Tag Archives: Medical Research Council

Britain, Health, Medical, Research, Science

New treatment options emerge for Parkinson’s disease…

RESEARCH LINKS THE DISEASE WITH A GENETIC DEFECT

… Scientists believe they are closer to treating debilitating Parkinson’s disease.

Parkinson’s disease is caused by a genetic defect that produces poor ‘housekeeping cells’, but scientists now believe they are closer to treating the debilitating condition.

Scientists have linked the disease to a genetic malfunction that prevents cells from clearing out defective mitochondria, metabolic generators within the body’s cells that supply energy. Dysfunctional mitochondria are potentially very harmful.

Normally, cells dispose of them through a ‘hazardous waste’ management system known as mitophagy. This causes bean-like bodies to be digested and broken down.

But researchers have now discovered and identified a biological pathway that allows mutations in a gene – called FBxo7 – to interfere with mitophagy.

For Parkinson’s sufferers, this leads to a build-up of defective mitochondria that may result in the death of brain cells.

The study, first published in the journal Nature Neuroscience, indicates that mitophagy might be the key to new treatment options for the disease.

Dr Helene Plun-Favreau is one of the researchers based at the Institute of Neurology at the University of London. She said:

… These findings suggest that treatment strategies that target mitophagy might be developed to benefit patients with Parkinson’s disease in the future.

… What makes the study so robust is the confirmation of defective mitophagy in a number of different Parkinson’s models, including cells of patients who carry a mutation in the Fbxo7 gene.

Co-author of the study, Dr Heike Laman, from Cambridge University, said:

… This research focuses the attention of the Parkinson’s disease community on the importance of the proper maintenance of mitochondria for the health of neurons.

… We are really only at the very beginning of this work, but perhaps we can use this information to enable earlier diagnosis for Parkinson’s disease patients or design therapies aimed at supporting mitochondrial health.

Professor Hugh Perry, chair of the neurosciences and mental health board at the Medical Research Council which part-funded the study, said the work raised ‘interesting questions’ about brain cell death related to Parkinson’s. He said:

… The more we understand about the basic molecular events which contribute to the onset and progression of Parkinson’s disease, the better placed we will be to develop treatments to stop it in its tracks.

Standard
Biotechnology, Health, Medical, Research, Science

Cell transplant gives hope for treating blindness…

A MAJOR ADVANCE

Scientists in Britain have made a major leap forward in efforts to treat blindness.

They have grown part of an eye in the laboratory and have extracted the light-sensitive cells which are the key to vision.

These cells were injected into mice, where they seemed to grow normally and formed the crucial connections between the eye and brain.

Such developments could pave the way for a treatment which could eventually give millions back their sight. It is hoped the first human patients could be treated in as little as five years. Transplanting just a small number of cells could have a big impact on quality of life.

Those who could benefit include men and women with age-related macular degeneration – the most common cause of blindness in the elderly. This condition affects more than 600,000 Britons and the number is expected to triple in the next 25 years as the elderly population swells.

There are few treatments for the condition – and no cure.

The research and experimentation is being carried out at University College London in which scientists are trying to replace damaged cells in the retina, the light sensitive ‘film’ at the back of the eye.

Last year, researchers used healthy cells from young mice to restore vision in adult animals. The results were said to be ‘dramatic’, with the treated animals able to quickly find their way out of a miniature swimming pool in dim light, while untreated ones swam around in circles.

Stem Cell Therapy for Blindness

The project’s lead-scientist, Professor Robin Ali, said the results amount to a ‘major advance’. His team took embryonic stem cells – ‘master cells’ capable of turning into other cell types and widely touted as a repair kit for the body – and used a cocktail of nutrients to coax them into turning into a retina.

They then raided the laboratory-grown retina for rods (key cells which pick up light and send it to the brain for conversion into images). The rods were then transplanted into the eyes of mice.

A retina has been grown in a dish before but Professor Ali’s team are the first to transplant cells from one successfully.

The journal Nature Biotechnology was the first to report the findings and has said that the lab-grown cells integrated into the existing eye was successful in forming the nerve connections needed to send information to the brain.

Professor Ali said:

… We are getting closer and closer to carrying out a trial.

However, the need to be highly confident that the treatment is safe and effective means that widespread use is at least 10-15 years away.

The Medical Research Council, which funded the team’s work, said in a statement:

… This study is an important milestone on the road to developing a widely available cell therapy for blindness.

Standard
Health, Medical, Research, Science, Scotland

Clot-busting drug boosts recovery for stroke victims…

STROKE victims who are given a vital clot-busting drug within six hours are more likely to have a long-lasting recovery than those who do not receive the treatment, new research has revealed.

A Scottish-led study of more than 3,000 patients across a dozen different countries reviewed the effects of the clot-busting drug rt-PA, which is given intravenously to patients who have suffered an ischaemic stroke.

An ischaemic stroke happens when the brain’s blood supply is interrupted by a blood clot. The damage caused can be permanent or fatal. Stroke symptoms include paralysis down one side and speech problems.

The international trial, led by Edinburgh scientists, found that 18 months after being treated with the drug, more stroke survivors were able to look after themselves. Patients who received rt-PA had fewer long-term problems with self-care and mobility, and experienced less pain and discomfort than those who did not.

Treatment also reduced the number of patients who needed help from other people from 51 per cent to 43 per cent.

However, the benefits of using rt-PA come at a price. Patients are at risk of death within seven days of treatment because the drug can cause a secondary bleed in the brain.

Stroke experts stress that these mortality figures need to be taken in context of deaths from stroke. Without treatment, one third of people who suffer a stroke die, with another third left permanently dependent and disabled.

Professor Peter Sandercock, the Chief investigator from Edinburgh’s University Centre for Clinical Brain Sciences, said:

… The trial team is delighted that, even for the elderly, rt-PA significantly improves life after stroke in the longer-term.

… Our results underline the benefits of treating patients with the drug as soon as possible and justify extending treatment to those aged 80 and over. We hope that this new data will encourage wider use.

Researchers said that because of the threat of death and disability, many stroke patients are prepared to take the early risks of being treated with rt-PA to avoid being disabled.

About half of those who took part in the trial were aged over 80.

The study builds on the results of the world’s largest ever trial of the drug, which was published last year. It found that treatment with rt-PA improved health for stroke survivors up to six months following an ischaemic stroke.

The latest results from the trial show the quality of life of stroke patients 18 months after receiving rt-PA.

It involved stroke patients in 12 countries between 2000 and 2011 – half of whom were treated with intravenous rt-PA and half of whom were not.

Researchers found that for every 1,000 patients given rt-PA within six hours of stroke, by 18 months, 36 more will not need help from others than if they had not been given the drug.

The multi-centre, randomised trial was supported by the University of Edinburgh, the Stroke Association UK, the Medical Research Council, the Health Foundation UK, NIHR Stroke Research Network and NHS Lothian Health Board. The results have been published in the academic journal The Lancet Neurology.

Standard