Experimental treatment

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Trethera Corporation’s experimental oral therapy TRE-515 for demyelinating optic neuritis, an eye condition that may progress to multiple sclerosis (MS) or occur during the disease course. The first-in-class therapy is expected to lessen the inflammation…

CNM-Au8, Clene Nanomedicine’s experimental oral therapy, safely and effectively improves vision and neurological function in adults with stable relapsing-remitting multiple sclerosis (RRMS) and disease-related visual impairment. Those are the findings of the VISIONARY-MS Phase 2 clinical trial (NCT03536559), a proof-of-concept study that investigated CNM-Au8 in people with…

EMBOLD Study of ATA188 in Progressive MS Is Given Go-ahead Is this another small step toward an MS cure? AT188 is an experimental therapy designed to kill cells infected with the Epstein-Barr virus (EBV). As you probably know, researchers have found a lot of evidence that EBV is connected…

Note: This story was updated July 20, 2022, to correct the headline to reflect that Soliris might be a potential treatment for progressive MS based on data from a single case report. A female patient with undiagnosed secondary progressive multiple sclerosis (SPMS) was placed on Soliris (eculizumab)…

An independent committee of experts has recommended that the Phase 2 portion of the EMBOLD clinical trial continue as planned without a sample size adjustment, following an analysis of safety and effectiveness data. The trial is testing Atara Biotherapeutics‘ experimental medication ATA188 in progressive forms of multiple…

Treatment with foralumab nasal spray resulted in marked clinical improvements after three months in the second secondary progressive multiple sclerosis (SPMS) patient treated under a single-patient access program. The findings were generally consistent with those seen in the first SPMS patient and supported a decision by the U.S. Food…

A Phase 1/2 clinical trial is evaluating the safety and effectiveness of a cutting-edge immunotherapy called extracorporeal photopheresis in people with multiple sclerosis (MS). The study, dubbed PHOMS (NCT05168384), is enrolling up to 45 adults with relapsing-remitting MS (RRMS) and secondary progressive MS (SPMS) at a…

A single infusion of FibroBiologics‘ investigational cell therapy, CYMS101, was able to inhibit disease activity in a mouse model of multiple sclerosis (MS), and appeared safe in early MS clinical trials, the company announced in a press release. Data from the mouse and human studies were…

A new scoring system might more precisely evaluate disease development, severity, and treatment response in a commonly used mouse model of multiple sclerosis (MS) known as the experimental autoimmune encephalomyelitis or EAE model, scientists report. This scoring system, called “I AM D EAE,” was described in the study, “…

ABX-002, an experimental treatment for multiple sclerosis (MS) being developed by Autobahn Therapeutics, promoted the restoration of the myelin sheath in preclinical models of the disease, new data show. The research, which specifically tested an analog of ABX-002 called LL-341070, will be presented by Autobahn’s academic collaborators at…

The experimental therapy XPro1595 was found to promote remyelination — the repair of the myelin sheath — in a mouse model of myelin loss, new data show. Myelin, the fatty covering that helps nerve fibers send electrical signals, is damaged by the immune system in …

Repeat harvesting of bone marrow cells and infusing them back into the bloodstream is safe and feasible in people with active progressive multiple sclerosis (MS), according to data from a small, proof-of-concept clinical trial. Results were detailed in the study, “Repeat infusion of autologous bone marrow…

A Phase 1/2 clinical trial evaluating Imcyse’s experimental therapy IMCY-0141 in people with relapsing-remitting multiple sclerosis (RRMS) has dosed its first patient. The trial, called IMCY-MS-001, is expected to enroll nearly 150 adults with RRMS; sites were not disclosed in a release by the Belgium-based company. Interim results may be available…

Based on findings from the first two secondary progressive multiple sclerosis (SPMS) patients given foralumab nasal spray, an experimental therapy, the U.S. Food and Drug Administration (FDA) has approved starting treatment in up to eight other patients under a special access program. Those enrolled in this intermediate-size expanded access…

NVG-291, an experimental therapy designed to promote nervous system repair, has so far been well tolerated in an ongoing Phase 1 study with healthy volunteers. “The Phase 1 study is progressing very well, and we are encouraged by the data we are seeing so far,” Daniel Mikol, MD, PhD, chief…

