Experimental treatment

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…

Clomipramine, an approved antidepressant, shows potential in treating people with progressive multiple sclerosis (MS)  — a disease form with few treatments — by protecting nerves from various processes thought to underly progressive MS, early research shows. The lab and animal study, which focused on already-approved treatments, was titled “Systematic…

A pregnancy hormone called estriol may be effective in controlling relapses in women with multiple sclerosis (MS), says a researcher at the University of California, Los Angeles (UCLA). In clinical trials, estriol has also lowered fatigue and improved thinking — work that originated in Rhonda Voskuhl’s quest to understand…

This is a special edition of Multiple Sclerosis News Today's daily Alexa Flash Briefing, covering the latest news from the 7th Joint ECTRIMS-ACTRIMS Meeting currently underway in Paris, France. The MS News today team in on-site at the conference, providing exclusive coverage of the presentations and speakers.

Atara Biotherapeutics recently published an update of the company’s quarterly financial results and operational highlights, including the advancement of its T-cell based immunotherapy strategies for multiple sclerosis (MS) and cancer. One of the investigational therapies featured in the report is ATA188, a potential treatment for MS.

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…