ViroPharma Licenses Rights From Intellect Neurosciences for Product Candidate for Friedreich's Ataxia

EXTON, Pa., Sept. 30, 2011 /PRNewswire via COMTEX/ -- ViroPharma Incorporated (VPHM) today announced the license of worldwide rights from Intellect Neurosciences, Inc. (ILNS) to its clinical stage drug candidate, OX1, being developed for the treatment of Friedreich's Ataxia (FA), a rare, hereditary, progressive neurodegenerative disease. read more ...

Gene therapies advance towards finish line

Nature Reviews Drug Discovery 10, 719-720 (October 2011) | doi:10.1038/nrd3572

Asher Mullard

News and Analysis. Over a decade since gene therapy development came to a near standstill with the death of a clinical trial participant, the field is overcoming issues of immunogenicity, carcinogenicity, manufacturing and small patient populations.....

“I am certain that a gene therapy is going to cross the finish line within the next year or two, in either the United States or the European Union,” says Coté.

Neural Substrates for the Motivational Regulation of Motor Recovery after Spinal-Cord Injury

I am aware that the casuistry of a spinal injury is very distant from the AF, but this paper shows a link between the willingness and recovery progress , or in our case, to preserve the faculties for longer.

PLoS ONE, 2011; 6 (9): e24854 DOI: 10.1371/journal.pone.0024854

Yukio Nishimura1,2,3,7*, Hirotaka Onoe3,4, Kayo Onoe5, Yosuke Morichika1, Hideo Tsukada3,6, Tadashi Isa1,3,7

1 Department of Developmental Physiology, National Institute for Physiological Sciences, Okazaki, Japan, 2 Precursory Research for Embryonic Science and Technology, Japan Science and Technology Agency, Tokyo, Japan, 3 Core Research for Evolutional Science and Technology, Japan Science and Technology Agency, Kawaguchi, Japan, 4 Functional Probe Research Laboratory, RIKEN Center for Molecular Imaging Science, Kobe, Japan, 5 Molecular Dynamics Laboratory, RIKEN Center for Molecular Imaging Science, Kobe, Japan, 6 Central Research Laboratory, Hamamatsu Photonics, Hamamatsu, Japan, 7 Graduate University for Advanced Studies (SOKENDAI), Hayama, Japan

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"It is believed that depression impedes and motivation enhances functional recovery after neuronal damage such as spinal-cord injury and stroke. However, the neuronal substrate underlying such psychological effects on functional recovery remains unclear. "

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In layman's words

ScienceDaily (Sep. 28, 2011) — An effective recovery has been observed in stroke patients and those with spinal cord injuries who have strong vitality and motivation to rehabilitate in clinical practice. However, it was not really clear how motivation facilitates functional recovery in brain science. read more....

Understanding the function of frataxin in eukaryotes

PhD Research Project, Medical Research Council, National Institute for Medical Research, PhD Supervisor: Dr A Pastore
Application Deadline: 30 November 2011

The work should lead both to a better understanding of the mechanisms of iron-sulfur cluster formation and to the establishment of the frataxin function.

Mitochondrial disorders and the eye

Eye and Brain, September 2011 Volume 2011:3 Pages 29 - 47
DOI: http://dx.doi.org/10.2147/EB.S16192

Van Bergen NJ, Chakrabarti R, O’Neill EC, Crowston JG, Trounce IA

Keywords: mitochondria, disease, retina, eye, aging, neuroprotection, Friedreich’s ataxia.

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1st iissue off EFACTS NEWS

The EFACTS (European Friedreich’s Ataxia Consortium for Translational Studies) Consortium will publish the newsletter EFACTS NEWS aimed at communicating the activities of the Network and progress in FRDA research to affected families, the general public, health care professionals and the scientific community. The newsletter will be published annually and the issues will appear on the EFACTS Website

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Mutation in Fe-S scaffold Isu bypasses frataxin deletion

Biochem. J. (2011) Immediate Publication, doi:10.1042/BJ20111637

Heeyong Yoon, Ramesh Golla, Emmanuel Lesuisse, Jayashree Pain, Jason Donald, Elise R. Lyver, Debkumar Pain and Andrew Dancis
University of Pennsylvania, Philadelphia, U.S.A.

Keywords: Frataxin, Friedreich’s ataxia, iron homeostasis, Fe-S cluster assembly, cysteine desulfurase (Nfs1), accessory protein (Isd11), scaffold protein (Isu), single amino acid substitution.

