Cladribine May Be Beneficial for Managing Progressive Multiple Sclerosis

cerebrospinal fluid csf
cerebrospinal fluid csf
Patients with increased disease activity seen on MRI or with elevated CSF NfL should be considered for disease-modifying immune therapy.

According to 2 case studies recently published in Multiple Sclerosis and Related Disorders, cladribine, a disease-modifying immune therapy (DMT), may benefit patients with progressive multiple sclerosis (pwPMS).  

Ocrelizumab, a DMT, is currently the only available licensed medication for pwPMS, and it is found to be only partially effective. Phase 2 clinical trials have suggested that cladribine, a medication licensed for hairy cell and chronic lymphoid leukemia treatment, may be a viable option for pwPMS. 

Investigators reported case studies of 2 patients with pwPMS, both seen at The Royal London Hospital, London, UK. Prior to attempting cladribine, each patient had undergone multiple unsuccessful medication trials.   

Subcutaneous cladribine was administered in 2 courses of 3 consecutive days each, 10 mg per dose. Before and after treatment, data were collected through clinical assessment, magnetic resonance imaging (MRI), and cerebrospinal fluid (CSF) neurofilament light chain (NfL) testing to measure disease severity. The Expanded Disability Status Scale (EDSS) was administered as part of the clinical assessment. CSF NfL marks neuroaxonal damage and was selected as a potential biomarker for treatment outcome.   

Following treatment, one patient had mild symptom progression, as suggested by a change in EDSS score from 6.0 to 6.5. The other patient’s symptoms remained stable; EDSS was 5.5 before and after the medication trial. 

Both patients showed a decline in disease activity per MRI and CSF NfL testing. CSF NfL decreased by 73% in one patient and by 80% in the other patient.   

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No side effects were reported by either patient from the medication. It was well-tolerated. 

“pwPMS with detectable disease activity (MRI, elevated NfL) should be considered for DMT. NfL appears to be a sensitive index of treatment effect in pwPMS and may be a useful outcome in clinical trials targeting this patient group. Over and above its licensed indication (relapsing MS), cladribine may be an effective treatment option for pwPMS,” the authors concluded.  

Disclosures. Several authors reported affiliations with the pharmaceutical industry. Please refer to the full-length text for a list.


Yildiz O, Maoa Z, Adams A, et al. Disease activity in progressive multiple sclerosis can be effectively reduced by cladribine. Mult Scler Relat Disord. 2018;24:20-27.