Experimental treatment

The Partners Multiple Sclerosis Center at Brigham and Women’s Hospital has joined BrainStorm Cell Therapeutics in a Phase 2 trial exploring the safety and efficacy of NurOwn in the treatment of progressive multiple sclerosis (MS). This is the fifth and final clinical site participating in…

Mount Sinai Medical Center has joined with BrainStorm Cell Therapeutics to explore the safety and efficacy of NurOwn as a potential treatment for progressive multiple sclerosis (MS) in an ongoing Phase 2 trial. The New York center is the fourth clinical site participating in the trial, in addition…

A single dose of CD45-ADC, an investigational targeted therapy being developed to treat different types of autoimmune diseases, is enough to reset the normal function of the body’s immune system in a mouse model of multiple sclerosis (MS), and to delay onset of the disease. Those findings…

SARM1 inhibitors are a potential oral treatment to slow disease progression in neurodegenerative diseases like multiple sclerosis (MS), according to preclinical results that show the inhibitors protect nerve cells from damage in mice and cell cultures. Researchers at Disarm Therapeutics presented the findings in a poster titled “…

People with progressive forms of multiple sclerosis (MS) and past exposure to the Epstein-Barr virus are responding to a potential immunotherapy known as ATA188, tolerating the treatment well and with signs that suggest effectiveness, early updated data from an ongoing Phase 1 trial show. The research, “…

Eliminating SARM1, an enzyme that plays a key role in nerve cell degeneration, protects neurons in mice with experimental autoimmune encephalomyelitis (EAE), a condition that mimics the key pathological features of multiple sclerosis (MS) in humans. The preclinical findings were presented by researchers at Disarm Therapeutics in a…

A new study demonstrates that intracellular sigma peptide (ISP) can promote remyelination in a mouse model of multiple sclerosis (MS). Importantly, this study was independent of NervGen, a pharmaceutical company that is developing an ISP-like compound — NVG-291 — for the treatment of nerve injury and MS. The study,…

The Phase 2 clinical trial testing BrainStorm Cell Therapeutic’s investigational NurOwn therapy for progressive multiple sclerosis (MS) has added a third clinical site, the company announced. The trial (NCT03799718) now will enroll patients at the Keck School of Medicine of The University of Southern California (USC), and its academic…

An experimental treatment known as OB-002, that works to block an inflammatory molecule in the brain, prevented the development of lesions there after an early-in-life viral infection in a mouse model of multiple sclerosis (MS). The research “Brain-resident memory T cells generated early in life…

EHP-101, a cannabidiol-based experimental therapy for multiple sclerosis, was able to recover myelin in damaged nerve fibers and lessen neuroinflammation in a mouse model of MS, data show. Emerald Health Therapeutics, the manufacturer of EHP-101, also announced plans to open Phase 2 clinical trials in patients by…

A protein leads to nerve fiber and myelin damage, particularly in progressive forms of multiple sclerosis, by activating brain immune cells called microglia, according to a new study. Its researchers also noted this protein is the target of experimental MS treatment called temelimab (GNbAC1), which showed potential in Phase 2 clinical trials. The…

Cyxone submitted an application to the Netherlands research ethics committee to start the first clinical trial in humans testing T20K, its investigational preventive treatment for multiple sclerosis (MS). Approval by the Netherlands regulating authority…

Early, positive safety results from a Phase 1 trial testing a potential immunotherapy, ATA188, in people with progressive multiple sclerosis will be detailed at the 5th Congress of the European Academy of Neurology (EAN) late next month, its developer, Atara Biotherapeutics, announced. ATA188 is an investigational and “off-the-shelf”…

Researchers have developed a compound based on the thyroid hormone T3 that is able to repair damaged myelin in the brain of mice, a discovery that holds promise for healing myelin loss in patients with multiple sclerosis (MS), results of an early study reveal.

The first patient has been enrolled in a Phase 2 multicenter clinical trial testing the use of NurOwn cellular therapy to treat progressive multiple sclerosis (MS), BrainStorm Cell Therapeutics announced. The open-label trial (NCT03799718), titled Safety and Efficacy of Repeated Administration of neuron (MSC-NTF Cells) nin Participants…

A clinical trial to test the effects of antioxidant treatment with lipoic acid on progressive forms of multiple sclerosis (MS) is recruiting 118 participants at seven sites in North America. The study sites include: Birmingham, Alabama; Burlington, Vermont;  Portland, Oregon; Salt Lake City, Utah; Seattle, Washington; Washington, D.C., and;…

A request for a potential cannabis-based treatment for multiple sclerosis to be given Fast Track designation, speeding its development as it readies to enter clinical testing, is now before the U.S. Food and Drug Administration (FDA), MMJ International Holdings announced. MMJ-001, as this lead candidate is known, aims to treat…

Treating a common animal model of multiple sclerosis (MS) with a typhoid vaccine eased disease symptoms by prompting T helper cells to stop production of a pro-inflammatory factor — interleukin (IL)-17 — and by promoting greater numbers of anti-inflammatory  regulatory T-cells, researchers report. Their study, “Targeting prohibitins at the…

The U.S. Food and Drug Administration (FDA) has approved BrainStorm Cell Therapeutics‘ request to open a Phase 2 clinical trial testing the safety and effectiveness of its proprietary NurOwn mesenchymal stem cell (MSC) treatment in progressive multiple sclerosis (MS) patients. The request was in the form of Investigational New Drug…

Inactivation of S1PR2, a cell surface protein, helps improve clinical disability and reduce demyelination in a mouse model of experimental autoimmune encephalitis (EAE), a condition similar to multiple sclerosis (MS) in humans, a study shows. This finding suggests that therapies blocking S1PR2 could have the potential to treat MS. The…

