November 7, 2022 News by Lindsey Shapiro, PhD #ECTRIMS2022 ā ATA188 Could Be ‘Game Changing’ for Progressive MS In an early clinical trial, Atara Biotherapeuticsā investigational treatment ATA188 stabilized or eased disability in most people with nonactive, progressive forms of multiple sclerosis (MS) ā with those benefits now having been sustained for up to four years. For MS patients, in whom disability progressively accumulates over time,…
April 29, 2022 Columns by John Connor The MSer Who Lay in Bed for 2 Years and Can Now Walk Again My column’s handle is “Fall Down, Get Up Again” because the first piece I wrote for Multiple Sclerosis News Today was titled “A Mountain to Climb with MS ā in My Living Room.” That column got me this gig five years ago. It was set in 2012, mind you,…
December 10, 2021 Columns by John Connor An Interview With My Primary Carer Iāve been meaning to do this for a while. My wife, Jane, whoās also my primary MS carer, went away for a few days last week, and with absence making the heart grow fonder and all that, I thought Iād strike ā if she came back! Fortunately, she did,…
June 30, 2021 News by Marisa Wexler, MS Ublituximab Quite ‘Reassuring’ as Potential MS Therapy, Experts Say In clinical trials, the investigational anti-CD20 therapy ublituximab was the first to push the annualized relapse rate (ARR) below the 0.1 threshold among patients with relapsing forms of multiple sclerosis (MS), while improving disability outcomes in a significant proportion of patients. “The [relapse] rate was below a tenth…
September 1, 2020 News by Marisa Wexler, MS Kesimpta Meets Safety, Efficacy and Flexibility Needs of Relapsing MS, Experts Say Kesimpta (ofatumumab) is a safe and effective treatment for relapsing multiple sclerosis (MS), with an ease of delivery that makes it more convenient than comparable therapies, experts say. Developed and marketed by Novartis, Kesimpta was recently approved by the…
March 12, 2020 News by Marisa Wexler, MS Ponesimod, Up for Approval, Shows ‘Clear Superiority’ Over Aubagio, Janssen Exec Says PonesimodĀ may soon be a new oral therapy for people with relapsing multiple sclerosis (MS) in Europe, and a filing for its approval in the U.S. is likely within weeks. Janssen Pharmaceuticals, its developer,Ā submitted an applicationĀ to the European Medicines Agency (EMA) inĀ early March, a first ponesimod filing…
December 4, 2019 News by Mary Chapman National MS Society Establishes Partnership with Jon Strum’s ‘RealTalk MS’ Podcast When Jon Strum began his āRealTalk MSā podcast two years ago, he did not imagine he soon would have thousands of regular listeners in more than 60 countries. Now, Strum’s popular online audio series has a new partner: the National Multiple Sclerosis Society (NMSS). Strum uses his weekly…
September 20, 2019 News by Ana Pena PhD #ECTRIMS2019 – Ahead for Mavenclad: Fuller Understanding of What Makes It ‘Unique,’ Serono Exec Says in Interview Real-world data continues to support the safety and effectiveness ofĀ MavencladĀ (cladribineĀ tablets) in treating multiple sclerosis (MS), and several studies underway will help scientists gain in-depth understanding of how Mavenclad works, its impact on the immune system, and the durability of its benefits, an executive with EMD SeronoĀ said in an…
September 18, 2019 News by Ana Pena PhD #ECTRIMS2019 – Ozanimod’s ‘Key Advantages’ May Lead to New First-line MS Therapy: Interview with Neurologist Jeffrey Cohen Celgene‘s oralĀ ozanimod, if approved,Ā could be a first-line oral treatment option for people with relapsing multiple sclerosis (MS), and one with relatively minor side effects. Recent results from the Phase 3 trials RADIANCE (NCT02047734) and…
August 20, 2019 News by Ana Pena PhD Exploring Early Treatment Strategies for RRMS: An Interview with Neurologist Daniel Ontaneda When it comes to initial treatment selection for relapsing-remitting multiple sclerosis (RRMS), there is one question that has yet to be answered: Is it better to start with potentially safer moderately effective disease-modifying treatments (DMTs) or to hit the disease immediately with a high-efficacy DMT that may be…
May 15, 2019 News by Marisa Wexler, MS #AANAM – Biogen Offers Update on Development Plans for MS Therapies At the 2019 annual meeting of the American Academy of Neurology (AAN),Ā Multiple Sclerosis News TodayĀ sat down with Bernd Kieseier, MD, global head of multiple sclerosis at Biogen, to discuss the company’s portfolio, latest data, and therapeutic development plans in the field of multiple sclerosis (MS). Kieseier said…
