Showing posts with label visit-UK. Show all posts
Showing posts with label visit-UK. Show all posts

Tuesday, December 12, 2017

Drug lowers deadly Huntington’s disease protein

reposted from


Drug lowers deadly Huntington’s disease protein

11 December 2017
The first drug targeting the cause of Huntington’s disease was safe and well-tolerated in its first human trial led by UCL scientists. It successfully lowered the level of the harmful huntingtin protein in the nervous system.
HDbrain
After over a decade in pre-clinical development, this first human trial of huntingtin-lowering drug began in late 2015, led by Professor Sarah Tabrizi (UCL Institute of Neurology) and sponsored by Ionis Pharmaceuticals.
The trial involved enrolling 46 patients with early Huntington’s disease at nine study centres in the UK, Germany and Canada.
Each patient received four doses of either IONIS-HTTRx or placebo, given by injection into the spinal fluid to enable it to reach the brain. As the phase 1/2a trial progressed, the dose of IONIS-HTTRx was increased several times according to the ascending-dose trial design. 
Patient safety was monitored throughout the study by an independent safety committee.
Today’s announcement at completion of the trial confirms that IONIS-HTTRx was well-tolerated by the trial participants and its safety profile supports further testing in patients.
Professor Tabrizi, Director of the UCL Huntington’s Disease Centre and IONIS-HTTRx Global Chief Investigator, said: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families. For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”
A major unknown was whether the trial would show that IONIS-HTTRx could lower the level of mutant huntingtin protein in the nervous system. Using an ultra-sensitive assay, concentrations of the protein were measured in each patient’s spinal fluid before and after treatment.
As hoped, IONIS-HTTRx­ produced significant, dose-dependent lowering of the level of mutant huntingtin – the first time the protein known to cause Huntington’s has been lowered in the nervous system of patients.
As a result of these successful outcomes, Ionis’ partner, Roche, has exercised its option to license IONIS-HTTRx and assumes responsibility for further development, regulatory activities and commercialization activities.   Meanwhile, Ionis announced in June that all patients in the completed trial would be offered a place in an open-label extension to receive IONIS-HTTRx.
The results of the trial and plans for the ongoing IONIS-HTTRx programme will be presented in detail at forthcoming scientific meetings and prepared for peer-reviewed publication.
The research is supported by The National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research CentreThe centre is a partnership between UCL and University College London Hospitals NHS Foundation Trust funded by the NIHR to translate scientific breakthroughs into better patient treatments.

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Media contact

Margaret-Anne Orgill

Tel: +44 (0)20 3108 8515
Email: m.orgill [at] ucl.ac.uk

Thursday, September 26, 2013

Ballet dancers' brains adapt to stop them getting in a spin

reposted from:
http://www3.imperial.ac.uk/newsandeventspggrp/imperialcollege/newssummary/news_26-9-2013-17-43-4



