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4 results on '"Panzara, M. A."'

1. The MSOAC approach to developing performance outcomes to measure and monitor multiple sclerosis disability

Author

LaRocca, NG, Hudson, LD, Rudick, R, Amtmann, D, Balcer, L, Benedict, R, Bermel, R, Chang, I, Chiaravalloti, ND, Chin, P, Cohen, JA, Cutter, GR, Davis, MD, DeLuca, J, Feys, P, Francis, G, Goldman, MD, Hartley, E, Kapoor, R, Lublin, F, Lundstrom, G, Matthews, PM, Mayo, N, Meibach, R, Miller, DM, Motl, RW, Mowry, EM, Naismith, R, Neville, J, Panagoulias, J, Panzara, M, Phillips, G, Robbins, A, Sidovar, MF, Smith, KE, Sperling, B, Uitdehaag, BM, Weaver, J, Multiple Sclerosis Outcome Assessments Consortium (MSOAC), Neurology, Amsterdam Neuroscience - Neuroinfection & -inflammation, and Medical Research Council (MRC)

Subjects
STATUS SCALE, medicine.medical_specialty, Multiple Sclerosis, Databases, Factual, IMPACT, Clinical Neurology, CONTRAST LETTER ACUITY, Measure (physics), Disability Evaluation, 03 medical and health sciences, 0302 clinical medicine, Physical medicine and rehabilitation, Outcome Assessment, Health Care, medicine, EMPLOYMENT, Humans, MS disability, performance outcome measures, data standards, clinical trial database, regulatory qualification, 030212 general & internal medicine, VALIDITY, SPEED, Functional composite, PROGRESSIVE MS, Science & Technology, Neurology & Neurosurgery, business.industry, Multiple sclerosis, Neurosciences, FUNCTIONAL COMPOSITE, 1103 Clinical Sciences, IMPAIRMENT, medicine.disease, Multiple Sclerosis Outcome Assessments Consortium (MSOAC), Clinical neurology, Neurology, Physical therapy, Neurosciences & Neurology, HEALTH, Neurology (clinical), 1109 Neurosciences, business, Life Sciences & Biomedicine, Original Research Papers, 030217 neurology & neurosurgery
Abstract

Background: The Multiple Sclerosis Outcome Assessments Consortium (MSOAC) was formed by the National MS Society to develop improved measures of multiple sclerosis (MS)-related disability. Objectives: (1) To assess the current literature and available data on functional performance outcome measures (PerfOs) and (2) to determine suitability of using PerfOs to quantify MS disability in MS clinical trials. Methods: (1) Identify disability dimensions common in MS; (2) conduct a comprehensive literature review of measures for those dimensions; (3) develop an MS Clinical Data Interchange Standards Consortium (CDISC) data standard; (4) create a database of standardized, pooled clinical trial data; (5) analyze the pooled data to assess psychometric properties of candidate measures; and (6) work with regulatory agencies to use the measures as primary or secondary outcomes in MS clinical trials. Conclusion: Considerable data exist supporting measures of the functional domains ambulation, manual dexterity, vision, and cognition. A CDISC standard for MS (http://www.cdisc.org/therapeutic#MS) was published, allowing pooling of clinical trial data. MSOAC member organizations contributed clinical data from 16 trials, including 14,370 subjects. Data from placebo-arm subjects are available to qualified researchers. This integrated, standardized dataset is being analyzed to support qualification of disability endpoints by regulatory agencies. The author(s) disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: MSOAC is funded largely through the National Multiple Sclerosis Society grant (no. RG 4869-A-1) to the Critical Path Institute. Annual dues from sponsors supplement the NMSS grant.

Published
2018
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3. Progressive MS Alliance Industry Forum: Maximizing Collective Impact To Enable Drug Development

Author

Paola Zaratin, Kathryn E Smith, Alan J. Thompson, G. Comi, M. Panzara, Timothy Coetzee, K. Ramsey, Zaratin, P, Comi, Giancarlo, Coetzee, T, Ramsey, K, Smith, K, Thompson, A, and Panzara, M.

Subjects
0301 basic medicine, Pharmacology, Progressive multiple sclerosis, Biomedical Research, Process management, Drug Industry, education, Multiple Sclerosis, Chronic Progressive, Toxicology, Collective impact, 03 medical and health sciences, 030104 developmental biology, 0302 clinical medicine, Alliance, Drug development, Drug Design, Drug Discovery, Humans, Business, Cooperative Behavior, health care economics and organizations, 030217 neurology & neurosurgery
Abstract

The Progressive MS Alliance Industry Forum describes a new approach to address barriers to developing treatments for progressive multiple sclerosis (MS). This innovative model promises to facilitate robust collaboration between industry, academia, and patient organizations and accelerate research towards the overarching goal of developing safe and effective treatments for progressive MS.

Published
2016
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4. Selection for T-cell receptor V beta-D beta-J beta gene rearrangements with specificity for a myelin basic protein peptide in brain lesions of multiple sclerosis.

Author

Oksenberg JR, Panzara MA, Begovich AB, Mitchell D, Erlich HA, Murray RS, Shimonkevitz R, Sherritt M, Rothbard J, Bernard CC, and Steinman L

Subjects
Amino Acid Sequence, Animals, Base Sequence, Brain pathology, Cloning, Molecular, Encephalomyelitis, Autoimmune, Experimental genetics, Encephalomyelitis, Autoimmune, Experimental immunology, HLA-D Antigens analysis, Humans, Molecular Sequence Data, Multiple Sclerosis pathology, Oligodeoxyribonucleotides, Phenotype, Polymerase Chain Reaction methods, RNA, Messenger genetics, Rats, Spinal Cord immunology, T-Lymphocytes immunology, Brain immunology, CD3 Complex genetics, Gene Rearrangement, beta-Chain T-Cell Antigen Receptor, HLA-D Antigens genetics, Multiple Sclerosis genetics, Multiple Sclerosis immunology, Myelin Basic Protein immunology
Abstract

Multiple sclerosis (MS) is an inflammatory demyelinating disease of the central nervous system in which a restricted cellular immune response has been observed. In order to establish whether such T cell responses are likely to be antigen-specific particularly with regard to myelin basic protein (MBP), we analysed T-cell receptor (TCR) gene rearrangements directly from MS brain plaques, using the polymerase chain reaction on reverse transcribed messenger RNA, and compared these with TCR of previously described MBP-specific T cell clones from MS and the rat model experimental allergic encephalomyelitis. Rearranged V beta 5.2 genes were detected in the brains of all patients who were HLA DRB1*1501, DQA1*0102, DQB1*0602, DPB1*0401. The V beta 5.2-D beta-J beta sequences in these MS brain plaques revealed five motifs. One of the common motifs was identical to that described for the VDJ region of a V beta 5.2 T-cell clone. This clone was from an MS patient who was HLA DRB1*1501, DQB1*0602, DPB1*0401, and it was cytotoxic towards targets containing the MBP peptide 89-106 (ref. 1). The deduced amino-acid sequence of this VDJ rearrangement, Leu-Arg-Gly, has also been described in rat T cells cloned from experimental allergic encephalomyelitis lesions, which are specific for MBP peptide 87-99 (ref. 2). VDJ sequences with specificity for this MBP epitope constitute a large fraction (40%) of the TCR V beta 5.2 N(D)N rearrangements in MS lesions. The capacity of rat T cells with these VDJ sequences to cause experimental allergic encephalomyelitis and the prevalence of such sequences in demyelinated human lesions indicate that T cells with this rearranged TCR may be critical in MS.

Published
1993
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