Transcript Document
MultipleSclerosis:Current&Emerging Treatments Personalized Strategies Dr. Suhail Al-Shammri Associate Professor& Head Division of Neurology, Mubark Al Kabir Hospital Overview • The diagnostic criteria of multiple sclerosis( MS) • Classification of idiopathic inflammatory demyelinating disorders • Clinical course of MS • Current and emerging MS therapies Idiopathic CNS Demyelinating Diseases • Typical CNS demyelinating Diseases: – Radiologically isolated syndrome (RIS) – Clinically isolated syndrome (CIS) – Relapsing –remitting multiple sclerosis (RRMS) – Secondary progressive MS (SPMS) – Primary progressive MS (PPMS) Atypical CNS Demyelinating Diseases • Acute disseminated encephalomyelitis (ADEM) • Acute hemorrhagic leukoencephalitis (AHLE) • Tumefactive MS • Balo’s concentric sclerosis • Marburg Radiologically Isolated Syndrome (RIS) – – – – – – – No typical symptoms of CNS demyelination No formally accepted diagnostic criteria MRI : Typical MS lesions CSF abnormalities Clinical MS Attack: 35% over 5 years MRI progression: • 59-83% in 2 years – DMT is initiated only in case of clinical/MRI progression Okuda DT et al, Neurology2011:76()8, 686-692 Diagnostic Criteria for MS • An effort to make the diagnostic process more objective • Formal criteria were devised to codify the typical MS features into indisputable diagnostic criteria • The primary driving force is identification of patients for research trials. ”a consensus on which patient has MS” • Criteria are designed to be specific • There are patients with MS who do not meet those criteria • “A patient has MS when an an experienced neurologist says he or she has MS” Schumacher Criteria- 1965 • Onset of symptoms between 10 and 50 years • Objective abnormalities on neurologic examination • The signs and symptoms indicate CNS white matter damage • The lesions are disseminated in space ( 2 or more separate lesions) • The lesions are disseminated in time (2 attacks at least 1 month apart) • No better explanation The Mc Donald Criteria • ‘the world consists of three types of person: those who have multiple sclerosis; those who do not; and those who might’. Polman CH et al,Ann Neurology, 2001 Episodes from history Objective clinical signs Additional data needed from MRI or clinical follow up 2 attacks 2 attacks 1 attack 1 attack 2 lesions 1 lesion 2 lesions 1 lesion None DIS DIT Both DIS&DIT Progressive course over 1 year DIS demonstrated by 2 : 1- MRI brain 2. MRI cord 3. CSF oligoclonal bands Dissemination in Space Polman CH et al, Ann Neurol 2011; 69:292–302 Dissemination in Time Polman CH et al, Ann Neurol 2011; 69:292–302 MRI Brain DIS MRI Cervical spine: DIS MRI Brain T2WI MRI Brain Enhanced T1WI:DIT CASE • On 18/3/08 patient complained of ocular pain on moving the left eye with blurred vision. • 2 days later she developed left frontal headache. • Seen by the ophthalmologist who diagnosed her as optic neuritis and advised to be on neurobion. • She had several attacks of Rt. Upper limb heaviness in the last 2 years, each was lasting for a week. • Her cousin has MS . CASE : Examination • Her vision was 20/200 in the left eye and 20/40 in the right eye. There was a central scotoma, and red and blue colors were less intense in the left eye. • RAPD on the left • Left fundus: disc is congested and swollen. • Central Scotoma • Treated with pulse IV methylprednisolone for 3 days and improved followed by short prednisolone taper Case 1: Fundoscopy Case : MRI Brain Case 2: MRI Spine Case : CSF Oligoclonal bands Clinically Isolated Syndrome (CIS) • Single characteristic clinical attack of CNS demyelination: – Optic neuritis – Acute partial myelitis – Brain