News: health

Imperial College London

Ballet dancers' brains adapt to stop them feeling dizzy

by Sam Wong
main image
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Scientists have discovered differences in the brain structure of ballet dancers that may help them avoid feeling dizzy when they perform pirouettes.
The research suggests that years of training can enable dancers to suppress signals from the balance organs in the inner ear.
The findings, published in the journal Cerebral Cortex, could help to improve treatment for patients with chronic dizziness. Around one in four people experience this condition at some time in their lives.
Normally, the feeling of dizziness stems from the vestibular organs in the inner ear. These fluid-filled chambers sense rotation of the head through tiny hairs that sense the fluid moving. After turning around rapidly, the fluid continues to move, which can make you feel like you’re still spinning.
Ballet dancers can perform multiple pirouettes with little or no feeling of dizziness. The findings show that this feat isn’t just down to spotting, a technique dancers use that involves rapidly moving the head to fix their gaze on the same spot as much as possible.
Researchers at Imperial College London recruited 29 female ballet dancers and, as a comparison group, 20 female rowers whose age and fitness levels matched the dancers’.
The volunteers were spun around in a chair in a dark room. They were asked to turn a handle in time with how quickly they felt like they were still spinning after they had stopped. The researchers also measured eye reflexes triggered by input from the vestibular organs. Later, they examined the participants’ brain structure with MRI scans.
In dancers, both the eye reflexes and their perception of spinning lasted a shorter time than in the rowers.
Dr Barry Seemungal, from the Department of Medicine at Imperial, said: “Dizziness, which is the feeling that we are moving when in fact we are still, is a common problem. I see a lot of patients who have suffered from dizziness for a long time. Ballet dancers seem to be able to train themselves not to get dizzy, so we wondered whether we could use the same principles to help our patients.”
The brain scans revealed differences between the groups in two parts of the brain: an area in the cerebellum where sensory input from the vestibular organs is processed and in the cerebral cortex, which is responsible for the perception of dizziness.
The area in the cerebellum was smaller in dancers. Dr Seemungal thinks this is because dancers would be better off not using their vestibular systems, relying instead on highly co-ordinated pre-programmed movements.
“It’s not useful for a ballet dancer to feel dizzy or off balance. Their brains adapt over years of training to suppress that input. Consequently, the signal going to the brain areas responsible for perception of dizziness in the cerebral cortex is reduced, making dancers resistant to feeling dizzy.
“If we can target that same brain area or monitor it in patients with chronic dizziness, we can begin to understand how to treat them better.”
Another finding in the study may be important for how chronic dizzy patients are tested in the clinic. In the control group, the perception of spinning closely matched the eye reflexes triggered by vestibular signals, but in dancers, the two were uncoupled.
“This shows that the sensation of spinning is separate from the reflexes that make your eyes move back and forth,” Dr Seemungal said. “In many clinics, it’s common to only measure the reflexes, meaning that when these tests come back normal the patient is told that there is nothing wrong. But that’s only half the story. You need to look at tests that assess both reflex and sensation.”
The research was funded by a Health Foundation / Academy of Medical Sciences Fellowship and the Medical Research Council.

Y Nigmatullina et al. ‘The Neuroanatomical Correlates of Training-Related Perceptuo-Reflex Uncoupling in Dancers’ Cerebral Cortex, 27 September 2013. doi:10.1093/cercor/bht266




http://www.eurekalert.org/pub_releases/2013-09/icl-bdb092513.php


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Contact: Sam Wong
sam.wong@imperial.ac.uk
44-207-594-2198
Imperial College London 