May 15, 2019 News by Jose Marques Lopes, PhD #AANAM – 2 Trials Aim to Decide Best Early Way of Treating RRMS, Cleveland Clinic Doctor Says Two ongoing clinical trials may help doctors better understand which type of disease-modifying therapy ā those considered highly effective or those with low-to-moderate efficacy used in an escalating treatment approach ā would be best for people in the early stages of Ā relapsing-remitting multiple sclerosis (RRMS), according to a Ā …
April 8, 2019 News by Jose Marques Lopes, PhD Mayzent ‘Will Change Lives’ of MS Patients Transitioning to SPMS, Novartis Says The “regulatory environment” favoredĀ Mayzent (siponimod) beingĀ approved as an oral treatment for people withĀ relapsing multiple sclerosis (MS) ā specifically,Ā clinically isolated syndromeĀ (CIS),Ā relapsing-remitting multiple sclerosisĀ (RRMS), and activeĀ secondary progressive MSĀ (SPMS) ā a top executive with NovartisĀ said, althoughĀ the pharmaceutical company had requested a label covering all with SPMS. Dan…
April 3, 2019 News by Marisa Wexler, MS Cleveland Clinic Neurologist Applauds Mayzent’s FDA Approval, But Surprised by Those It May Not Treat When theĀ U.S. Food and Drug Administration approvedĀ the disease-modifying therapy Mayzent forĀ relapsing types of multiple sclerosis, itĀ specified in its label that the treatment was for people withĀ clinically isolated syndrome, relapsing-remitting MS, and ā importantly āĀ secondary progressive MSĀ provided they have "active" disease. The approval is good news, an MS researcher and physician saidĀ toĀ Multiple Sclerosis News TodayĀ in an interview, but "surprising" in that the FDA's decision was largely based on a trial that didn't involve CIS patients and wasn't focused on responses among particular types of SPMS. āIt's the first time that I've seen in the MSĀ field that regulatorsĀ made an approval designation ā activeĀ secondary progressive MS ā based on an underpowered subgroupĀ analysis,ā saidĀ Robert Fox, MD, a neurologist at the Mellen Center for Multiple Sclerosis at the Cleveland Clinic. Novartis'Ā medication, as a first oral therapy approved in the U.S. forĀ a form ofĀ SPMS,Ā is a big step forward in MS treatment, he said. But details of the FDA's decisionĀ caughtĀ him off guard. Fox served on the steering committee for the EXPAND Phase 3 clinical trial ,Ā on which the FDA decision was largely based.Ā His clinic was also one of the sites treating and evaluating patients in this pivotal study. Results of the EXPAND trial showed thatĀ Mayzent could reduce the risk of disability progression at three months (the trialās primary endpoint, or goal) by 21% in treatedĀ SPMS patients, compared to those given aĀ placebo. Among those with active SPMS (meaning with relapses), a 33% reduction was observed. The treatment, an S1P modulator that works in part to keep lymphocytes from entering the brain to trigger inflammation,Ā alsoĀ decreased the annualized relapse rate by 55% and improved cognitive processing speed in all treated patients.Ā āWhat was found, and I think quite clearly found in a large-size study, was that siponimod in patients with secondary progressive MS clearly slowed the progression of clinical disability over the course of the trial,ā Fox said. āIt's a statistical concept ā obviously patients either progress or they don't progress ā but on an overall basis there was a 21% slowing in the rate of progression of clinical disability.ā The FDAās decision is particularly important for SPMS patients. While Ocrevus (ocrelizumab) alsoĀ treats all relapsing MS forms and people with primary progressive disease (PPMS), it's an intravenous therapy given every six months. Mavenclad (cladribine), approved for relapsing patients in the U.S. just days after Mayzent, is another oral and active disease therapy. To Fox, Mayzent seemed to reach beyond only those secondary progressive patients with clinically active disease. āReally, this is the only drug that's been found to be effective in secondary progressive MS," he said. āTo that degree, it stands alone.ā That's why two points in the FDA's decisionĀ surprised him. The firstĀ is the label's specific mention of clinically isolated syndrome. CISĀ is defined asĀ theĀ first clinical presentationĀ of this diseaseĀ ā aĀ neurological episode that lasts at least 24 hours, and is characterized by inflammatory demyelination (the loss of myelin, the protective coat surrounding neurons). Ā For clinicians like Fox, CIS is a first manifestation of MS ā a kind of "mono sclerosis."