Six months of treatment with foralumab, an experimental nasal spray, safely and effectively improved motor and cognitive function while easing immune activation and inflammatory responses in a person with secondary progressive multiple sclerosis (SPMS), Tiziana Life Sciences, the therapy’s developer, announced. The patient — the first with MS to…

Masitinib, AB Science’s experimental oral therapy, significantly slows disability progression in adults with primary progressive multiple sclerosis (PPMS) and non-active secondary progressive MS (SPMS), according to final data from the AB07002 Phase 2b/3 clinical trial. These findings, indicating that the trial met its main goal, support…

Treatment with an autologous hematopoietic stem cell transplant (aHSCT) seems to reboot the immune system in multiple sclerosis (MS) patients for at least three years, a small study found. The study, “Sustained immunotolerance in multiple sclerosis after stem cell transplant,” was published in Annals of Clinical and…

Atara Biotherapeutics has reached an agreement with Fujifilm Diosynth Biotechnologies for the production of its cell therapy pipeline, including ATA188, an experimental therapy being investigated for the treatment of progressive multiple sclerosis (MS). Under the agreement, Fujifilm will acquire Atara’s T-Cell Operations and Manufacturing (ATOM) facility in Thousand…

Cyxone will work with a European organization to scale up production of the active ingredient in T20K, its experimental treatment for multiple sclerosis (MS), the company announced. The collaboration, which will cover the development of a new manufacturing method and later the…

The French Health Authority has approved AB Science’s request to launch a Phase 3 clinical trial to confirm the safety and effectiveness of its lead candidate masitinib in adults with primary progressive multiple sclerosis (PPMS) and nonactive secondary progressive MS (SPMS). “We are very excited to initiate…

Certain cell therapies that hold promise for treating autoimmune diseases like multiple sclerosis might work not because of the way the cells live in the body during treatment, but because of how they die. A better understanding of how these cell therapies work “is important in designing treatment protocols…

ImStem Biotechnology announced that the first patient in its Phase 1 clinical trial for IMS001, an investigational treatment for multiple sclerosis (MS), has been dosed at the Shepherd Center in Atlanta. The trial (NCT04956744) is recruiting participants with an MS diagnosis ages 18 to 65. More information…

Nerve Repair Therapy NVG-291 Safe, Well-tolerated in Healthy People This experimental therapy has a long way to go before it becomes reality. But it’s encouraging that this first step found NVG-291’s side effects to be mild and short-lasting. NVG-291 is designed to promote remyelination by modulating an enzyme called…

A single dose of NVG-291, a potential nerve repair therapy for multiple sclerosis (MS), was safe and well-tolerated among healthy volunteers in a Phase 1 safety trial, and was found to have favorable pharmacological properties, new data show. Given the positive results thus far, a safety committee supervising…

Extracellular vesicles or EVs — tiny sacs of material excreted by cells — that have been coated with protein receptors were found to safely reduce disease progression in a mouse model of multiple sclerosis (MS). Researchers at the Karolinska Institutet, in Sweden, coated these vesicles with receptors that block pro-inflammatory…

Treatment with evobrutinib, an experimental therapy for relapsing forms of multiple sclerosis (MS), reduced the size of inflammation-associated brain lesions in a Phase 2 clinical trial, data show. The oral medication was also found to be generally safe and well tolerated, according to an analysis of trial data in…

An investigational immunotherapy, ATA188 showed it could reverse disability and improve exercise capacity in people with progressive forms of multiple sclerosis (MS), Atara Biotherapeutics, its developer, reported in an update on a Phase 1 trial and its long-term extension study. EMBOLD trial findings based on MRI brain imaging…

NervGen Pharma announced that its Phase 1 clinical trial evaluating the safety and pharmacological properties of NVG-291 — its lead therapeutic candidate for multiple sclerosis (MS) and other neurologic conditions — in healthy volunteers is going according to plan, with early top-line data expected later this year. “The…

High-dose biotin aided walking speed in people with progressive multiple sclerosis after 12 to 15 months as an add-on treatment, an analysis of placebo-controlled clinical trials shows. However, the therapy failed to improve other measures of disability, and was associated with inaccurate lab test results caused by high levels…