugs effectively across the blood-brain barrier (BBB). The team is investigating intrathecal delivery of masitinib as a treatment for multiple sclerosis (MS), hypothesizing that direct cerebrospinal fluid (CSF) administration will achieve higher drug concentrations in the spinal cord and brain while reducing peripheral toxicity. The approach combines pharmacological innovation with a novel steerable catheter technology designed to navigate from the lumbar region to the basal cisterns of the brain.
The platform has strong potential applications for MS, amyotrophic lateral sclerosis (ALS), and other CNS conditions where existing oral or intravenous therapies are limited by peripheral side effects and inadequate brain penetration.
Technical specifications
Drug delivery approach:
- Direct intrathecal administration bypasses the blood-brain barrier, delivering masitinib into cerebrospinal fluid
- Targets reduction of inflammatory markers, axonal degeneration, and immune cell infiltrates in the CNS
- Comparison of intrathecal versus subcutaneous delivery in an experimental autoimmune encephalomyelitis (EAE) model using MOG:35-55 peptide
Steerable catheter technology:
- Novel catheter designed for lumbar-to-chiasmatic cistern navigation
- Distal end compliance enabled by femtosecond laser processing
- Two sets of antagonist/agonist tendons providing three degrees of freedom for directional steering
- Cadaver validation to confirm placement without damage to spinal cord or brainstem
Preliminary validation data:
- Canine model studies with intrathecal riluzole demonstrated 17-fold increase in spinal cord tissue drug concentration (8.1±2.1 µg/g vs. 0.48±0.41 µg/g) compared to oral delivery
- Animal-to-animal variability reduced from 87% to 26% standard deviation
- Well-tolerated for at least six months in preclinical testing
Technology readiness level
The research is currently at the preclinical validation stage. Preliminary work optimizing intrathecal riluzole delivery for ALS has generated robust proof-of-concept data in a canine model. The masitinib program is advancing through two specific aims: (1) efficacy comparison of intrathecal versus peripheral delivery in an EAE disease model, and (2) catheter design and cadaver testing for basal cistern access. Upon successful completion of these preclinical milestones, the platform would advance toward IND-enabling studies and early-phase clinical trials. The approach leverages established regulatory pathways for intrathecal drug delivery and catheter-based medical devices.
About Emory University
Emory University is a comprehensive private research university in Atlanta, anchored by a major academic health center and a mid-sized, research-active faculty. Integration with its academic health system provides direct access to patients, clinicians, and real-world data, enabling rapid clinical translation and large, multisite trials. A hospital-embedded innovation hub and proximity to the CDC and Atlanta’s corporate ecosystem create frequent pathways for co-development, testing, and deployment with industry partners; a long-standing partnership with a neighboring engineering powerhouse broadens prototyping and scale-up options. Research is supported by competitive federal funding, including the National Institutes of Health and the National Science Foundation. A dedicated technology transfer office supports IP strategy, licensing, startup formation, and streamlined industry agreements.
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