Ā Since thereās only one documented attack, it canāt yet be considered multiple sclerosis, āas the multiple hasn't happened,ā Fox said, but many "in the field consider CIS to be ā¦ an early stage of MS." āIf the patient has a whole bunch of lesions on their brain [as seen on an MRI scan] and they had a single clinical event, ah, probably, they have MS,ā he said. Regulatory bodies like the FDA,Ā however,Ā have historically considered CIS to be its own separate entity. That makes this decision doubly surprising, according to Fox, since the EXPAND trial only enrolled patients with SPMS, not CIS. Ā āIt's the first time I've seen them approve for CIS specifically when there wasn't a trial in CIS,āĀ Fox said. āI agree with it ā I don't have a problem with it ā it just surprised me that the regulators were so progressive in their appreciation of MS.ā The second ā and far more unsettling ā surpriseĀ wasĀ the FDAās decision toĀ only approve Mayzent for āactiveā SPMS patients, instead of all SPMS patients. This decision didnāt come out of nowhere, he noted, but it remains puzzling in the context of the EXPAND trial.Ā InĀ compiling trial results, investigators did a subgroup analysis ā as they often do, almost as an aside for research reasons ā and found more favorableĀ responses to Mayzent treatmentĀ in patients with active inflammation beforeĀ the trial's start, those it determined to be with "active" disease. Ā āThere was a third of patients who had a relapse in the two years prior to enrollment, and those patients actually had a 30% slowing in disability progression, compared to the 21% overall,ā Fox said. This certainly does suggest that Mayzent can be more effective in people with active disease ā but there's a catch. The trial itself was not designed to make such a distinction. It enrolled SPMS patients regardless of activity, and its priority goal was changes in disease progression across all who were treated with Mayzent or given a placebo. Ā āWhat's important is that the trial was powered for the overall outcome. It was not powered for subgroup analysis,ā Fox said,Ā considering this a crucial point.Ā In clinical studies, being āpoweredā refers to theĀ enrollingĀ of whatever specific number of participants a study needs to ensureĀ itsĀ results will reach statistical significance. More people are redundant and, as such, an unnecessary cost; fewer could mean that trial's conclusions cannot be supported by rigorous scientific measures.Ā In other words, Fox said, the only conclusions that can be drawn from the EXPAND study reliably ā with rigor ā are based on data drawn from all its SPMSĀ patients, not aĀ subgroup with active disease. This trial āfollowed over 1,600 patients for the clinicalĀ disability. These are purposely powered so that you're not following twice as many people as you need toĀ ā¦ you're powered for that primary outcome,ā he said. āSo, how could they [the FDA] look at a subgroupĀ analysis and make an approval decision based on a subgroupĀ analysis that was underpowered?ā The neurologist gave as examplesĀ other subgroup differences found in trial analyses that didn't affect regulatory approval ā but to his mind, equally could have.Ā One was an analysis findingĀ female SPMS patients respondedĀ to the therapy better than males,Ā showing lesser disease progression. "So why didn't they just approve it for the females and not the males?" Fox asked. But, when asked, Fox did not think the labelĀ toĀ necessarily be an error. "My point is the absurdity of it," he said. "How could they make the regulatory approval based on a subgroupĀ analysis that wasn't powered for conclusions?" He was also particularly troubled becauseĀ the FDAĀ ādidn't define what āactiveā means āĀ is it just a relapse, or is it MRI disease activity?"Ā For many clinicians, āactiveā SPMS refers to ongoing inflammation that can be observed on MRI (magnetic resonance imaging) scans. In EXPAND, however, the active subgroup was defined as patients with clinical relapses within two years of being enrolled in the trial. Fox worries about this apparent lack of a regulatory definition of "active" SPMS, since āobviously, the insurance companies are going to seize upon that, and they're going to look for every way they can to avoid covering it for patients.ā Mayzent, Fox agreed,Ā is likely to be expensive. The therapy is reported to carry a U.S. list price ofĀ $88,500 a year. āI always have a concern about the cost of these drugs. They're all fearfully expensive,ā he said, noting he treats SPMS patients. His focus now is on working to ensure that possible regulatory and financial hurdles wonāt pose too much of an obstacle for patients, especially those with SPMS. āI don't know what the insurance companies are going to do with this, but I'm hoping that it is available for my patients, and I say that as their clinician,ā Fox concluded.
