Trial results

Advancells says its stem cell-based therapy completely reversed multiple sclerosis (MS) in an Indian pilot trial with only one MS patient. The patient, Rahul Gupta, was diagnosed with MS seven years ago and has since suffered multiple relapses. His disease was progressing fast and he was quickly losing his ability…

A new analysis of Phase 2 clinical data on Innate Immunotherapeutics’ investigational drug MIS416 to treat secondary progressive multiple sclerosis has confirmed that the drug failed to improve neuromuscular function or patient reported outcomes. The initial evaluation of data obtained from the one-year trial, announced in June, showed disappointing results. These results, gleaned from 70 patients who were randomly designated to receive either weekly injections of MIS416 or a placebo control, failed to demonstrate significant differences or clinically meaningful improvements in patients treated with MIS416 compared to those in the control group. After this initial setback, the Australian company sponsored an additional analysis of the trial results to identify any potential subgroup of clinical responders that could benefit from MIS416 and who might have been masked in the first population-based analysis. However, this post-hoc analysis also failed to show any positive effects of MIS416. Although the detailed report of this second analysis has not yet been released, the Sydney-based company conceded that the final outcome will not change. "All previous reports of MIS416 making a meaningful difference in the lives of many patients must either be dismissed as a very robust placebo effect or the trial failure is attributable to some other reason. It is my view that there may be other reasons," Innate CEO Simon Wilkinson said in a press release. "Patients with SPMS have a complex mix of symptoms and their disease can't be monitored by a simple blood test or MRI scan," he added. "We used the best assessment tools available as recommended by expert practitioners in MS, but we suspect they weren't sensitive enough to pick up the small but potentially significant changes that can lead to a substantial impact on patients' activities of daily living and quality of life." The lack of efficacy of MIS416 shown by the trial results is inconsistent with previous clinical experience, and the benefits reported by those receiving MIS416 for the past eight years.

First-line treatment with Copaxone (glatiramer acetate) benefits relapsing-remitting multiple sclerosis (RRMS) patients by boosting the number of anti-inflammatory immune cells and restoring the balance of regulatory immune cells, an Italian study shows. The study, “Biological activity of glatiramer acetate on Treg and anti-inflammatory monocytes persists for more than 10…

New analyses of how Merck’s Mavenclad (cladribine tablets) act to treat relapsing multiple sclerosis (MS) give researchers an entirely new picture of immune processes leading to the disease. Data showed that the drug lowers both immune B-cells and, to a lesser degree, T-cells. But the numbers of both cell…

Innate Immunotherapeutics' MIS416 has failed to help secondary progressive multiple sclerosis (SPMS) patients in a Phase 2 clinical trial. The company said it will continue testing the therapy, made up of natural compounds, to see if it can benefit any MS subgroups. Trial participants who received MIS416 had no meaningful improvements in neuromuscular function or the outcome of their disease, compared with those who took received a placebo. “It is disappointing that these results don’t show benefit for people with secondary progressive MS, for whom there are few treatment options,” Dr. Bruce Bebo, executive vice president of research at the National MS Society, said in a news release. Scientists hoped the injected therapy would modulate the activity of immune cells that affect the protective myelin coating around nerve cells, decreasing the inflammation and brain tissue damage associated with MS. Deterioration of the coating is a hallmark of the disease. The one-year trial (NCT02228213) tested the safety and effectiveness of MIS416 on 93 patients with SPMS in Australia and New Zealand. The patients randomly received MIS416 or a placebo once a week. There were no differences in the groups' scores on a disability index — the expanded disability status scale — or in brain volume changes detected by magnetic resonance imaging. In addition, there were no differences between in disease outcomes that patients reported. The self-reported barometers included the Multiple Sclerosis Impact Scale, the Neurological Fatigue Index, and the Brief Pain Inventory. "I am extremely disappointed by this outcome," Professor Pam McCombe, a principal trial investigator, said in a company press release. "Looking for measurable changes in patients with progressive MS using the assessment tools currently at our disposal is frustrating and complicated. We were hopeful that MIS416 would be an option to treat this group of patients who currently do not have effective treatment options." In addition to MIS416 failing to be effective, the group who received it had more treatment-related adverse events than the placebo group. The events were mainly related to the first dose, Innate said. The main problems were fever, chills, and muscle weakness. The company has been providing MIS416 to Australian MS patients under a compassionate use program. It said it will continue evaluating the safety and tolerability of the drug to see if it helps any subgroups of patients. Those findings will determine the future of the compassionate use program, it said. “These results are a shock, and definitely not what we were expecting based on our previous clinical experience with MIS416 and the reporting of treatment benefits we have received from many compassionate use patients over an extensive eight-year period," said Simon Wilkinson, Innate Immunotherapeutics' chief executive officer. "These data will be as distressing to them as they will be for all the stakeholders who were relying on the outcome of this study."