stem syndrome – Cortical • Clinically Isolated Syndrome (CIS) • MRI: – Low risk: 1 or no other asymptomatic brain lesion – High risk: 2 or > asymptomatic lesions • Treatment approved for high risk patients – IFN-B, GA reduces second attack: ARR 15% Baseline MRI and Risk of CDMS for Monofocal onset CIS (BENEFIT Placebo N=93) CHAMPS CHAMPS2 ETOMS BENEFIT25 Unifocal/multifocal/ No pt Unifocal/ 383 Unifocal Unifocal+ multifocal /309 Unifocal/ 487 IFN-B Avonex+Pulse MP Avonex Rebif Betasron Dose/ 30µg/im/weekly 30µg/IM/ weekly 22µg/SC/ Weekly 250µg/SC/EOD Duration/years 3 5 2 2 Pre-MRI T2 load 2or> 4 or > 2 OR> %CDMS,P value 35 VS50, p=0.002 34 VS 45 P=0.047 28 VS 45 (69%/85% +MRI effect Yes Yes, P=0.001 Yes, P=0.001 36 VS 49, P=0.03 TOPIC Key inclusion criteria: • Patients 18 to 55 years of age with a first acute/subacute neurologic event consistent with demyelination. • MS symptom onset within 90 days of randomization. • Screening MRI scan with 2 T2 lesions 3 mM diameter that are characteristic of MS. Primary end point: Conversion to CDMS (as defined by the occurrence of a relapse) Placebo (n=197) Screened (N=846) R Randomized n=618 Teriflunomide 7 mg (n=205) Teriflunomide 14 mg (n=216) 108-Week Treatment Phase Miller A. Plattform presentation ECTRIMS 2013 Long-Term Extension Primary / Key Secondary Endpoint Primary Endpoint: Time to Clinically Definite MS (CDMS) Gd-enhancing T1 Lesions) 21% p=0.43 66 59% p=0.00 08 43% Safety / Tolerability: Adverse events observed in the trial were consistent with previous clinical trials with Aubagio. Miller A. Plattform presentation ECTRIMS 2013 CIS: When To Initiate Therapy? • Patients with normal MRI or with fewer than 2 – Low risk of developing early clinical attacks – Clinical and MRI monitoring – Without immediately commencing immunotherapy (DMT) • Those with abnormal MRI with2or> lesions consistent with MS or with evidence of intrathecal synthesis of antibodies should be considered for DMT, • Patients with atypical clinical or MRI presentation require further diagnostic evaluation. Relapsing-Remitting MS • Subacute repeated onset of CNS dysfunction with resolution ( sometimes incomplete , over days to weeks) • Revised McDonald criteria • MRI: Periventricular, brainstem, juxtacortical prominent T2, often Gad enhancing lesions, T1 hypointense (black holes) • Treatment: Interferon-B, Glatiramer acetate, natalizumab, mitoxantrone Features Consistent With MS • • • • • • • Relapses and remissions Age Onset between ages 15 and 50 Optic neuritis Lhermitte's sign Internuclear ophthalmoplegia Fatigue Uhthoff's phenomenon Features Inconsistent With MS • Steady progression • Onset before age 10 or after age 50 • Cortical deficits such as aphasia, apraxia, alexia, neglect • Rigidity, sustained dystonia • Convulsions • Early dementia • Deficit developing within minutes Secondary Progressive MS • Majority of RRMS many years following onset • Progressive impairment (spastic gait disturbance) between or in absence of attacks • No clear effect of DMT without ongoing attacks or inflammation • Role of DMTs in SPMS patients: – with ongoing relapses – Substantial ongoing accrual on new MRI inflammatory lesions Primary Progressive MS • Presents with progressive myelopathic gait, cerebellar ataxia or cognitive impairment without clear history of any clinical attacks • Clinical progression must be for at least 1 year and accompanied by a combinstion of brain&spinal abnormalities and/or CSF anormalities consistent with MS • Lack of clinical attacks/ relative paucity of MRI lesions • No approved DMTs Multiple Sclerosis (MS) • Multiple Sclerosis is the commonest disabling neurological condition to afflict