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Orphan Drugs, Big Pharma

Human Gene Therapy. September 2011, 22(9): 1035-1038. doi:10.1089/hum.2011.2515.

—Alex Philippidis, Senior News Editor

Safety and Efficacy of Intravenous Immune Globulin in Treating Friedreich's Ataxia and Spinocerebellar Ataxia

Sponsor: University of South Florida
Collaborator: Baxter Healthcare Corporation
Information provided by (Responsible Party): Theresa Zesiewicz, University of South Florida
ClinicalTrials.gov Identifier: NCT01350440

This study is currently recruiting participants. (Last Updated on September 9, 2011)

Neuroprotective and metabolic effects of resveratrol: Therapeutic implications for huntington's disease and other neurodegenerative disorders

doi:10.1016/j.expneurol.2011.08.014

Giulio Maria Pasinettilow asterisk, a, E-mail The Corresponding Author, Jun Wanga, Philippe Marambauda, Mario Ferruzzia, Paul Gregora, Lindsay Alexis Knablea and Lap Hoa
a Center of Excellence for Research in Complementary and Alternative Medicine in Alzheimer's Disease, Department of Neurology, Mount Sinai School of Medicine, New York, NY 1002

Keywords: Resveratrol, polyphenolic compound, aging, metabolic disorders, inflammation , cancer, currently being tested in numerous clinical trials, key metabolic sensor/effector proteins, potential neuroprotective effects, SRT501 , Huntington's disease (HD)., diabetes mellitus.

Iron Efflux from Oligodendrocytes Is Differentially Regulated in Gray and White Matter

The Journal of Neuroscience, 14 September 2011, 31(37): 13301-13311; doi: 10.1523/​JNEUROSCI.2838-11.2011

Katrin Schulz 1, Chris D. Vulpe 2, Leah Z. Harris 3, and Samuel David 1
1Center for Research in Neuroscience, The Research Institute of the McGill University Health Center, Montreal, Quebec H3G 1A4, Canada,
2Department of Nutritional Sciences and Toxicology, University of California, Berkeley, California 94720, and
3Department of Pediatrics, Vanderbilt University, Nashville, Tennessee 37232

Keywords: Accumulation of iron, neurodegenerative diseases, generate toxic free radicals, Iron homeostasis, ferroportin, ferroxidase, astrocytes, oligodendrocytes, hephaestin.

"Dysregulation of such efflux mechanisms leads to iron accumulation in the CNS."

Evaluation of histone deacetylase inhibitors as therapeutics for neurodegenerative diseases.

Methods Mol Biol. 2011;793:495-508.

Soragni E, Xu C, Cooper A, Plasterer HL, Rusche JR, Gottesfeld JM.
Department of Molecular Biology, The Scripps Research Institute, La Jolla, CA, USA.

Keywords: neurodegenerative diseases, aberrant gene expression, pimelic o-aminobenzamide histone deacetylase (HDAC) inhibitors, Friedreich's ataxia (FRDA), Huntington's disease (HD), cellular models.

Superando la barrera hematoencefálica

Traduccion de EurekAlert!.org, 13 septiembre 2011, que se encuentra en Bitnavegantes


"Por primera vez, los investigadores descubrieron que, cuando los receptores de adenosina se activan en las células que forman la barrera hematoencefálica, se abre una puerta de entrada a través dicha barrera."

Breaching the blood-brain barrier

EurekAlert!, Public release date: 13-Sep-2011

"For the first time, the researchers discovered that when adenosine receptors are activated on cells that comprise the blood-brain barrier, a gateway into the blood-brain barrier can be established."

Adenosine Receptor Signaling Modulates Permeability of the Blood–Brain Barrier

The Journal of Neuroscience, 14 September 2011, 31(37): 13272-13280; doi: 10.1523/​JNEUROSCI.3337-11.2011

Aaron J. Carman, Jeffrey H. Mills, Antje Krenz, Do-Geun Kim, and Margaret S. Bynoe
Department of Microbiology and Immunology, Cornell University, College of Veterinary Medicine, Ithaca, New York 14853

Acute myocardial infarction after botulinum toxin injection.