Ocrevus (ocrelizumab) significantly reduces disease activity and disability progression in patients with relapsing multiple sclerosis (MS) and primary progressive MS (PPMS), according to results of post-hoc analyses of Genentech’s Phase 3 clinical trial program assessing the drug.

Researchers managed to change the immune system — replacing inflammation with immune tolerance — in a mouse model of multiple sclerosis (MS) using so-called quantum dots, or nano-sized particles carrying pieces of myelin. Experiments with this advanced technological solution may help researchers design MS therapies that are based on promoting regulatory T-cells rather…

A high daily dose of simvastatin improves multiple sclerosis patients’ cognitive function, according to a new analysis of Phase 2 clinical trial results. The British team that did the research will start a study soon on whether simvastatin, which goes by the brand name Zocor and other labels, can also slow…

Researchers at Cincinnati Children’s Hospital Medical Center (CCHMC) have developed a new experimental method to specifically target unwanted activation of the immune system without the toxicity of current immunoregulatory drugs. According to the study “Manipulating DNA damage-response signaling for the treatment of immune-mediated diseases,” published in the journal …

Multiple sclerosis patients who benefited from Ocrevus (ocrelizumab) in two Phase 3 clinical trials continued to benefit when they extended their treatment, researchers reported. In fact, their annual relapse rates have fallen even further during the extension study than during the trials. The study, “Preliminary Results of the OPERA I and…

Long-term treatment for up to 12o weeks, with the investigational drug Ozanimod (RPC-1063), found to be effective and safe in patients with relapsing multiple sclerosis (MS) who participated in the RADIANCE clinical trial. Celgene, Ozanimod’s developer, presented the study, “Efficacy and Safety of Ozanimod in the Blinded Extension (120…

Cladribine tablets reduced relapsing multiple sclerosis patients’ annual relapse rate by 55 to 57 percent, depending on the dose, according to clinical trials. EMD Serono, a unit of Cladribine’s developer, Merck, presented the trial results at the 31st annual meeting of the Consortium of Multiple Sclerosis Centers in New Orleans,…

Multiple sclerosis (MS) patients who experienced a relapse between their first and second rounds of Lemtrada (alemtuzumab) had good treatment outcomes over the long run, according to a Phase 3 clinical trial. Those who relapsed after their first round ended up with annual relapse rates similar to those who didn’t…

Fampyra (prolonged-release fampridine tablets) — sold in the U.S. as Ampyra (dalfampridine) — has now been granted standard marketing authorization in Europe. The approval was based on the results of a Biogen-sponsored Phase 3 clinical trial confirming the drug’s safety and efficacy in improving walking in patients with multiple…

Relapsing multiple sclerosis (RMS) patients taking the investigational drug ozanimod, also known as RPC-1063, had lower relapse rate than those on weekly Avonex (interferon β-1a) therapy, according to Celgene in an announcement updating results of its Phase 3 RADIANCE trial. Ozanimod is a new orally administrated drug that selectively inhibits the…

A real-world medical-facilities setting has confirmed clinical trial findings that Gilenya (fingolimod) can reduce multiple sclerosis relapses, according to a Spanish study published in Plos One. Gilenya, developed by Novartis Pharmaceuticals, was the first oral disease-modifying therapy to obtain U.S. and European approval. The Food and Drug Administration and European Medicines Agency authorized…

