Late breaking newsSunday, October 14, 2007, 09:30 - 09:45
Exploring recombinant human erythropoietin in chronic progressive multiple sclerosisH. Ehrenreich, B. Fischer, C. Norra, F. Schellenberger, N. Stender, M. Stiefel, A-L. Sirén, W. Paulus, K-A. Nave, R. Gold, C. Bartels (Gottingen, D)
Objective: 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 (1) evaluating safety of long-term high-dose intravenous rhEPO treatment in MS, and (2) collecting first evidence of potential efficacy on clinical outcome parameters.
Methods and Results: Eight MS patients, five randomly assigned to high-dose (48000 IU), three to low-dose (8000 IU) rhEPO treatment, and, as disease controls, two drug-naïve Parkinson patients (receiving 48000 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 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. Interpretation: This first pilot study demonstrates the necessity and feasibility of controlled trials using high-dose rhEPO in chronic progressive MS