Medical breakthroughs are rarely the result of ‘eureka’ moments. More often, they are the product of years of rigorous research – in the laboratory, and in clinical trials. Over the course of the Centenary Campaign, organisations, alumni and friends of the University have donated almost £26 million to support medical research at Bristol. That support is helping turn clinical knowledge into practical solutions, giving thousands of people and their families hope for the future.
Multiple sclerosis (MS) is a disabling, lifelong neurological condition, that occurs when the immune system attacks the coating around the nerve fibres (known as myelin), disrupting the transmission of electrical impulses around the nervous system. Around 100,000 people in the UK suffer from MS. We don’t yet know what causes it, nor how to cure it.
Thanks to significant philanthropic support, researchers at Bristol are testing whether stem cells derived from a patient’s bone marrow can help repair the damaged nerve tissue and improve function. In 2009/10, they conducted a world first: a safety and feasibility trial of a ground-breaking treatment, where a patient’s own stem cells were injected straight into the bloodstream. That same treatment is now being tested in a largescale clinical trial.
If the trial is successful, the therapy could be approved in just a few years. The team is carrying out further tests to refine the treatment, and are also working to establish both how nerve damage in MS occurs and, ultimately, how it might be prevented.
Almost half a million people in the UK suffer from Alzheimer’s disease. It is the most common cause of dementia and, currently, incurable. Caring for patients with dementia costs the UK £26 billion – more than cancer, heart disease and diabetes combined – but the emotional cost to patients and their families is immeasurable.
Alzheimer’s disease occurs when a protein known as amyloid beta (Aß) is not broken down by enzymes in the brain. Aß builds up and activates other damaging biochemical processes that kill healthy brain cells.
Members of Bristol’s Dementia Research Group have access to one of the largest brain banks in the UK and, thanks to donations like yours, are using state-of-the-art technology to store high volumes of data about their patients. They have already found links between Alzheimer’s disease and enzymes involved in regulating blood pressure and blood supply to the brain. Now they are conducting a clinical trial to test whether Losartan, a drug commonly used to treat high blood pressure, might also help combat Alzheimer’s disease.
If the results of the trial are positive, the next stage will be a definitive international study. Scientists at Bristol are already acting as advisors and co-investigators in three global trials involving similar drugs, and have started further research into what links the different neurological pathways that are activated in Alzheimer’s disease. Developing effective and efficient treatments that can target these pathways – perhaps using drugs already available for other conditions – will mean thousands of patients and their families could soon enjoy a much improved quality of life.
Every day, a dozen babies in the UK are born with a heart defect. Thankfully, today, most of these children can look forward to good quality of adult life. But, because current treatments rely on artificial implants, they’ll need frequent operations as they grow up.
Stem cells from the umbilical cord (usually discarded after birth) might hold the key to a new generation of implants – implants that both grow at the same rate as the children they’re used to treat, and contain the child’s own DNA, reducing the risk of rejection after transplant.
Thanks to two major donations, scientists at Bristol have completed the first two phases of research, and are now preparing to start a clinical trial in newborn babies.
Bowel cancer is the second most common cause of cancer death in the UK. Late diagnosis often means a poor prognosis: advanced tumours tend to be beyond the reach of chemotherapy and radiotherapy.
Evidence suggests that up to 80 per cent of bowel cancers may actually be preventable, and common medicines like aspirin could be the answer. Scientists at Bristol were the first in the world to show that, in the laboratory, aspirin induces bowel cancer cells to commit ‘cell suicide’ – a far-reaching breakthrough, made possible thanks to a number of major donations. The team has also been involved in an international clinical study that has shown aspirin can reduce the risk of bowel cancer in very high-risk patients.
This research, as well as studying how genetic changes to cells can lead to cancer, will inform the development of new, targeted and individualised treatments for tumours resistant to conventional therapies.
Every year, around one in 1,000 full-term babies in the UK suffer severe oxygen deprivation during birth. Seventy per cent of these children will either die or suffer permanent brain damage. In the 1990s, Professor Marianne Thoresen showed that therapeutic cooling (reducing a baby’s body temperature by between 3 and 4°C) reduced that risk to 50 per cent. This treatment is now widely used in neonatal intensive care units across the UK, but a 50 per cent risk of brain damage is still too severe.
Professor Thoresen’s team is now working on a ground-breaking treatment to reduce the risk further. Research in the lab suggests that, if a newborn inhales xenon gas during therapeutic cooling, their brain receives double the protection of cooling alone. And thanks to one generous donor, Professor Thoresen is conducting a clinical trial which has already shown promising results. Her work, together with other cutting-edge research in obstetric and neonatal practice taking place at Bristol, received special recognition in 2014, when the University was awarded the Queen’s Anniversary Prize for Higher Education.
The most difficult patients to treat are those with the most severe brain injury. Professor Thoresen wants to determine when, and for how long, xenon gas (together with therapeutic cooling) needs to be administered to deliver the best results. By recording electrical brain activity, observing changes in blood results and using MRI scans, she will be able to predict the severity of brain damage when the child is just ten days old. The current clinical trial aims to validate these biomarkers by comparing this prediction with the results of a functional examination (the current method of diagnosing brain damage) at 18 months of age. Developing effective treatments and methods of early diagnosis is essential for improving the future quality of life for these children.
Around one in 500 people in the UK suffer from Parkinson’s disease, for which there is currently no cure. Parkinson’s is caused by a loss of dopamine-producing neurons (nerve cells) in the brain. As dopamine plays a key role in controlling body movement, tremors, stiffness and slow movement are all common symptoms of this progressive, degenerative condition..
Partly supported by a significant donation, Bristol scientists are investigating whether stem cells can be induced to act as dopamine-producing neurons and restore function to the affected parts of the brain. They are also using stem cells taken from patients’ skin to model the disease in the laboratory – a technique they hope will help them better understand why dopamine-producing neurons die, and prevent them from doing so.
By comparing cells from sufferers with those from non-sufferers, the team also hope to establish whether some people have a genetic predisposition to the disease. Ultimately, their research will open up new avenues for research into treatments and possibly even ways to reverse the disease.