young adults • MS is an autoimmune disease triggered by environmental agents acting in a genetically susceptible people • Auto-aggresive autoimmune attack on the myelin sheath and other components of CNS • Current&emerging DMTs are based in the above paradigm • Is MS a primary neurodegenerative disease MS: Pathology MS: Pathology Demographic Characteristics of Multiple Sclerosis in Kuwait Total recruited patients in study: 195 Gender Distribution N(M/F): 195(76/119) Cross sectional or retrospectively included patients:134 Years 35 SD±10.3 30 SD±9.3 25 20 15 10 Newly diagnosed drug naïve patient:65 SD±5.4 5 0 Mean Current age Mean age at presentation Mean duration of Disease Clinical Characteristics of Multiple Sclerosis in Kuwaiti Population 25.00% 20.50% 20.00% 15.00% 11.70% 10.00% 9.40% 8.60% 5.00% 7.30% 2.10% 1.50% 1.20% 0.60% 0.00% Sensory Brain stem and cerebeller Visual Motor Multiple Sphicter L' Hermitte Seizures Symptoms disturbance sign Presenting symptoms Others PRESENTING SYMPTOMS IN MS Total % SENSORY LOSS IN LIMBS 30.7 VISUAL LOSS 15.9 MOTOR WEAKNESS 14.2 DIPLOPIA 6.8 GAIT DISTURBANCE 4.8 NCOORDINATION 2.9 SENSORY LOSS-FACE 2.8 LHERMITTE’S 1.8 VERTIGO 1.7 BLADDER SYMPTOMS 1 AUTE TRANSVERSE MYELOPATHY 0.7 PAIN 0.5 OTHERS 2.5 POLYSYMPTOMATIC 13.7 Medication details in studied Kuwaiti MS patients 60.00% 50.00% 48.90% 40.00% 30.00% 20.00% 10.00% 0.00% 15.40% 8.70% 9.70% 3.10% 0.50% 0.50% 1% 0.50% 2.60% MS Therapy: Deciding on which Medication • Determine Therapeutic Goals – To reduce clinical relapse – To reduce accumulation of new MRI lesions – new T2 lesions – Gadolinium-enhancing lesions – black holes – Brain and spinal cord atrophy • Reduce short-term relapse related disability How To Determine of The Goals are Met? • Compare with baseline relapse rate – Recall bias – Regression to the mean • Assessment of improvement or stability in neurological impairment – Assess functional ambulatory limitation • May indicate progression • MRI ongoing/new inflammatory activity – Serial MRI to assess radiologic stability, worsening or improvement- q12-24 month except How To Determine of The Goals are Met? • If goals of DMT or symptomatic treatment are being met no change in DMT unless problems with medication tolerability • A detailed evaluation of common and idiopathic side effects will be required – Switching of medication based on adherence and tolerability ma be needed What if Goals are not being Met • If pre-therapy relapse rate is not improved – A therapeutic switch may be indicated • Relapse rate is incomplete indicator of ongoing inflammatory disease activity – Cranial and spinal MRI • May show therapy resistant inflammatory disease • Guide switch to a more potent anti-inflammatory medication • Clinical attack or definitive worsening disability is may lacking Case 2 • Mr. A.M.J is a 33 years old Kuwaiti male, diagnosed to have MS in 2008. • In Jan 2008, he developed diplopia, followed by paresthesia in feet, ascending to abdomen, chest and forearms. • These symptoms persisted • By June 2008, he was ataxic and on a wheel chair, when he sought medical advice • MRI was consistent with MS • Marked imrovement was noted in sensory symptoms after pulse steroids. Case 2 • His symptoms showed a rapid progression, by Sept 2008, he had optic neuritis, sphincteric disturbance, and positive Lhermitte’s sign. • In Oct 2008, he started to take Rebif. • His disease remained stable, with no new relapses and no new lesions on MRI till 2011. • In April 2011, he went for CCSVI treatment and discontinued Rebif without our knowledge or advice. CASE 1 • • • • • • • • Lhermitte’s sign was positive Cranial nerves were normal No motor