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The SAGE Encyclopedia of Stem Cell Research


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and benefits of intravenous infusion of mesenchymal cells from autologous bone marrow in patients with MS.

       Sclerosis. Study designed to evaluate the safety and tolerability of intrathecal (drug injected directly into the spinal canal) administration of autologous mesenchymal stem cell–derived neural progenitor cells (MSC-NP) in patients with progressive MS.

       Investigating Mechanism of Action of DAC HYP in the Treatment of High-Inflammatory Multiple Sclerosis (MS). To investigate the mechanism of action and to assess long-term safety and efficacy of CD25-blocking therapies in HI-MS.

       Double Blind Placebo-Controlled Phase I/II Clinical Trial of Idebenone in Patients With Primary Progressive Multiple Sclerosis (IPPoMS). Idebenone is a drug that is similar to coenzyme Q10, a common dietary supplement. Research data suggest that idebenone may be able to limit demyelination and death of brain cells and thereby slow or halt the progression of neurological dysfunction in MS.

       Comparison of the Efficacy and Mechanisms for MBCT and CT for Multiple Sclerosis (MS) Chronic Pain. Cognitive therapy (CT) and mindfulness-based cognitive therapy (MBCT) is a psychosocial treatment that has been proven to be potentially beneficial for chronic MS pain. The aim is to teach patients to identify and replace unhelpful thoughts about pain with helpful, more adaptive thoughts.

       Lipoic Acid as a Treatment for Acute Optic Neuritis. Explores the hypothesis that oral lipoic acid can safely help relieve permanent optic nerve injury in patients diagnosed with acute optic neuritis. It will also help determine how the body absorbs and breaks down the study drug, and what effects it has on the immune system.

      Phase II Clinical Trials

      Phase II studies test the efficacy of a treatment. Most are “double-blinded” randomized trials (neither the patients nor the researchers know who receives the experimental treatment and who gets placebo) aimed at determining comparisons of relative safety and effectiveness of the new drug. The results are then forwarded to the FDA and the pharmaceutical company for approval to progress to Phase III trials. This phase can last from several months to two years, and involves up to several hundred patients. Some noteworthy phase II trials in the research for MS include the following.

       Alternative Treatment Paradigm for Natalizumab Trial. A study being done to determine the difference between natalizumab therapy followed by two different withdrawal strategies using glatiramer acetate (GA), that is, GA alone and GA plus corticosteroids in preventing clinical relapses and decreasing other markers of disease activity in patients diagnosed with MS. It is predicted that the latter regimen will help reduce recurrences of post natalizumab discontinuation earlier than the effect of GA alone and early reconstitution of peripheral and CNS immune homeostasis.

       Caprylic Triglyceride for Treatment of Cognitive Impairments in Multiple Sclerosis. This study aims at exploring the possibility of providing an alternative energy source, namely, caprylic triglyceride in place of glucose (a type of sugar), the natural fuel for healthy brain cells. It is believed that in MS, glucose is not converted into energy as efficiently as in a healthy brain, which can lead to a decrease in cognitive function. Caprylic triglyceride may work to bypass this problem by being metabolized in the liver and used by the brain.

       Estriol Treatment in Multiple Sclerosis (MS): Effect on Cognition. This trial will ascertain whether treatment with an estrogen pill combined with standard MS anti-inflammatory drugs can improve cognition as compared to treatment with a placebo pill in combination with standard anti-inflammatory drugs in women with MS.

       Comparison of Subcutaneous Interferon Beta-1a With Glatiramer Acetate in Patients With Relapsing Multiple Sclerosis.

       Assessment of Clemastine Fumarate as a Remyelinating Agent in Multiple Sclerosis. Efficacy, Safety, and Tolerability of Plovamer Acetate (Pathway 1): a Phase II trial that will test four different doses of plovamer acetate against the active comparator copaxone in subjects with RRMS.

       Study of Tcelna (Imilecleucel-T) in Secondary Progressive Multiple Sclerosis. To ascertain the use of biological agents like Tcelna synthesized from autologous pool of myelin reactive T cells (MRTC) in treatment of MS.

       Safety and Brain Protection Effects of the Green Tea Extract Theaphenon 95 Percent (95 Percent Pure EGCG) in Multiple Sclerosis. Epigallocatechin-3-gallate, a potent antioxidant found in tea, can protect brain cells in patients with MS. The study will compare changes in n-acetyl-aspartate (a chemical that reflects the number of neurons and their metabolism) between people with MS treated with EGCG with MS patients treated with a matching sugar pill.

       Trial of Ginkgo as a Treatment for Cognitive Problems in Multiple Sclerosis.

       Testosterone Treatment for Multiple Sclerosis.

      Phase III Clinical Trials

      Phase III studies provide the pharmaceutical company and the FDA with a more thorough understanding of the effectiveness of the drug/treatment, benefits, and the range of possible adverse reactions. These studies involve several hundred to several thousand patients and can last several years. Once Phase III is complete, a pharmaceutical company can request FDA approval for marketing the drug. Some of the recent phase III trials in the US are listed below.

       A Clinical Evaluation of the Safety of Baclofen Er Capsules (Grs) When Administered Once Daily to Subjects With Spasticity Due to Multiple Sclerosis. A Randomized Trial of Two Formal Group Programs for Multiple Sclerosis: this trial involves behavioral therapy and techniques to help relieve fatigue and other symptoms associated with MS.

       Vitamin D in Multiple Sclerosis: low vitamin D levels are known to be associated with increased risk of developing MS, and patients with MS who have lower vitamin D levels are at increased risk of having attacks. However, it is not known if giving supplemental vitamin D to those with MS reduces the risk of attacks, and some research suggests that vitamin D could even be harmful to people with MS.

       Oral Cladribine in Early Multiple Sclerosis (MS).

       Combination Therapy in Patients with Relapsing-Remitting Multiple Sclerosis (MS).

       Duloxetine for Multiple Sclerosis Pain. A Study of Ocrelizumab in Patients with Primary Progressive Multiple Sclerosis

      Phase IV Clinical Trials

      Phase IV studies, also called post-marketing surveillance trials, are conducted after a drug is introduced into the market for consumer sale. The objectives are to compare a drug with other drugs already in the market; to monitor long-term effectiveness and impact on a patient’s quality of life; and to determine the cost effectiveness of a drug therapy relative to other traditional and new therapies. Phase IV studies can result in drugs being taken off the market or use restrictions could be placed on the product, depending on the results. Important Phase IV studies in the United States include the following.

       Rebif Advanced Magnetic Resonance Imaging (MRI) and Immunology Pilot Trial. This trial will evaluate the effects of Rebif® on remyelination/demyelination; lesion and brain volume; CNS iron deposition, and immune status in subjects with RRMS using MRI techniques.

       A Phase IV Trial of Neuroprotection With ACTH in Acute Optic Neuritis. This study will use ocular coherence tomography (OCT) to compare the melanocortin-mediated anti-inflammatory effects of adrenocorticotropic hormone (ACTH) and intravenous methylprednisone therapy on axonal injury following ON by limiting inflammatory optic nerve injury.

       An fMRI (Functional Magnetic Resonance Imaging) Research Study to Learn More About MS and Individuals Potentially Experiencing Memory Difficulties. This study will determine if medication designed to improve memory, for example, donepezil will help patients with MS who are experiencing memory problems using fMRI to track brain activity.

       Safety and Efficacy Study of Doxycycline in Combination With Interferon-B-1a to Treat Multiple Sclerosis. Evaluated the efficacy, safety, and tolerability of combination therapy with intramuscular