Largest gene therapy trial for cystic fibrosis begins
The trial will see patients 'breathe in' the gene therapy using a nebulizer.
(Medical Xpress) -- British scientists are to carry out the largest trial anywhere in the world of a gene therapy for cystic fibrosis.
A consortium of researchers from Oxford University, Imperial College London and the University of Edinburgh will start enrolling patients on the trial this month.
The UK Cystic Fibrosis Gene Therapy Consortium hopes that the study will show for the first time whether the gene therapy they have developed can improve the health of patients. The phase two clinical trial will involve 130 cystic fibrosis patients using an inhaler to breathe in a working copy of the cystic fibrosis gene once a month for a year.
The trial will go ahead thanks to £3 million in funding from the Medical Research Council (MRC) and the National Institute for Health Research (NIHR) that is announced today.
Additional laboratory studies to develop a potentially more efficient delivery method for the gene therapy will receive a further £1.2 million in support from the MRC.
"No one has ever done a gene therapy study like this in cystic fibrosis before," said Dr. Deborah Gill of the Nuffield Department of Clinical Laboratory Sciences at Oxford University. "Its a worldwide first in terms of the length of the study, the number of patients involved and the number of doses of gene therapy. By giving the therapy over a whole year, we will have the best chance yet of seeing an improvement in patients."
Professor Eric Alton, the coordinator for the consortium from Imperial College London, said: 'Conventional treatments have extended the life expectancy for people with cystic fibrosis. Were hoping that this therapy will achieve a step change in the treatment of cystic fibrosis that focuses on the basic defect rather than just addressing the symptoms.
"This trial will assess if giving gene therapy repeatedly for a year will lead to the patients' lungs getting better. Eventually we hope gene therapy will push cystic fibrosis patients towards a normal life expectancy and improve their quality of life significantly."
Cystic fibrosis is an inherited disease that affects around 9,500 people in the UK. There is currently no cure and the only available treatments rely on alleviating the symptoms, not treating the underlying cause. The average life expectancy for cystic fibrosis patients is only 35 years.
The disease is caused by a single faulty gene that leads to thick, sticky mucus to build up in the lungs and digestive system.
The researchers in the UK Cystic Fibrosis Gene Therapy Consortium have developed a successful method for delivering a working copy of the defective gene directly into the lungs of patients.
The gene is administered via a nebulizer much like inhalers used in asthma. Patients simply inhale a fine mist of fat globules which carry the DNA for the gene wrapped up inside.
The researchers have previously shown in smaller-scale clinical trials that their nebulizer approach can get a working copy of the cystic fibrosis gene into the cells of the lung, and that the gene continues to work for a period of weeks and months.
The new clinical trial will assess whether the gene therapy can provide measurable clinical benefits for people with cystic fibrosis. That is: whether it can actually improve peoples lung function, and reduce the amount of mucus, inflammation and infection seen in patients.
Patients will be seen at the Royal Brompton Hospital, London, and the Western General Hospital and the Royal Hospital for Sick Children, Edinburgh.
The second lab-based study, with a further £1.2 million in funding from the MRC, will investigate a potentially more efficient delivery method for the gene therapy. The aim is to use a modified virus to carry the replacement gene into the lungs, which could lead to an even more effective treatment in the future.
Dr. Gill said: "The new virus delivery approach has never been tried before but it could be more efficient. It is specifically designed to deliver the gene therapy to the lungs, but it will take several more years of development before it gets to the point of human trials."
The consortiums research, which has been supported by the Cystic Fibrosis Trust for a decade, had faced an uncertain future due a funding shortfall. The new funding from the MRC and NIHR will ensure the next stage of the research can continue as planned.
Professor Eric Alton of Imperial College London said: "With funding for this key part of the consortium's program secure, we will begin preparations for the trial immediately."
Dr. Gill said: "A lot of cystic fibrosis patients in this country have either been directly involved in trials or in fundraising to support the research. That this new trial is going ahead is a really great result for everyone. It could find out if gene therapy has a chance of working for all cystic fibrosis patients."
