High-Efficacy DMTs PROVEN
Ocrelizumab (Ocrevus) — Anti-CD20 FDA 2017
B-cell depleter. PROVEN for both RRMS AND PPMS. First approved PPMS treatment ever. RRMS: 46% reduction in relapse rate vs interferon (OPERA I+II). PPMS: slows confirmed disability progression. 6-monthly infusion.
Hauser et al. 2017 NEJM (OPERA I+II) · Montalban et al. 2017 NEJM (ORATORIO for PPMS)
Natalizumab (Tysabri) PROVEN
Anti-α4-integrin. Blocks T-cell entry to CNS through BBB. 68% relapse reduction. PML risk: JC virus reactivation. Annual JCV antibody monitoring required. Hold if JCV Ab titre rises.
Polman et al. 2006 NEJM (AFFIRM trial)
Alemtuzumab (Lemtrada) PROVEN
Anti-CD52. Depletes most lymphocytes. Annual short-course infusion. 49% reduction vs interferon. High efficacy with significant secondary autoimmunity risk (thyroid, ITP, nephropathy). Used for highly active RRMS.
Cohen et al. 2012 Lancet (CARE-MS II)
Moderate-Efficacy
Interferons + Glatiramer Acetate
First-generation. ~30% relapse reduction. Well-tolerated, long track record. Injectable. Now often replaced by higher-efficacy oral/infusion options for active disease. Still used for mild/moderate RRMS.
IFNB-1b Study Group 1993 · Johnson et al. 1995 (GA)
Cladribine (Mavenclad) PROVEN
Oral purine analogue. Selective lymphocyte depletion. 2 short annual courses over 2 years. ~60% relapse reduction. Long lymphocyte recovery — avoid in pregnancy.
Giovannoni et al. 2010 NEJM (CLARITY trial)
Open Pipeline
Remyelination Therapy FAILED TRIALS
Opicinumab (anti-LINGO-1), bexarotene, clemastine — all failed Phase 2/3. Remyelination works in mouse models. Human translation has failed so far. SORRY: PPMS treatment still limited to ocrelizumab with modest benefit.
SORRY: progressive MS neurodegeneration independent of inflammation not yet targetable
Established Biomarkers
| MRI T2 FLAIR PROVEN | White matter lesions. Periventricular (Dawson's fingers = perpendicular to ventricles), juxtacortical, infratentorial, spinal cord. McDonald criteria require DIS (3+ regions) + DIT (active + inactive). Gold standard. |
| Gd-enhancing PROVEN | Gadolinium-enhancing lesions = active BBB breakdown = acute inflammation. Confirms DIT when combined with non-enhancing. Reflects current disease activity. |
| CSF OCBs PROVEN | Oligoclonal bands. IgG synthesised intrathecally. 95% sensitivity for MS. Not specific (also positive in CNS infections, neurosarcoid). Fulfils McDonald DIT criterion. |
| VEP latency PROVEN | Visual evoked potential latency increase = optic nerve demyelination. Most common MS first symptom (optic neuritis). McDonald criterion for DIS. Can detect subclinical lesions. |
| serum NfL | Monitors treatment response. Drops with effective DMT. Tracks neurodegeneration. Research use, increasingly clinical. Kappos et al. 2019 |
Open Sorries
SORRY-1: PPMS treatment limited — only ocrelizumab, modest 25% slowing of confirmed disability. Most progression still occurs.
SORRY-2: Remyelination therapy has failed in every trial. Mouse → human translation failed.
SORRY-3: Progressive MS mechanism (neurodegeneration independent of inflammation?) poorly understood. DMTs that work for RRMS barely touch SPMS/PPMS.
Novel Patterns (8)
PROVENNP-MS-001 · Oligodendrocyte serves 50 axons — one cell death = 50 demyelinated segments · CNS cannot remyelinate efficiently unlike PNS
PROVENNP-MS-002 · Ocrelizumab: first drug proven for PPMS (2017) — changed landscape of progressive MS management · anti-CD20 B-cell mechanism unexpected
PROVENNP-MS-003 · VEP latency increase: subclinical optic neuritis detectable before visual symptoms — DIS criterion fulfilled silently
PROVENNP-MS-004 · Uhthoff's phenomenon: heat blocks conduction in demyelinated axon · explains exercise-induced temporary worsening · not relapse
PROVENNP-MS-005 · OCBs 95% sensitive for MS — IgG made inside CNS, not in blood · intrathecal synthesis confirms CNS inflammation
OPENNP-MS-006 · Serum NfL as treatment response monitor — drops within weeks of effective DMT · could enable personalised dosing
OPENNP-MS-007 · Progressive MS neurodegeneration: independent of acute inflammation? PPMS has fewer visible lesions than RRMS but more disability · mechanism gap
SORRYNP-MS-008 · Remyelination therapy failed in every trial · PPMS still largely untreatable · progressive mechanism unknown · mouse models don't translate