March 4, 2019 News by BioNews Staff #ACTRIMS2019 ā Jeffrey Cohen, MD, is New President of ACTRIMS Jeffrey Cohen, MD, director of the experimental therapeutics program at the Mellen Center for Multiple Sclerosis Treatment and ResearchĀ at the Cleveland Clinic, is the newly named Ā president of ACTRIMS, the Americas Committee for Treatment and Research in Multiple Sclerosis. Cohen’s appointment concluded the 2019 ACTRIMS ForumĀ that ran…
November 7, 2018 News by Jose Marques Lopes, PhD #ECTRIMS2018 – Better MS Monitoring and Disease Understanding Among Goals of FLOODLIGHT Open, Genentech Director Says The ability to track multiple sclerosis (MS) at any time, gain a more complete picture of each patientās disease course and a better understanding of MS are among the possible benefits of the FLOODLIGHT app, according to Laura Julian, PhD, principal medical science director at Genentech. The company…
October 24, 2018 News by Jose Marques Lopes, PhD #ECTRIMS2018 – Ocrevus Used Early in MS Course Key to Slowing Disability, Genentech Director Says Treating patients withĀ primary progressive or relapsing multiple sclerosis (MS) early with Ocrevus (ocrelizumab) is key to slowing disease progression, according to Hideki Garren, global head of Multiple Sclerosis and Neuroimmunology at Genentech. In an interview withĀ Multiple Sclerosis NewsĀ TodayĀ at the recentĀ 34thĀ congress of the European Committee for Treatment…
October 22, 2018 News by Jose Marques Lopes, PhD #ECTRIMS2018 – Finding Best Treatment for ‘Right Patient’ and Progressive MS Among Work of Interest, Cleveland Clinic Doctors Say Tailored, highly effective therapies early in the disease’s course may be a way forward in multiple sclerosis (MS)Ā treatment, according to Cleveland Clinic neurologist Robert Bermel. Another neurologist with the Cleveland Clinic,Ā Robert Fox, talked about potential and upcoming progressive MS treatments.Ā In interviews with Multiple Sclerosis News…
October 17, 2018 News by Jose Marques Lopes, PhD #ECTRIMS2018 – Serum Neurofilament Light Show Promise as Biomarker, But Not There Yet, Neurologists Say Despite its lack of specificity to multiple sclerosis (MS) in particular, monitoring patients with a blood biomarker ā serum neurofilament light (sNfL) ā may hold promise as a relatively easy way to assess treatment response and brain damage, according to MS experts. But there’s considerable work still to be…
October 16, 2018 News by Jose Marques Lopes, PhD #ECTRIMS2018 – Evobrutinib and Other Reasons for Hope in Pursuit of MS Treatments, Jerry Wolinsky Says in Interview Advances in multiple sclerosisĀ research and the development of new treatments over the last several decades give sustained reasons for hope as continue moving toward our future, according toĀ Jerry S. Wolinsky, a neurologist and MS specialist whose career spans more than 40 years. In a wide-ranging interview with Multiple…
October 16, 2018 News by BioNews Staff #ECTRIMS2018 – MS PATHS Just Starting to See Potential, Aims for Remission as Treatment Goal, Biogen Exec Says MS PATHS, a way of capturing data on disease progression and treatment response in thousands of multiple sclerosis (MS) patients being treated at any of 10 participating clinics, has among its goals that of making clinical remission ā a prolonged absence of any disease activity ā possible, said an…
October 15, 2018 News by BioNews Staff #ECTRIMS2018 – Post-hoc Analyses Support Safety and Efficacy of Mavenclad, Merck KGaA Says Substantial data supporting both the effectiveness and safety of Mavenclad (cladribine tablets) is before the U.S. Food and Drug Administration (FDA), and may lead to its approval as a short-course oral treatment for people with relapsing-remitting multiple sclerosis (RRMS) some seven years after a first such request…
