High-dose cyclophosphamide for moderate to severe refractory multiple sclerosis: 2-year follow-up (Investigational new Drug no. 65863)

Douglas E. Gladstone, Robert Peyster, Edward Baron, Sharon Friedman-Urevich, Patrick Sibony, Patricia Melville, Malcolm Gottesman

Research output: Contribution to journalArticlepeer-review

10 Scopus citations


High-dose cyclophosphamide (HDC) is a chemotherapy treatment designed to eradicate autoreative B- and T-cells responsible for lymphocyte-mediated autoimmune illness while sparing the pluripotent blood stem cell of any ill effects. Multiple sclerosis (MS) is the most common inflammatory and demyelinating immune-mediated disorder of the central nervous system in young adults. Patients with moderate to severe, refractory MS, defined as an Expanded Disability Status Scale (EDSS) score of 3.5 or higher after two or more Food and Drug Administration-approved disease-modifying agents, received 200 mg/kg of cyclophosphamide over 4 days. For the next 2 years, quarterly EDSS score evaluations and biannual brain magnetic resonance imaging and neuro-ophthalmologic evaluations were obtained. Fifteen patients were evaluated for clinical response. During follow-up, one patient increased their baseline EDSS score by 1.0. EDSS score stability of decrease was realized in five of seven (71%) patients with relapsing-remitting MS and six of eight (75%) patients with secondary progressive MS. Four patients required additional immunomodulatory treatment after treatment. Treatment response was seen regardless of the baseline presence or absence of contrast lesion activity. HDC can effectively decrease symptoms, stop disease progression, and allow for disability regression in relapsing-remitting MS and secondary progressive MS patients. The most appropriate candidates for HDC, its duration of benefit, and the potential need for prophylactic preventative immune manipulation after HDC all require further investigation.

Original languageEnglish (US)
Pages (from-to)23-30
Number of pages8
JournalAmerican Journal of Therapeutics
Issue number1
StatePublished - Jan 2011


  • chemotherapy
  • high-dose cyclophosphamide
  • multiple sclerosis
  • relapsing-remitting multiple sclerosis
  • secondary progressive multiple sclerosis

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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