The online BrainHQ adaptive training program developed by Posit Science is better than any computer game at helping multiple sclerosis (MS) patients improve their cognitive skills, according to a study by researchers at New York University (NYU). The study, “Cognitive function in multiple sclerosis improves with telerehabilitation: Results from…

The expression by immune B-cells of a protein called T-bet is crucial to promoting production of autoantibodies that recognize and destroy the tissues of one’s own body, finds a new study by researchers at National Jewish Health in Denver. The study, “B cells expressing the transcription factor T-bet drive lupus-like autoimmunity,”…

A global Phase 3 clinical trial assessing MD1003 — also known as high-dose biotin — for progressive multiple sclerosis (MS) might lead to the approval of one of the first treatments helping select progressive patients to improve. The trial aims to prove that high-dose biotin can reverse disability in non-active progressive MS.

A multiple sclerosis (MS) trial now underway in Colorado is assessing the safety and tolerability of switching from Rituxan (rituximab) to Ocrevus (ocrelizumab), and its lead investigator, Dr. Timothy L. Vollmer, largely expects no problems. The neurologist believes the two Genentech therapies — both antibody-based drugs that target the CD20 molecule on B-cells —…

Although a Phase 2b trial of the remyelination drug candidate opicinumab (also known as anti-LINGO-1 and BIIB033) failed to meet its primary goal of improving disability in relapsing and secondary progressive multiple sclerosis (MS), researchers believe the drug did cause “fairly strong” improvements. The trial evaluated four doses of the…

I don’t usually write about drug studies, especially ones that are tiny and preliminary. But an unexpected result has peaked my interest in this one. Researchers at The University of Queensland in Brisbane, Australia report that half of the progressive MS patients in their study of…

Siponimod slows the progression of multiple sclerosis patients’ disability, a Phase 3 clinical trial indicates. The therapy reduced the risk of disability progression in patients with secondary progressive multiple sclerosis (SPMS) by 21 percent over three months, researchers said. At six months, the reduction was 26 percent, they said. Researchers…

The latest results on Tecfidera (dimethyl fumarate) and Tysabri (natalizumab) use in a clinical practice setting suggest that early treatment can improve outcomes in multiple sclerosis (MS) patients. This and other recent data on Tecfidera and Tysabri for the treatment of MS will be presented by Biogen at the…

A ruling by the  U.S. District Court for the District of Delaware invalidated four of five patents held by Acorda Therapeutics that pertained to Ampyra (dalfampridine), a treatment for walking difficulties in multiple sclerosis (MS) patients, raising the possibility of generic forms of the drug coming onto the market in a…

Ampyra (dalfampridine) shows long-term efficacy in improving walking ability in people with multiple sclerosis, according to a study evaluating the treatment’s use in progressive and relapsing MS patients over two years. The study, “Monitoring long-term efficacy of fampridine in gait-impaired patients with multiple sclerosis,” was published in the…

Using a small RNA molecule belonging to the family of microRNAs (miRs), scientists could restore myelin in nerve cells and improve limb function in mouse models of human multiple sclerosis (MS). The study, “miR-219 Cooperates with miR-338 in Myelination and Promotes Myelin Repair in the CNS,” was published in…

Four newly published articles, the focused work of a group of researchers, evaluate the validity of four outcome measures commonly used in clinical trials of multiple sclerosis (MS) to set standards for such measures, the National MS Society recently reported. The effort by the Society-sponsored MS Outcome Assessments Consortium…

Liberation therapy was seen to be an ineffective treatment — both in the short and long term — in a Canadian study in people with multiple sclerosis (MS), its researchers reported, advising patients not to assume the procedure’s risks or cost. Lindsay Machan, a radiology professor at the University of British…

Australia’s Innate Immunotherapeutics confirmed that its Phase 2b clinical trial evaluating the drug MIS416 in patients with secondary progressive multiple sclerosis (SPMS) will wrap up by April 30, as scheduled, and data is expected to be release in the fall. MIS416 is a biologically derived new immune modulator that can…