Provided by Oxford University
- Breathe deep: Which patients could benefit from inhaled steroids in cystic fibrosis? Nov 29, 2007 | not rated yet | 0
- Scientists develop first ever drug to treat 'Celtic gene' in cystic fibrosis sufferers Jun 20, 2011 | not rated yet | 0
- Cystic fibrosis treatments may have unseen long-term benefits Jul 16, 2009 | not rated yet | 0
- Severity of cystic fibrosis may be determined by presence of newly-identified modifier genes May 25, 2011 | not rated yet | 0
- Researchers take first step to create cystic fibrosis model using pigs Mar 07, 2008 | not rated yet | 0
- Motion perception revisited: High Phi effect challenges established motion perception assumptions Apr 23, 2013 | 3 / 5 (2) | 2
- Anything you can do I can do better: Neuromolecular foundations of the superiority illusion (Update) Apr 02, 2013 | 4.5 / 5 (11) | 5
- The visual system as economist: Neural resource allocation in visual adaptation Mar 30, 2013 | 5 / 5 (2) | 9
- Separate lives: Neuronal and organismal lifespans decoupled Mar 27, 2013 | 4.9 / 5 (8) | 0
- Sizing things up: The evolutionary neurobiology of scale invariance Feb 28, 2013 | 4.8 / 5 (10) | 14
Why is zone 1 in liver more prone to ischemic injury?
May 23, 2013 Hi, Is it because around central vein, there is only deoxygenated blood from the vein where as in the periphery there is hepatic artery. Also why...
How can there be villous adenoma in colon, if there are no villi there
May 22, 2013 As title suggest. Thanks :smile:
How can there be a term called "intestinal metaplasia" of stomach
May 21, 2013 Hello everyone, Ok Stomach's normal epithelium is simple columnar, now in intestinal type of adenocarcinoma of stomach it undergoes "intestinal...
Pressure-volume curve: Elastic Recoil Pressure don't make sense
May 18, 2013 From pressure-volume curve of the lung and chest wall (attached photo), I don't understand why would the elastic recoil pressure of the lung is...
If you became brain-dead, would you want them to pull the plug?
May 17, 2013 I'd want the rest of me to stay alive. Sure it's a lousy way to live but it beats being all-the-way dead. Maybe if I make it 20 years they'll...
MRI bill question
May 15, 2013 Dear PFers, The hospital gave us a $12k bill for one MRI (head with contrast). The people I talked to at the hospital tell me that they do not...
- More from Physics Forums - Medical Sciences
More news stories
By discovering the new mechanism by which estrogen suppresses lipid synthesis in the liver, UC Irvine endocrinologists have revealed a potential new approach toward treating certain liver diseases.
Medical research May 23, 2013 | 5 / 5 (1) | 0 |
Aortic arch pulse wave velocity, a measure of arterial stiffness, is a strong independent predictor of disease of the vessels that supply blood to the brain, according to a new study published in the June issue the journal ...
Medical research May 23, 2013 | not rated yet | 0
Since the discovery of Prontosil in 1932, sulfonamide antibiotics have been used to combat a wide spectrum of bacterial infections, from acne to chlamydia and pneumonia. However, their side effects can include serious neurological ...
Medical research May 23, 2013 | 3 / 5 (1) | 0 |
Scientists at the National Institutes of Health report they have discovered in mouse studies that a small molecule released in the spinal cord triggers a process that is later experienced in the brain as ...
Medical research May 23, 2013 | 5 / 5 (3) | 0 |
Spanish researchers have discovered that the daily clearance of neutrophils from the body stimulates the release of hematopoietic stem cells from the bone marrow into the bloodstream, according to a report published today ...
Medical research May 23, 2013 | 5 / 5 (2) | 0
Coenzyme Q10 decreases all cause mortality by half, according to the results of a multicentre randomised double blind trial presented today at Heart Failure 2013 congress. It is the first drug to improve heart failure mortality ...
10 hours ago | 5 / 5 (2) | 5
(HealthDay)—Animals make great companions for senior citizens, but elderly people who always drive with a pet in the car are far more likely to crash than those who never drive with a pet, researchers have ...
2 hours ago | not rated yet | 0
Heart failure accelerates the aging process and brings on early andropausal syndrome (AS), according to research presented today at the Heart Failure Congress 2013. AS, also referred to as male 'menopause', was four times ...
10 hours ago | not rated yet | 1
Mortality and length of stay are highest in heart failure patients admitted in January, on Friday, and overnight, according to research presented today at the Heart Failure Congress 2013. The analysis of nearly 1 million ...
10 hours ago | not rated yet | 0
(AP)—Department of Justice lawyers have again asked a federal appeals court in New York to delay lifting age restrictions and prescription requirements on an emergency contraceptive popularly known as the morning-after ...
10 hours ago | not rated yet | 0