Non-viral vehicles, called niosomes, can deliver DNA to glial cells in the central nervous system, a new study shows. The findings open new avenues for gene therapy and the treatment of neurological disorders, including multiple sclerosis (MS). The study “Non-viral vectors based on cationic niosomes as efficient gene delivery…

Homotaurine, a compound proven safe for humans in long-term clinical trials, has eased autoimmune responses, brain inflammation, and multiple sclerosis-like symptoms in a mouse model of the disease, a study has found. The findings represent proof-of-principle evidence that homotaurine may represent a new potential class…

Atara Biotherapeutics’ investigational ATA190, a cell therapy that wipes out immune B-cells infected with the Epstein-Barr virus (EBV), led to neurological improvements and reduced symptoms in patients with primary and secondary progressive multiple sclerosis (MS), a Phase 1 trial shows. The trial results were published in the Journal…

A Phase 3 clinical trial intending to confirm the potential of MD1003, a high-dose biotin, in treating progressive multiple sclerosis (MS) patients is completely enrolled, MedDay Pharmaceuticals, the investigational therapy’s developer, announced. The randomized, double-blind, and placebo-controlled study (NCT02936037), called SPI2, follows the previous Phase 3 trial (NCT02220933),…

Progressive multiple sclerosis patients — with primary or secondary progressive disease — treated with high doses of oral ibudilast 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…

MMJ Bioscience, which specializes in medical cannabis products, has filed a request with the U.S. Food and Drug Administration (FDA) to open Phase 2 clinical trials evaluating the company’s THC/CBD pharmaceutical compounds as possible treatments for symptoms of multiple sclerosis (MS). THC refers to the tetrahydrocannabinol compound, part of the…

Investigational therapy ibudilast leads to a significant reduction of brain atrophy, supporting its potential to effectively treat progressive multiple sclerosis (MS), new data from a Phase 2 clinical trial show. These results will be shared at the upcoming 2018 Annual Meeting of the American…

Atara Biotherapeutics has received a green light to enroll U.S. patients into a Phase 1 trial of ATA188 for progressive or relapsing-remitting multiple sclerosis (MS). The study was initially launched in Australia, but with the U.S. Food and Drug Administration (FDA) having cleared the company’s application, the trial…

American CryoStem has received a warning letter from the U.S. Food and Drug Administration (FDA) for marketing its adipose-derived stem cell product Atcell without required regulatory approval, and for "significant deviations" from manufacturing processes that potentially raise safety concerns. The company has 15 working days to respond to the concerns raised by the agency and detail how they will be corrected, or risk "enforcement actions," the FDA said in a Jan. 4 press release. Studies suggest that mesenchymal stem cells can be used to alleviate symptoms and possibly treat several degenerative disorders, including multiple sclerosis. Atcell is a therapy based on the ability of mesenchymal stem cells isolated from adipose tissue (fatty tissue) to transform into a subset of mature cells, which include adipose cells, bone cells, and cartilage cells. Although not approved for use, Atcell is being distributed directly to physicians to treat patients affected by several life-threatening diseases, including Parkinson’s disease, amyotrophic lateral sclerosis, stroke, and multiple sclerosis, the FDA said in its release. It is administered intravenously, intrathecally (injection or infusion into the central nervous system) or by aerosol inhalation. The product is designed to be used in the same individual (autologous use) the cells are collected from, an approach intended to reduce risk. The cells are extracted using the company’s proprietary Cellect collection system. They are then expanded in the laboratory using the company's ASCelerate SF-M serum free (animal-product free) media, providing compounds needed for the cells to survive and proliferate. Stem cells put through this process are  ready to be used as therapy or to be stored for future use. A recent FDA inspection found that Atcell’s manufacturing steps are not in line with current good manufacturing practice requirements. Specifically, the manipulation of the adipose tissue was more than "minimal," the FDA reported, changing "relevant characteristics" of the original tissue that could introduce contamination by microorganisms or product defects and represent a "risk of harm" to patients. Because of this manipulation, FDA review is required by law to ensure Atcell's safety and efficacy, the agency said. Evidence of an inadequately controlled manipulation environment, lack of control of components used in production, and insufficient and inadequately validated product testing were also reported. “The use of Atcell raises potential significant safety concerns, due in part to the fact that there is little basis on which to predict how the product will perform in a patient,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in the release. “In addition, this product may also cause harm to patients who may put their trust in an unproven therapy and make the decision to delay or discontinue medical treatments proven to be safe and effective,” Marks added. American CyroStem, based in New Jersey, did respond to observations raised by the FDA at the time of its inspection. But they were found inadequate to support Atcell’s marketing, and failed to acknowledge that FDA approval was required, either by filing a biological license or investigational new drug application. “As part of our comprehensive policy framework for the efficient development and regulation of cell-based regenerative medicine, we’re going to be stepping up enforcement activities against those who manufacture and market products in ways that put patients at risk,” said Scott Gottlieb, FDA commissioner. “We see great promise from the field of cell based regenerative medicine, but there are also novel risks,” Gottlieb added. Healthcare professionals and patients who have used Atcell are asked to report any adverse events related to the treatment using the FDA’s MedWatch Online Voluntary Reporting Form. Completed forms can be submitted online, or via fax to 1-800-FDA-0178.  

A Phase 3 clinical trial evaluating AB Science’s masitinib as a treatment for progressive multiple sclerosis can continue without having to add patients, an independent review board has decided. The decision indicates that the therapy has been effective enough that its population base does not need to be expanded, the…