Ballet dancers' brains adapt to stop them getting in a spin

Scientists have discovered differences in the brain structure of ballet dancers that may help them avoid feeling dizzy when they perform pirouettes.
The research suggests that years of training can enable dancers to suppress signals from the balance organs in the inner ear.
The findings, published in the journal Cerebral Cortex, could help to improve treatment for patients with chronic dizziness. Around one in four people experience this condition at some time in their lives.
Normally, the feeling of dizziness stems from the vestibular organs in the inner ear. These fluid-filled chambers sense rotation of the head through tiny hairs that sense the fluid moving. After turning around rapidly, the fluid continues to move, which can make you feel like you're still spinning.
Ballet dancers can perform multiple pirouettes with little or no feeling of dizziness. The findings show that this feat isn't just down to spotting, a technique dancers use that involves rapidly moving the head to fix their gaze on the same spot as much as possible.
Researchers at Imperial College London recruited 29 female ballet dancers and, as a comparison group, 20 female rowers whose age and fitness levels matched the dancers'.
The volunteers were spun around in a chair in a dark room. They were asked to turn a handle in time with how quickly they felt like they were still spinning after they had stopped. The researchers also measured eye reflexes triggered by input from the vestibular organs. Later, they examined the participants' brain structure with MRI scans.
In dancers, both the eye reflexes and their perception of spinning lasted a shorter time than in the rowers.
Dr Barry Seemungal, from the Department of Medicine at Imperial, said: "Dizziness, which is the feeling that we are moving when in fact we are still, is a common problem. I see a lot of patients who have suffered from dizziness for a long time. Ballet dancers seem to be able to train themselves not to get dizzy, so we wondered whether we could use the same principles to help our patients."
The brain scans revealed differences between the groups in two parts of the brain: an area in the cerebellum where sensory input from the vestibular organs is processed and in the cerebral cortex, which is responsible for the perception of dizziness.
The area in the cerebellum was smaller in dancers. Dr Seemungal thinks this is because dancers would be better off not using their vestibular systems, relying instead on highly co-ordinated pre-programmed movements.
"It's not useful for a ballet dancer to feel dizzy or off balance. Their brains adapt over years of training to suppress that input. Consequently, the signal going to the brain areas responsible for perception of dizziness in the cerebral cortex is reduced, making dancers resistant to feeling dizzy. If we can target that same brain area or monitor it in patients with chronic dizziness, we can begin to understand how to treat them better."
Another finding in the study may be important for how chronic dizzy patients are tested in the clinic. In the control group, the perception of spinning closely matched the eye reflexes triggered by vestibular signals, but in dancers, the two were uncoupled.
"This shows that the sensation of spinning is separate from the reflexes that make your eyes move back and forth," Dr Seemungal said. "In many clinics, it's common to only measure the reflexes, meaning that when these tests come back normal the patient is told that there is nothing wrong. But that's only half the story. You need to look at tests that assess both reflex and sensation."
###
The research was funded by a Health Foundation / Academy of Medical Sciences Fellowship and the Medical Research Council.
For more information please contact:
Sam Wong
Research Media Officer
Imperial College London
Email: sam.wong@imperial.ac.uk
Tel: +44(0)20 7594 2198
Out of hours duty press officer: +44(0)7803 886 248
Notes to editors
1. Y Nigmatullina et al. 'The Neuroanatomical Correlates of Training-Related Perceptuo-Reflex Uncoupling in Dancers' Cerebral Cortex, 27 September 2013. doi:10.1093/cercor/bht266
2. About Imperial College London
Consistently rated amongst the world's best universities, Imperial College London is a science-based institution with a reputation for excellence in teaching and research that attracts 14,000 students and 6,000 staff of the highest international quality. Innovative research at the College explores the interface between science, medicine, engineering and business, delivering practical solutions that improve quality of life and the environment - underpinned by a dynamic enterprise culture.
Since its foundation in 1907, Imperial's contributions to society have included the discovery of penicillin, the development of holography and the foundations of fibre optics. This commitment to the application of research for the benefit of all continues today, with current focuses including interdisciplinary collaborations to improve global health, tackle climate change, develop sustainable sources of energy and address security challenges.
In 2007, Imperial College London and Imperial College Healthcare NHS Trust formed the UK's first Academic Health Science Centre. This unique partnership aims to improve the quality of life of patients and populations by taking new discoveries and translating them into new therapies as quickly as possible.
3. About the Health Foundation
The Health Foundation is an independent charity working to continuously improve the quality of healthcare in the UK. We want the UK to have a healthcare system of the highest possible quality – safe, effective, person-centred, timely, efficient and equitable. We believe that in order to achieve this, health services need to continually improve the way they work. We are here to inspire and create the space for people, teams, organisations and systems to make lasting improvements to health services. Working at every level of the healthcare system, we aim to develop the technical skills, leadership, capacity, knowledge, and the will for change, that are essential for real and lasting improvement.
You can follow the Health Foundation on Twitter:http://www.twitter.com/healthfdn
4. About the Academy of Medical Sciences
The Academy of Medical Sciences is the independent body in the UK representing the diversity of medical science. Our mission is to promote medical science and its translation into benefits for society. The Academy's elected Fellows are the United Kingdom's leading medical scientists from hospitals, academia, industry and the public service. We work with them to promote excellence, influence policy to improve health and wealth, nurture the next generation of medical researchers, link academia, industry and the NHS, seize international opportunities and encourage dialogue about the medical sciences. http://www.acmedsci.ac.uk


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