Serum and CSF Neuroflament Light Chain levels normalise following Bone Marrow Transplant in MS Patients (P5.036)

Objective: To quantify neurofilament light chain (NF-L) levels in paired serum and CSF samples taken from MS patients before and after BMT. Background: NF-L is a potential biomarker for tissue damage in Multiple Sclerosis (MS) and other neurological conditions. It has the potential for denoting disease severity and possibly guiding treatment response. Highly sensitive and validated assays can quantify NF-L in serum as well as cerebrospinal fluid (CSF). A serum test might inform clinicians regarding disease severity, for initiating a particular treatment or guide in treatment changes. Autologous bone marrow transplantation (BMT) is reserved for aggressive forms of MS that has been shown to halt all detectable CNS inflammatory activity for prolonged periods in the absence of any concomitant disease-modifying drugs. Such cases of MS are thought to have significant tissue damage that should be reflected in the amount of NF-L released and thus measured. Design/Methods: Paired serum and CSF were collected from 23 MS patients at baseline, 1 and 3 years post BMT. An additional 5 pairs were taken from non-inflammatory neurological controls. NF-L was quantitated in both serum and CSF using the SIMOA platform (Quanterix). Results: MS NF-L levels were significantly elevated relative to controls in serum and CSF. Following BMT, the high baseline levels of NF-L significantly reduced in both compartments (p Conclusions: We show that serum and CSF NF-L levels are elevated in patients with aggressive MS and are both significantly reduced following BMT. This suggests that serum NF-L can be used as a biomarker for response to therapy and CSF may not be required. Further research will establish serum NF-L as a marker of MS disease severity to guide treatment options. Study Supported by: Quanterix: kindly provided NF-L assay kits Disclosure: Dr. Thebault has nothing to disclose. Dr. Tessier has nothing to disclose. Dr. Stonebridge has nothing to disclose. Dr. Tabard-Cossa has nothing to disclose. Dr. Freedman has received personal compensation for activities compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Actelion, Bayer Healthcare, Biogen, Canada Innovation, Chugai, Clene Nanomedicine, EMD Canada, Hoffman-La Roche, Merck Serono, Novartis, Opexa, Sanofi Aventis, Sanofi Genzyme, and Teva.