weakness Mild sensory deficit for light touch and vibration on left side Plantars were flexor bilaterally Romberg’s sign was mildly positive Moderate left sided dysmetria, with tandem ataxia EDSS :2; AI :1. Case 1: MRI Cervical spineTWI Case 2 • • • • • In August 2011 he reported dizziness, ataxia and diplopia He was treated with pulse steroids with marked recovery. He was clinically stable, and was advised to restart Rebif. In Jan 2012, EDSS:1, AI:0. MRI in June 2012 showed new cerebellar lesions, with no enhancement. • In Oct 2012, he came in with a mild relapse and was treated with pulse steroids. • An MRI in Dec 2012 showed worsening lesion load, and he was advised to start Tysabri after JCV serology. • He started Tysabri in Dec 2012, and till 5 months post Tysabri , there were no active lesions. Case 2 • In Sept 2013, patient came with another severe relapse , with homonymous hemianopia, sphincteric problems, gait ataxia, and sensory disturbance. • Treated with pulse steroids with partial improvement in urinary symptoms and ataxia, but not in visual symptoms. Case 2: MRI Brain 2 Case 2: MRI Cervical spine 2 Case 2 • MRI showed marked worsening, with tumefactive enhancing lesions • A CSF study was done, which was normal, negative for JCV. • Considering this as a failure of Tysabri, it is planned to treat him with Rituximab Is Clinical Worsening due to Attack related Disease or Progression? • If it is due to non-inflammatory MS progressive disease – Neurodegenerative MS – ?subclinical ( and sub-radiologic) inflammation unresponsive to current DMTs – Switching to alternative MS therapy is futile • Escalating therapy If clinical impairment is strongly associated with ongoing relapses or marked new inflammatory MRI activity Existing & Emerging MS therapies Other Phase I CS-0777 BIIB033 Lymphocyte trafficking Phase II Idebenone Phase III Interferons Firategrast ONO-4641 Siponimod Ponesimod Marketed Peg IFNb (BIIB017) Extavia Azathioprine BG12 Novantrone Teriflunomide Copaxone generics x2 LV Copaxone Pixantrone ATX-MS-1467 PI2301 Vaccine, tolerization GRC4039 Laquinimod AIN457 Copaxone Cladribine CCX-140 Immune regulation Daclizumab Sativex Ampyra IPX-056 Alemtuzumab Ocrelizumab Ofatumumab LY-2127399 RTL1000 AZD5904 Fingolimod Tysabri Avonex Rebif Betaferon Antiproliferative agents ELND-002 NI-0801 RPI-78M Nerispirdine Symptomatic Tx Belimumab = Oral administration Cytolytic mAbs Modified from P. Vermersch = Injectable The Changing Landscape of MS Disease Modifying Treatment Of Approved and Emerging Therapies IFNβ-1a IM qwk Teriflun PO qd Alemtuz IV IFNβ-1a SC tiw IFNβ-1b SC qod GA SC qd Mitox IV q 90 d wks BG-12 PO bid Natalizumab IV q 4 wks Fingolimod 0.5 mg gd Daclizumab SC Laquin PO How are MS medication is selected? • Injectable interferon-β and glatiramer acetate remain the first line DMT for many clinicians – Their side effects are manageable with minimum of serious side effects • First line DMTs are effective in reducing clinical attacks and new MRI lesions Relapsing inflammatory MS clinical course Severe relapsing First line? inflammatory MS/JCV First line negative Injectable therapies Interferon β Parallel switch Glatiramer Inadequate response/inj intolerance Inadequate response/oral intolerance Oral therapies Consider side effects BG 12 Fingolimod Terflunomide Inadequate response/JCV negative Natalizumab Drawback of injectable Medication • Interferon-β – “Flue-like illness” often transient – Liver enzyme monitoring – Rarely depression • Glatiramer acetate – Flushing, eosinophilia, rare allergic reaction, injection-site reactions (skin liopatrophy) – Conbination therapy+interferon-β1a IM/weekly and glatiramer acetate