October 15, 2018 News by BioNews Staff #ECTRIMS2018 ā As Siponimod Awaits FDA Decision, Mouse Work Helps in Understanding Benefits Seen in SPMS Mouse studies ofĀ siponimod ā a potential progressive multiple sclerosis (MS) treatment that’s up for approval in the U.S. and EU ā were among presentations given by NovartisĀ at the 34th European Committee for Treatment and Research in Multiple SclerosisĀ (ECTRIMS), held Oct. 10-12 in Berlin. Animal work might seem…
September 18, 2018 News by Jose Marques Lopes, PhD Ibudilast Slows Brain Shrinkage 48% in Progressive MS Patients in Phase 2 Trial; Lead Researcher Says Finding is ‘Remarkable’ Progressive multiple sclerosis patients ā with primary or secondary progressive disease ā treated with high doses of oralĀ ibudilastĀ in a Phase 2 clinical trial showed a 48 percent slowing in the progression of brain atrophy, or shrinkage, relative to those given a placebo, study data show. What this…
April 17, 2018 News by Larry Luxner 5 MS Patients Across US Talk About How Ocrevus Has Changed Their Lives Itās been a little over a year since U.S. regulators approved GenentechāsĀ Ocrevus (ocrelizumab)Ā as the first treatment for both relapsing and progressive forms of multiple sclerosis (MS) ā a disabling neurological diseaseĀ now believed to affect nearly one million Americans. While the juryās still outĀ regarding the therapyās…
March 28, 2018 News by Larry Luxner Ocrevus a Year After Approval: Views of Some MS Experts A year after U.S. regulators approved Genentech’sĀ Ocrevus (ocrelizumab)Ā as the first treatment for both the relapsing and progressive forms of multiple sclerosis, a prominent neurologist involved in the Phase 3 clinical trials that led to its authorization says it has been beneficial for some MS patients. But itās simply…
February 19, 2018 News by Patricia Inacio, PhD New Ocrevus Data, Post-FDA Approval, Supports Range of Benefits, Genentech’s Hideki Garren Says OcrevusĀ (ocrelizumab), GenentechāsĀ humanized anti-CD20 monoclonal antibody, continues to show clear evidence that it helps to slow disease progression and enable better function ā including in the hands and limbs ā ofĀ relapsing multiple sclerosis (MS)Ā and primary progressive multiple sclerosis (PPMS), latest data reveals. TheĀ first FDA-approved therapy ā in March…
November 21, 2017 News by Larry Luxner Patients Tell Their Stories in Consortium of Multiple Sclerosis Centers’ Online Videos Maryland special education teacher Ingrid Hanson says that since she was diagnosed with multiple sclerosis five years ago, sheās developed a better understanding of her students’ needs and the importance of teamwork. Flavia Nelson helps treat MS in the Hispanic community in Houston. Flavia Nelson, an MS specialist at…
November 20, 2017 News by Larry Luxner Nearly 1 Million Americans Have Multiple Sclerosis, NMSS Prevalence Study Finds An estimated 947,000 people in the U.S. have multiple sclerosis (MS) ā more than double the long-accepted figure of 400,000 ā according to a newly completed study organized and funded by the National Multiple Sclerosis Society (NMSS). āThis is definitely not what we expected,ā Ā Nicholas G. LaRocca, vice…
October 31, 2017 News by BioNews Staff #MSParis2017 ā Advances in Progressive and Pediatric MS Top ECTRIMS Highlights, NMSS Says A giant leap in research and interest in progressive multiple sclerosis ā from a few people pushing for such work to 2,000 listening to updates on it ā a successful first trial in children, and a growing body of potentially safer treatments for relapsing MS were among the highlights of…