Journal Article

Exploring recombinant human erythropoietin in chronic progressive multiple sclerosis

Hannelore Ehrenreich, Benjamin Fischer, Christine Norra, Felix Schellenberger, Nike Stender, Michael Stiefel, Anna-Leena Sirén, Walter Paulus, Klaus-Armin Nave, Ralf Gold and Claudia Bartels

in Brain

Published on behalf of The Guarantors of Brain

Volume 130, issue 10, pages 2577-2588
Published in print October 2007 | ISSN: 0006-8950
Published online August 2007 | e-ISSN: 1460-2156 | DOI:
Exploring recombinant human erythropoietin in chronic progressive multiple sclerosis

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The neurodegenerative aspects of chronic progressive multiple sclerosis (MS) have received increasing attention in recent years, since anti-inflammatory and immunosuppressive treatment strategies have largely failed. However, successful neuroprotection and/or neuroregeneration in MS have not been demonstrated yet. Encouraged by the multifaceted neuroprotective effects of recombinant human erythropoietin (rhEPO) in experimental models, we performed an investigator-driven, exploratory open label study (phase I/IIa) in patients with chronic progressive MS. Main study objectives were (i) evaluating safety of long-term high-dose intravenous rhEPO treatment in MS, and (ii) collecting first evidence of potential efficacy on clinical outcome parameters.

Eight MS patients, five randomly assigned to high-dose (48 000 IU), three to low-dose (8000 IU) rhEPO treatment, and, as disease controls, two drug-naïve Parkinson patients (receiving 48 000 IU) were followed over up to 48 weeks: A 6-week lead-in phase, a 12-week treatment phase with weekly EPO, another 12-week treatment phase with bi-weekly EPO, and a 24-week post-treatment phase. Clinical and electrophysiological improvement of motor function, reflected by a reduction in expanded disability status scale (EDSS), and of cognitive performance was found upon high-dose EPO treatment in MS patients, persisting for three to six months after cessation of EPO application. In contrast, low-dose EPO MS patients and drug-naïve Parkinson patients did not improve in any of the parameters tested. There were no adverse events, no safety concerns and a surprisingly low need of blood-lettings.

This first pilot study demonstrates the necessity and feasibility of controlled trials using high-dose rhEPO in chronic progressive MS.

Keywords: recombinant human erythropoietin; EPO; primary and secondary progressive multiple sclerosis; neuroprotection; neuroregeneration; neuropsychology; expanded disability status scale (EDSS); walking distance

Journal Article.  6385 words.  Illustrated.

Subjects: Neurology ; Neuroscience

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