does not appear to be significantly more efficacious than monotherapy Lublin FD et al, Ann Neurology 2013 BG-12 Phase III trials BG-12 Integrated analysis • Compared with PBO, BG-12 240 mg BID and TID significantly reduced ARR, risk of relapse, adjusted ARR requiring steroids, disability progression, and MRI outcomes. • Demonstrated consistent benefits on clinical efficacy across prespecified subgroups of RRMS pts with varied baseline demographics and disease characteristics • Overall incidence of AEs, SAEs, and discontinuations due to AES similar across tx groups; flushing and GI events most common AEs Nrf2 Pathway May Induce a Cytoprotective Response and Inhibit NFkB Mediated Inflammation BG-12 Nrf2 - Detox Enzymes - Antioxidant Enzymes - NADPH Generating Enzymes - GSH Biosynthesis Enzymes - Chaperones - Ubiquitination/Proteasome Cell and Tissue Protection NFkB - Proinflammatory cytokines - Leukocyte adhesion molecules - Lymphocyte activation Inflammation, Tissue Damage DEFINE Trial BG12 240mg bid vs tid: Primary endpoint - Relapses p<0.0001 41.3% p<0.0001 52.7% DEFINE: MRI Placebo BG12 bid BG12 tid p<0.0001 85% p<0.0001 90% BG-12 (dimethyl fumarate, DMF): CONFIRM — Annualized Relapse Rate Daclizumab • Anti-CD25 mAb Daclizumab • Effects similar in patients with highly active MS compared with pts with less aggressive disease prior to tx initiation • Treatment resulted in significant increase in number of pts who were disease- activity free following 1 year of tx in SELECT trial • Patients had reductions in % change in volume of T1-hypointense and T2-hyperintense lesions over 52 wks of treatment vs increases in PBO group S1P receptor modulators/ agonists • In phase 2b study, ponesimod significantly reduced inflammatory MRI activity at all doses tested, with a significant dose response; lower ARR also observed with ponesimod compared with PBO, and was generally well tolerated • Primary endpoint met in phase 2 DreaMS trial: ONO-4641 demonstrated significant efficacy on all key MRI measures of disease activity at all 3 doses compared with PBO, and was generally • well tolerated Compared with PBO, reduction in mean CUAL observed as early as • month 1 for siponimod 0.25 mg/day and 2.0 mg/day, and in all doses at month 2 in phase 2 BOLD trial; effect maintained at each month up to month 6; similar pattern for new/enlarging T2 lesions for all siponimod doses at month 2 and maintained at each month up to month 613 Glatiramer acetate • Compared with PBO, GA 40 mg SC TIW significantly reduced: • ARR by 34.4.% (P < .0001) Cumulative # GdE lesions by 44.8% (P < .0001) Cumulative # new/enlarging T2 lesions by 34.7% • (P < .0001) Safety profile consistent with GA 20 mg/day SC Teriflunomide Pyrimidine Synthesis Inhibitor (antimetabolite) Teriflunomide • Compared with PBO, 7 mg/day and 14 mg/day teriflunomide significantly reduced ARR by 22.3% and 36.3%, respectively (P = .0183 and P = .0001, respectively) • Compared with PBO, 14 mg/day teriflunomide significantly reduced 12-wk CDP (HR = .685; P = .0442) • Both teriflunomide doses generally well tolerated; safety profile consistent with prior studies • Update from TEMSO trial Mean reductions in lymphocyte and neutrophil observed in TEMSO were small in magnitude and were reversible after treatment discontinuation or on treatment in some cases; no other clinically significant complications to blood cytopenias reported TEMSO: Relapse Rate Teriflunomide Placebo 0.539 7 mg 0.370 RRR: 31.2% p=0.0002 14 mg 0.369 RRR: 31.5% p=0.0005 0 0.1 0.2 0.3 0.4 0.5 0.6 Adjusteda annualized relapse rate a)Adjusted for Expanded Disability Status Scale (EDSS) score strata at baseline and takes duration