Research Training Fellowship: Dr Valerie Saw.
This research was completed on 16 July 2009
Published on 1 June 2006
Committed to battling blindness
Each year, Action Medical Research awards these prestigious grants to help the brightest and best doctors and scientists develop their career in medical research. Dr Saw's grant of £185,610 will fund a three-year search for new ways to treat a devastating form of blindness.
Dr Valerie Saw is a dedicated and enthusiastic young doctor, who graduated from medical school with the top mark in her year. Now she's focusing her talents on helping patients who suffer a devastating form of blindness, known as MMP.
MMP causes debilitating eye pain and sensitivity to light. Up to 30% of patients with diseased eyes go on to become blind.1 Some undergo distressing, chemotherapy-like treatment, which doesn't always work.
Based at the prestigious Institute of Ophthalmology, University College London, Dr Saw will use her fellowship to search for better treatments for people with MMP
I am extremely excited about the opportunity to do this research. I am determined to help find better treatments for patients with MMP, and to make steps towards preventing their disheartening progression to blindnessDr Valerie Saw.
The relentless progression to blindness
In this fellowship, Dr Saw is focusing on a distressing condition called mucous membrane pemphigoid, or MMP.
People with MMP suffer debilitating pain in their eyes, and sensitivity to light, due to severe inflammation. Up to 30% of patients develop scars on the surface of their eyes that are so bad they eventually go blind. Sight loss can be rapid - sometimes taking just six months.2
MMP is poorly understood and there is no cure. An aggressive, chemotherapy-like treatment can control inflammation in patients' eyes, but it doesn't always stop them from going blind. To make matters worse, treatment can cause severe side effects, such as sterility, a lowered immunity to infection and an increased risk of cancer.
MMP doesn't just affect the eyes. Patients can suffer a range of other symptoms, including difficulties with swallowing, breathing and passing urine. Some need life-saving surgery to help them swallow or breathe.
'It seems very unfair that this devastating disease stops patients from enjoying their lives,' says Dr Saw. 'So I am thrilled that this fellowship will allow me to carry out this much-needed research, which has the potential to improve the well-being of people who would otherwise be quite desolate.'
In the clinic and the laboratory
Dr Saw's research has a two-fold approach. Firstly, she is conducting a clinical trial to find out whether intravenous steroids can benefit patients with MMP by reducing their eye pain and sensitivity to light.
Dr Saw's second approach is to take tiny biopsies from the surface of patients' eyes so she can investigate an important but poorly understood aspect of the disease process: the molecular mechanisms that lead to scarring of patients' eyes. She hopes to reveal whether any potential new treatments might reduce that scarring.
'I am very grateful to all the generous people who have contributed to this funding and to those who devote their time to Action Medical Research,' says Dr Saw.
|Project Leader||Dr Valerie Saw MBBS|
|Location||Department of Clinical Ophthalmology, Institute of Ophthalmology, London|
|Grant awarded||1 March 2006|
|Start date||17 July 2006|
|End date||16 July 2009|
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From alleviating pain to saving sight
Almost 2,000 people in the UK have MMP, with nearly 200,000 suffering worldwide.3 Whilst the illness most commonly afflicts the elderly, aged 60 to 69, it can also strike young patients and children, whose illness tends to be more severe. In the shorter term, if the steroid treatment proves successful, these patients may be spared from some of the severe eye pain and sensitivity to light that MMP causes.
Longer-term benefits may come from Dr Saw's work in the laboratory. The ultimate goal is to develop a treatment that stops scarring, saves the sight of more patients than conventional, chemotherapy-like treatment and causes fewer side effects. Dr Saw hopes her laboratory work will pinpoint whether any drugs from a class of anti-scarring agents are possible candidates for development.
'Preventing the relentless progression to blindness will prevent the overwhelming and frustrating loss of independence, dignity and enjoyment of life which this entails.' explains Dr Saw.
Potential broader benefits
Anti-scarring therapies may also help prevent the disabling scarring that occurs elsewhere in the bodies of patients with MMP - for example, in the throat and breathing passages. Other patients may even benefit too: 'My research may also help patients suffering other conditions that cause blindness by scarring, including trachoma and glaucoma which are two of the leading causes of blindness worldwide,' says Dr Saw.
Dr Saw's research will involve a clinical trial and laboratory studies. She hopes the clinical trial will reveal whether adding intravenous steroids to conventional, chemotherapy-like treatment for MMP can help control inflammation in patients' eyes.
Twenty patients will be randomly assigned to two groups. Patients in one group will receive just conventional treatment; the others will receive intravenous steroids as well. 'More rapid and effective control of inflammation would reduce eye pain and avoidance of light,' explains Dr Saw.
Dr Saw will also conduct a series of in-depth laboratory studies to find out more about MMP. Doctors already know that it's an autoimmune disease - the patient's own immune system causes the damage to their eyes. But exactly how the scars develop is poorly understood.
Dr Saw will take tiny biopsies of the skin on the surface of patients' eyes and study scar cells and immune cells grown in the laboratory. Dr Saw plans to investigate the molecular processes that underlie scarring, including the relationship between inflammation and scarring. She will also explore how treatments - both conventional treatment and potential new treatments such as anti scarring therapies - might influence the formation of scars.
I am extremely excited about the potential of this research. I hope to improve understanding of MMP and propose potential new therapies for patients with this difficult and devastating disease.Dr Valerie Saw
- Hardy KM, Perry HO, Pingree GC, Kirby TJ Jr. Benign mucous membrane pemphigoid. Arch Dermatol 1971;104: 467-75
- Foster CS. Cicatricial pemphigoid. Trans Am Ophthal Soc 1986;84:527-659
- Bernard P, Vaillant L, Labeille B, Bedane C, Arbeille B, Denoeux JP, Lorette G, Bonnetblanc JM, Prost C. Incidence and distribution of subepidermal autoimmune bullous skin diseases in three French regions. Bullous Diseases French Study Group. Arch.Dermatol. 1995;131:48-52