Experimental treatment

A group of experts recently concluded that clinical trials are the best way to explore whether cell-based therapies are viable options for treating multiple sclerosis. In a newly published article, MS researchers reviewed evidence on a range of cell therapies, including stem cell transplants and delivery or stimulation of various cell types. Clinical trials, the panel argued, would be the optimal way to examine which types of cells should be used, how they should be delivered, and the types and disease stages the treatments are suitable for. The article focused on four types of cell-based treatments: autologous stem cell transplants, mesenchymal and related stem cell transplants, use of drugs to manipulate stem cells in the body to boost their ability to repair, and transplants of oligodendrocyte progenitor cells to trigger new myelin production. Loss of the myelin that protects neurons is a hallmark of MS. Such treatments hold promise to attain what current disease-modifying therapies in MS have not: halting the disease without lifelong treatment that has potential side effects, and regenerating damaged tissue. In addition to reviewing the evidence surrounding cell-based treatments, the expert group focused on the availability of the treatment options outside of controlled trials. “Media attention has resulted in some cases of misrepresentation and exaggeration of therapeutic claims for cell-based therapies for multiple sclerosis and other diseases,” the team wrote. This has caused patients to seek the treatments — paying out-of-pocket — at unregulated clinics. The panel noted that several drugs in development, including opicinumab, are aimed at promoting remyelination. In addition, drugs that are already approved for other conditions might have remyelinating properties, and might be repurposed to treat MS. Although studies are ongoing, the panel noted that it is unclear if the drugs do promote remyelination. Despite ongoing research and — in some cases — clinical use of cell-based therapies for MS, these treatments should be considered experimental, the expert group concluded. They again underscored the importance of clinical trials in providing a controlled environment for patients wishing to have cell therapy, as well as a source of evidence for the feasibility of these approaches.

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…

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 …

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.

Scientists at the University of Maryland have developed an experimental treatment to control the immune system and recover movement in a paralyzed mouse model of multiple sclerosis (MS). The team presented its research April 2 during the 253rd National Meeting & Exposition of the American Chemical Society in San Francisco. In…

Doesn’t time fly? It seems only yesterday that I was preparing to go to Moscow, yet tomorrow will be three weeks since my return. I have written enough about my experiences at the A.A. Maximov center so, today, we’ll take a look at how HSCT progresses, step by step. The…

The experimental therapy laquinimod was seen to prevent the start of harmful immune responses and to disrupt the progression of multiple sclerosis (MS) in a mouse model of the disease. This finding may be especially promising, as the treatment is being developed for people with both relapsing MS and its rarer progressive…

In a presentation at a U.S. Food and Drug Administration (FDA) public hearing earlier this month, the chief scientific officer of StemGenex Medical Group, Steven A. Brody, MD, PhD, said succinctly: “Stem cells have arrived and have captivated the scientific and medical communities. With this excitement comes responsibility and with this…

Researchers from Oryzon will present efficacy data on the company’s oral epigenetic drug ORY-2001, a potential treatment for multiple sclerosis (MS), at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) congress taking place this week in London. The poster presentation, “LSD1 inhibition, a potential epigenetic therapeutic approach for the…

Data from an extension phase of a Phase 3 clinical trial, given in an oral presentation by MedDay, reported that the biotin  MD1003 showed effectiveness over time as a possible treatment of non-active, progressive multiple sclerosis (MS). The data were presented at the recent 2nd Congress of the European Academy of Neurology (EAN) in Denmark by Professor Ayman Tourbah,…

MedDay SAS recently announced that it has raised €34 million, about $38.5 million, in a Series B financing round. The money will enable the company to lead a confirmatory Phase 3 clinical trial, called SPI2, in the United States to assess its lead candidate, MD1003, as a treatment for progressive multiple sclerosis (MS). MedDay concluded…

PJSC Pharmsynthez, a pharmaceutical company based in Russia, recently announced completed follow-up findings and data analysis from a Phase 2a proof-of-concept clinical trial of its novel therapeutic vaccine Xemys for the treatment of multiple sclerosis (MS). Xemys utilizes Xenetic Biosciences patented ImuXen technology. In the open-label, dose-escalating trial, 20 patients…

RedHill Biopharma, Ltd. recently announced promising interim results from its ongoing CEASE-MS Phase 2a clinical trial evaluating the safety and efficacy of a fixed dose of RHB-104 as an add-on therapy for the treatment of relapsing-remitting multiple sclerosis (RRMS). RHB-104 consists of an oral capsule formulation of an antibiotic combination therapy —…

MediciNova, Inc., announced that MN-166 (ibudilast) has been approved for “fast track” development by the U.S. Food and Drug Administration (FDA) as a potential treatment for progressive multiple sclerosis (MS). Progressive MS includes both the primary progressive (PPMS) and secondary progressive (SPMS) forms of the disease. MediciNova’s MN-166 was licensed from Kyorin Pharmaceuticals for its potential…

RedHill Biopharma, Ltd., an Israeli biopharmaceutical company focused on the development and commercialization of oral-administered small-molecule medicines for the treatment of inflammatory and gastrointestinal diseases, recently announced it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a new patent covering its…

Synthetic Biologics, Inc., a clinical stage company focused on the development of therapeutics to protect the microbiome and to target disease-causing pathogens, recently announced disappointing results from an independent third-party analysis of a Phase 2 clinical trial evaluating Trimesta as a treatment for relapsing-remitting multiple sclerosis (RRMS) in women.

