Herbert-Worch-Foundation
A Spanish University developed cell models to study the effects of frataxin deficiency on human neuron-like cells.
Companies from all countries interested in putting on the market a new neuronal cell model for Friedreich´s ataxia are sought.
Current and Potential Domain of Application: Screening and testing of possible therapeutic compounds. Current Stage of Development: development phase - Laboratory tested.
Thursday, October 6, 2011
Tuesday, October 4, 2011
Les effets secondaires du principe de précaution
Article in: Association française contre les myopathies (AFM) Blog (3/10/11)
Health authorities (FDA, EMEA, national agencies, etc.) have the obligation to ensure our health and safety from new drugs or treatments, but sometimes look like they know very little about our disease, in this case, in France are blocking a compassionate use due a the very remote possibility of an side effect that can appears in old age, unfortunately, few patients will reach if there is no effective treatment, and can not test it.
It is very sad, french patients which participated in the Dr. Isabelle Husson and Pierre Rustin Pioglitazone clinical trial, when they finish the two years clinical trial, they can continue with the Pioglitazone as compassionate use, now, the authorities ban this option, although as some patiens explain, they feel that get improvements. The reason, a very little possibility to develop a side effec, prostate cancer, a possibility that unfortunately is not logical for FA patiens, due to the typical disease life expectancy.
Health authorities (FDA, EMEA, national agencies, etc.) have the obligation to ensure our health and safety from new drugs or treatments, but sometimes look like they know very little about our disease, in this case, in France are blocking a compassionate use due a the very remote possibility of an side effect that can appears in old age, unfortunately, few patients will reach if there is no effective treatment, and can not test it.
It is very sad, french patients which participated in the Dr. Isabelle Husson and Pierre Rustin Pioglitazone clinical trial, when they finish the two years clinical trial, they can continue with the Pioglitazone as compassionate use, now, the authorities ban this option, although as some patiens explain, they feel that get improvements. The reason, a very little possibility to develop a side effec, prostate cancer, a possibility that unfortunately is not logical for FA patiens, due to the typical disease life expectancy.
Sunday, October 2, 2011
What is the OX1?
The OX1 or OXIGON (TM) is the Indole-3-propionic acid (IPA) (SYNONYMS: 3-Indolepropionic Acid, 1H-Indole-3-propanoic acid, beta-Indole-3-propionic acid, 3-(3-Indolyl)propanoic acid, 3-(3-Indolyl)propionic acid, 3-Indolyl propionic acid). It is a compound closely related to melatonin, but with a much more powerful antioxidant action.
Has been previously proposed as a possible Alzheimer’s disease therapy, after, it is being investigated for other neurodegenerative diseases such HD, Friedreich's Ataxia, ataxia, ..., for its neuroprotective action. As usual, the major efforts are focused in AD, and most scientific "public" information is related to AD.
Curiously, it also has application in gardening, is an auxin (plant hormone), and it is used as a growth roots stimulator . :-)
References:
Potent neuroprotective properties against the Alzheimer beta-amyloid by an endogenous melatonin-related indole structure, indole-3-propionic acid. Chyan YJ, Poeggeler B, Omar RA, Chain DG, Frangione B, Ghiso J, Pappolla MA. J Biol Chem. 1999
Development of indole-3-propionic acid (OXIGON) for Alzheimer's disease. Bendheim PE, Poeggeler B, Neria E, Ziv V, Pappolla MA, Chain DG. J Mol Neurosci. 2002
Indole-3-propionic acid attenuates neuronal damage and oxidative stress in the ischemic hippocampus. Hwang IK, Yoo KY, Li H, Park OK, Lee CH, Choi JH, Jeong YG, Lee YL, Kim YM, Kwon YG, Won MH. J Neurosci Res. 2009
A novel endogenous indole protects rodent mitochondria and extends rotifer lifespan. Poeggeler B, Sambamurti K, Siedlak SL, Perry G, Smith MA, Pappolla MA. PLoS One. 2010
Melatonin treatment restores mitochondrial function in Alzheimer's mice: a mitochondrial protective role of melatonin membrane receptor signaling. Dragicevic N, Copes N, O'Neal-Moffitt G, Jin J, Buzzeo R, Mamcarz M, Tan J, Cao C, Olcese JM, Arendash GW, Bradshaw PC. J Pineal Res. 2011.
Intellect obtains pharmacokinetic data from OX1 Phase 1b trial for Alzheimer's disease
Intellect Neurosciences Files Orphan Drug Application in the United States for Its Clinical Candidate OX1 for the Treatment of Friedreich's Ataxia
Clinical proof of concept patient trials for OX1 in Friedreich's Ataxia
ViroPharma Licenses Rights From Intellect Neurosciences for Product Candidate for Friedreich's Ataxia
Has been previously proposed as a possible Alzheimer’s disease therapy, after, it is being investigated for other neurodegenerative diseases such HD, Friedreich's Ataxia, ataxia, ..., for its neuroprotective action. As usual, the major efforts are focused in AD, and most scientific "public" information is related to AD.
Curiously, it also has application in gardening, is an auxin (plant hormone), and it is used as a growth roots stimulator . :-)
References:
Potent neuroprotective properties against the Alzheimer beta-amyloid by an endogenous melatonin-related indole structure, indole-3-propionic acid. Chyan YJ, Poeggeler B, Omar RA, Chain DG, Frangione B, Ghiso J, Pappolla MA. J Biol Chem. 1999
Development of indole-3-propionic acid (OXIGON) for Alzheimer's disease. Bendheim PE, Poeggeler B, Neria E, Ziv V, Pappolla MA, Chain DG. J Mol Neurosci. 2002
Indole-3-propionic acid attenuates neuronal damage and oxidative stress in the ischemic hippocampus. Hwang IK, Yoo KY, Li H, Park OK, Lee CH, Choi JH, Jeong YG, Lee YL, Kim YM, Kwon YG, Won MH. J Neurosci Res. 2009
A novel endogenous indole protects rodent mitochondria and extends rotifer lifespan. Poeggeler B, Sambamurti K, Siedlak SL, Perry G, Smith MA, Pappolla MA. PLoS One. 2010
Melatonin treatment restores mitochondrial function in Alzheimer's mice: a mitochondrial protective role of melatonin membrane receptor signaling. Dragicevic N, Copes N, O'Neal-Moffitt G, Jin J, Buzzeo R, Mamcarz M, Tan J, Cao C, Olcese JM, Arendash GW, Bradshaw PC. J Pineal Res. 2011.
Intellect obtains pharmacokinetic data from OX1 Phase 1b trial for Alzheimer's disease
Intellect Neurosciences Files Orphan Drug Application in the United States for Its Clinical Candidate OX1 for the Treatment of Friedreich's Ataxia
Clinical proof of concept patient trials for OX1 in Friedreich's Ataxia
ViroPharma Licenses Rights From Intellect Neurosciences for Product Candidate for Friedreich's Ataxia
Friday, September 30, 2011
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.
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.
Thursday, September 29, 2011
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
OPEN ACCESS
"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. "
FULL TEXT PDF
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....
Wednesday, September 28, 2011
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.
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.
OPEN ACCESS
FULL TEXT PDF
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.
OPEN ACCESS
FULL TEXT PDF
Wednesday, September 21, 2011
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
Subscribe Newsletter
Subscribe Newsletter
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.
FULL TEXT PDF
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.
FULL TEXT PDF
Subscribe to:
Posts (Atom)
