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Related Experiment Video

Updated: Jun 12, 2026

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
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Intrathecal methotrexate treatment in multiple sclerosis.

Saud A Sadiq1, Elena V Simon, Lauren M Puccio

  • 1Multiple Sclerosis Research Center of New York, 4th Floor, New York, NY 10019, USA. ssadiq@msrcny.org

Journal of Neurology
|June 10, 2010
PubMed
Summary

Intrathecal methotrexate (ITMTX) shows promise for progressive multiple sclerosis (MS) patients unresponsive to other treatments. This study found ITMTX to be well-tolerated, with stable or improved disability scores in most patients.

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Published on: February 24, 2026

Area of Science:

  • Neurology
  • Immunology
  • Pharmacology

Background:

  • Multiple sclerosis (MS) is a chronic autoimmune disease affecting the central nervous system.
  • Progressive forms of MS often have limited treatment options, especially for patients unresponsive to standard therapies.
  • Intrathecal methotrexate (ITMTX) is an alternative treatment approach for severe or refractory cases.

Purpose of the Study:

  • To evaluate the feasibility and safety of intrathecal methotrexate (ITMTX) in patients with progressive multiple sclerosis (MS).
  • To assess the efficacy of ITMTX in stabilizing or improving neurological function and disability in treatment-unresponsive MS patients.

Main Methods:

  • A retrospective, open-label chart review of 121 patients with progressive MS receiving up to eight ITMTX treatments.
  • Patients were selected if unresponsive or intolerant to FDA-approved MS treatments.
  • Neurological assessments and Expanded Disability Status Scale (EDSS) evaluations were performed during a 1-year follow-up period.

Main Results:

  • No serious adverse events were reported during the study.
  • In 87 secondary progressive MS patients, 89% showed stable or improved EDSS scores, with a significant post-treatment improvement (P=0.014).
  • In 34 primary progressive MS patients, 82% had stable EDSS scores with no significant progression.

Conclusions:

  • Intrathecal methotrexate (ITMTX) appears to be a feasible and well-tolerated treatment option for progressive multiple sclerosis (MS).
  • ITMTX demonstrated potential benefits in stabilizing or improving disability in patients with secondary and primary progressive MS.
  • Further research may support ITMTX as a valuable therapeutic strategy for refractory progressive MS.