RegeneRx Biopharmaceuticals, Inc., announced the publication of a research article detailing the process by which Thymosin beta 4 (Tβ4), the company’s novel therapeutic peptide, effectively promoted the remyelination process in two separate animal models commonly used for multiple sclerosis (MS) research. The article, “Thymosin beta4 promotes oligodendrogenesis in the demyelinating…

Recently, Biogen released results from its Phase 2 acute optic neuritis (AON) RENEW trial which tested Anti-LINGO-1. Learn more about this results here. So what is Anti-LINGO-1? According to the MS Society, Anti-LINGO-1 (also known as BIIB033) is a treatment in development by the pharmaceutical company Biogen which is currently…

GeNeuro, a company developing therapies for neurological and autoimmune disorders, recently announced the initiation of a Phase 2b clinical trial to assess its lead investigational antibody GNbAC1 in patients with relapsing-remitting multiple sclerosis (RRMS). The trial, called “CHANGE-MS,” plans to enroll 260 patients across 68 centers across the European Union and…

Synthetic Biologics, which specializes in the development of therapies for pathogen-specific diseases, recently announced the publication of new and positive data on results from a Phase 2 clinical trial evaluating the company’s product Trimesta™ as a treatment for relapsing-remitting multiple sclerosis (RRMS) in women. Professor Rhonda Voskuhl, director of the Multiple Sclerosis Program…

In a new study entitled “Untargeted plasma metabolomics identifies endogenous metabolite with drug-like properties in chronic animal model of multiple sclerosis,” a team of researchers performed a comparative analysis of metabolites between control mice and a mouse model with experimental autoimmune encephalitis (EAE, the most commonly used…

Roche announced positive results for three pivotal Phase III studies of experimental MS therapy ocrelizumab in relapsing multiple sclerosis and primary progressive multiple sclerosis (PPMS) patients at this year’s ECTRIMS 2015 conference. The results, particularly for treating PPMS, indicate that the novel therapy may represent…

Acorda Therapeutics, Inc., a company focused on therapies for neurological disorders, will present new data on its Phase 1 clinical trial evaluating rHIgM22, and five-year post-marketing safety data on AMPYRA® (dalfampridine) Extended Release Tablets (10 mg)  at this year’s 31st Congress of the European Committee for Treatment and Research in…

Progressive multiple sclerosis patients treated with AB Science’s lead compound AB07002 (masitinib) in a Phase 3 clinical trial showed positive results in a non futility test (a test to determine if an experimental therapy shows some sign of efficacy). With the successful completion of the futility analysis, AB Science is…

Nutra Pharma, a US-based biotechnology a company specializing in the acquisition, licensing, and commercialization of pharmaceutical products and technologies for the management of neurological disorders, cancer, autoimmune, and infectious diseases, recently announced that it has filed an application with the US Food and Drug Administration (FDA) for orphan drug status…

MediciNova, Inc., a biopharmaceutical company focused on acquiring and developing novel, small-molecule therapeutics for the treatment of diseases with unmet medical needs, recently announced that the ongoing clinical trial of MN-166 (ibudilast) in patients with progressive multiple sclerosis (progressive MS) has finished the randomization of 255 patients, exceeding the…

Researchers at Monash University and the MIMR-PHI Institute of Medical Research in Australia recently proposed that specific human stem cells with immunomodulatory properties represent a new promising therapeutic strategy for diseases like multiple sclerosis (MS). The study was published in the Journal of Neuroinflammation and…

Exploratory research conducted at Virginia Commonwealth University and the University of Illinois at Chicago may translate into a new therapeutic agent to treat progressive multiple sclerosis. Researchers in the laboratories of Dr. Jefferey L. Dupree and Dr. Douglas L. Feinstein tested a new compound in mice with induced multiple sclerosis…

While multiple sclerosis (MS) is considered a rare condition with major unmet medical needs, the treatments for progressive forms of MS are even scarcer, with no viable, FDA-approved drugs available to specifically address these forms of the disease. Because of this, the MS community is eagerly following a major…

MediciNova, Inc., a publicly-traded biopharmaceutical company developing novel, small-molecule therapeutics for the treatment of diseases with unmet medical needs, recently announced that the National Institute of Neurological Disorders and Stroke (NINDS) notified the company of full enrollment of their ongoing clinical study evaluating ibudilast (MN-166) for the treatment of progressive…