QJM (2011) 104(7): 615-616

B.E. Stähli, L. Altwegg, T.F. Lüscher and R. Corti
Department of Cardiology, Cardiovascular Center, University Hospital Zürich, Zürich, Switzerland

Keywords: Friedreich ataxia, ventricular fibrillation, botulinum toxin A, neurogenic bladder dysfunction,thrombotic occlusion.

Shuttle-Mediated Drug Delivery to the Brain.

Angew Chem Int Ed Engl. 2011 Jun 30. doi: 10.1002/anie.201006565. [Epub ahead of print]

Malakoutikhah M, Teixidó M, Giralt E.

Institute for Research in Biomedicine (IRB Barcelona), Barcelona (Spain)

Keywords: shuttle-mediated drug delivery, blood-brain barrier (BBB), central nervous system, vector-mediated approach, ability to cross the BBB.

Mesenchymal stem cells: from experiment to clinic

Fibrogenesis & Tissue Repair 2011, 4:20 doi:10.1186/1755-1536-4-20
William R Otto1 and Nicholas A Wright2

1 Histopathology Laboratory, Cancer Research UK, London Research Institute, London, UK. 2 Blizard Institute, Barts and The London, Queen Mary’s School of Medicine and Dentistry, London, UK.

"Good review, defines the current state of research and use of Mesenchymal stem cells"

Importance of Iron Chelation in Free Radical-Induced Oxidative Stress and Human Disease.

Curr Pharm Des. 2011 Sep 9,

Jomova K, Valko M.
Faculty of Chemical and Food Technology, Slovak Technical University, Bratislava, Slovakia.

Keywords: Iron, redox active metal, hemochromatosis, Alzheimer's disease, Parkinson's disease, Huntington's disease, Friedreich's ataxia, neurological disorders, cancer, Fanconi anemia, stroke, ageing, Fenton reaction, reactive oxygen species (ROS), iron-designed chelators.

A listing of clinical trials currently looking for volunteers to enroll in Friedreich's Ataxia studies.

Austria, Innsbruck :

Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)

Roles of Porphyrin and Iron Metabolisms in the δ-Aminolevulinic Acid (ALA)-induced Accumulation of Protoporphyrin and Photodamage of Tumor Cells

Photochemistry and Photobiology, Volume 87, Issue 5, pages 1138–1145, September/October 2011, DOI: 10.1111/j.1751-1097.2011.00950.x

Yoshiko Ohgari, Yoshinobu Miyata, Taeko Miyagi, Saki Gotoh, Takano Ohta, Takao Kataoka, Kazumichi Furuyama and Shigeru Taketani

"The decrease of mitochondrial utilization of iron by the knockdown of mitoferrin-2 and frataxin also enhanced the ALA effect."

PEP-1-Frataxin Significantly Increases Cell Proliferation and Neuroblast Differentiation by Reducing Lipid Peroxidation in the Mouse Dentate Gyrus.

Neurochem Res. 2011 Sep 1,

Kim W, Kim DW, Shin BN, Yoo DY, Nam SM, Kim MJ, Choi JH, Yoon YS, Won MH, Choi SY, Hwang IK.

Department of Anatomy and Cell Biology, College of Veterinary Medicine, and Research Institute for Veterinary Science, Seoul National University, South Korea.

Keywords: Frataxin, cell proliferation, neuroblast differentiation, PEP-1-frataxin fusion protein, decreasing lipid peroxidation, blood-brain barrier, dentate gyrus.

Articulatory Kinematics in the Dysarthria Associated with Friedreich's Ataxia.

Motor Control. 2011 Jul;15(3):376-89.

Folker JE, Murdoch BE, Cahill LM, Rosen KM, Delatycki MB, Corben LA, Vogel AP.

School of Health and Rehabilitation Sciences, The University of Queensland, St Lucia, Qld, Australia.

KEYWORDS: Electromagnetic articulography (EMA), dysarthria, Friedreich's ataxia (FRDA).

Drug first to demonstrate neurological improvement in patients with Friedreich’s ataxia

Website: USF HEALTH (UNIVERSITY OF SOUTH FLORIDA)

Preliminary trial findings shared with FARA-USF research symposium participants

"For the first time, an investigative drug has significantly improved the neurological function of patients with Friedreich’s ataxia."

“This is the first clinical trial where patients were given a drug and they’ve shown neurological improvement,” Farmer said. “It’s incredibly hopeful… but there’s still a lot of work ahead. We need to show the drug really makes a difference over six months or a year.”

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