of treatment into account .ARR, annualised relapse rate; RRR, relative risk reduction TEMSO: EDSS progression (3 month confirmed) Disability progression (%) 40 Placebo 7 mg teriflunomide 14 mg teriflunomide 30 27.3% 21.7% 20.2% 20 Placebo vs 7 mg: HRR 23.7% p=0.0835 10 Placebo vs 14 mg: HRR 29.8% p=0.0279 0 0 Number at risk Placebo 363 7 mg teriflunomide 365 14 mg teriflunomide 358 12 24 36 48 60 72 84 96 108 224 238 234 211 234 227 200 224 217 160 178 175 Week 336 343 329 306 309 302 279 290 285 258 266 262 242 252 251 TENERE: Annualized relapse rate • The ARR in the 14 mg teriflunomide group was not statistically different from the ARR in the Rebif® group • The estimated ARR0.216 was higher in the 7mg Rebif® N=104 treatment group Teriflunomide14 mg N=109 0.259 Teriflunomide 7 mg N=111 0.410 0 0.1 0.2 0.3 Annualized Relapse Rate Genzyme, Press release, Cambridge, MA – December 20, 2011 0.4 0.5 FINGOLIMOD Sphingosine-1Phosphate (S1P) Receptor Agonist Fingolimod • Treatment with fingolimod 0.5 mg: – Significant benefits on relapse-related outcomes within first 3 months and on volume loss over 6 months compared with PBO in FREEDOMS and FREEDOMS II studies; concordant results from 2 large phase 3 trials, along with phase 2 data, allow better definition of expectations regarding time lag between initiation and effects of fingolimod treatment • Fingolimod treatment initiation effects in pooled population from FREEDOMS, FREEDOMS II (vs PBO), and TRANSFORMS (vs IM IFN à1a) a transient, mostly asymptomatic decrease in heart rate; symptomatic bradycardia and Mobitz I and 2:1 AVBs were dosedependent; AVB first occurrences most common <6 h post-dose5 • Analysis of TRANSFORMS trial demonstrated advantage of switching to fingolimod over remaining on IFN à-1a IM with regard to time to relapse in RRMS6 Fingolimod: Mechanism of Action Multiple sclerosis S1P receptor FTY720 traps circulating lymphocytes in peripheral lymph nodes T-cell FTY720-P LN Prevents T-cell invasion of central nervous system FTY720 Sphingosine-1-phosphate (S1P) receptor modulator Internalises S1P1, blocks lymphocyte egress from lymph node (LN) while sparing immune surveillance by peripheral memory T-cells FREEDOMS (Fingolimod) Annualized Relapse Rate 0.4 Annualised relapse rate 0.40 -54% vs placebo p < 0.001 -60% vs placebo p < 0.001 0.3 0.2 0.18 0.16 0.1 0.0 Placebo (n = 418) Fingolimod 0.5 mg (n = 425) Fingolimod 1.25 mg (n = 429) ITT population; negative binomial regression model adjusted for treatment group, country, number of relapses in previous 2 years and baseline Expanded Disability Status Scale (EDSS) as covariates FREEDOMS (Fingolimod) MRI Lesion Activity # new/enlarging T2 lesions at month 24 from baseline* p < 0.001 1 Mean (SD) lesion number 10 8 9.8 (13.2) 1.1 (2.4) 0.8 6 0.6 4 0.4 2 p < 0.001 p < 0.001 1.2 p < 0.001 12 # T1 Gd+ lesions at month 24** 2.5 (7.2) 2.5 (5.5) 0 0.2 0.2 (0.8) 0.2 (1.1) 0 Placebo (n = 339) Fingolimod 0.5 mg (n = 370) Fingolimod 1.25 mg (n = 337 ) Placebo (n = 332) Fingolimod 0.5 mg (n = 369) *Analysis performed using a negative binomial regression model adjusted for treatment group and country **Analysis performed using rank ANCOVA adjusted for treatment group, country and number of lesions at baseline Gd+, gadolinium-enhancing; MRI, magnetic resonance imaging Fingolimod 1.25 mg (n = 343 ) FREEDOMS (Fingolimod) Disability (Disability) Progression Fingolimod 1.25 mg vs placebo, HR = 0.68, p = 0.012 Patients with 3-month confirmed EDSS progression (%) 30 Fingolimod 0.5 mg vs placebo, HR = 0.70, p = 0.026 Placebo 25 20 Fingolimod 0.5 mg 15 Fingolimod 1.25 mg 10 5 0 0 90 180 270 360 Days on study HR, hazard ratio 450 540 630 720