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Related Experiment Videos

Stem cell transplantation for autoimmune disorders. Multiple sclerosis.

Athanasios Fassas1, Richard Nash

  • 1Bone Marrow Transplantation Unit, Department of Haematology, George Papanicolaou Hospital, 57010 Exokhi, Thessaloniki, Greece. hempap@otenet.gr

Best Practice & Research. Clinical Haematology
|August 11, 2004
PubMed
Summary

Autologous hematopoietic stem cell transplants may help severe multiple sclerosis (MS) by

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Area of Science:

  • Neuroimmunology
  • Hematopoietic Stem Cell Transplantation
  • Multiple Sclerosis Pathogenesis

Background:

  • Autologous transplants for multiple sclerosis (MS) have been explored since 1997.
  • Approximately 200 patients have undergone this procedure globally.
  • Early studies suggest potential for reducing CNS inflammation and delaying disease progression.

Purpose of the Study:

  • To evaluate the efficacy and safety of autologous stem cell transplantation in severe and refractory multiple sclerosis.
  • To understand the evolving concepts of immune modulation through transplantation in MS.

Main Methods:

  • High-dose immunosuppressive therapy followed by autologous stem cell transplantation.
  • Patient selection criteria focus on young individuals with rapidly progressing, inflammatory MS.

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  • Monitoring for treatment toxicity and infectious complications.

    • Main Results:

    • Phase I/II studies indicate suppression of central nervous system (CNS) inflammation.
    • Potential delay in clinical disease progression observed.
    • Procedure carries risks including toxicity, serious infections, and a 1-5% transplant-related mortality.

    Conclusions:

    • Autologous transplantation is a high-risk procedure reserved for carefully selected MS patients.
    • The concept has shifted from immune ablation to immune system 'resetting'.
    • Comparative studies are needed to definitively demonstrate the clinical effectiveness of transplantation in MS.