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BBC Radio 4 In Touch
29 April 2008

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Factsheet

GENE THERAPY
Guest: Professor Robin Ali

In Touch discuss the news that a team of ophthalmologists has successfully injected genetic material into the retina of an eighteen-year-old man with the form of retinal disease known as Leiber's. The patient, Stephen Howarth, is reporting substantially improved vision.

CONTACTS AND FURTHER INFORMATION

BBC News Online
http://news.bbc.co.uk/1/hi/health/7369740.stm

More information from Moorfields
http://www.moorfields.nhs.uk/Aboutus/Mediaoffice/Mediareleases/Patientinformationforgenetherapytrialresultsforinheritedblindness


BRITISH RETINITIS PIGMENTOSA SOCIETY
Tel. 0845 123 2354
http://www.brps.org.uk/home.php?home=yes
Registered charity dedicated to finding a treatment or cure for retinitis pigmentosa. They also provide information about RP.


VISION 20-20
The Vision 20-20 UK Strategy was launched earlier this month, with three main aims; to eradicate all preventable sight loss by 2020, to improve health care for people who are losing their sight, and to increase participation in society by blind and partially sighted people.

Mani Djazmi was at the launch and joined Peter to explain what it is about.

VISION 20-20
80 Elms Farm Road
Hornchurch
Essex
RM12 5RD
Tel: 01708 456832
Fax: 01708 446310
Email: m.brace@vision2020uk.org.uk
http://www.vision2020uk.org.uk/
The UK Vision Strategy is a VISION 2020 UK initiative led by RNIB to develop a unified plan for action on all issues relating to vision, across the four countries of the UK. The UK Vision Strategy has brought together people with sight loss, users of eyecare services, eye health and social care professionals and statutory and voluntary organisations for the very first time to set the direction for eye health and sight loss services across the UK.



GENERAL CONTACTS

RNIB
105 Judd Street
London
WC1H 9NE
Helpline: 0845 766 9999
Tel: 0207 388 1266 (switchboard/overseas callers)
Web: www.rnib.org.uk

The RNIB provides information, support and advice for anyone with a serious sight problem. They not only provide Braille, Talking Books and computer training, but imaginative and practical solutions to everyday challenges. The RNIB campaigns to change society's attitudes, actions and assumptions, so that people with sight problems can enjoy the same rights, freedoms and responsibilities as fully sighted people. They also fund pioneering research into preventing and treating eye disease and promote eye health by running public health awareness campaigns.


HENSHAWS SOCIETY FOR BLIND PEOPLE (HSBP)
John Derby House
88-92 Talbot Road
Old Trafford
Manchester
M16 0GS
Tel: 0161 872 1234
Email: info@hsbp.co.uk
Web: www.henshaws.org.uk
Henshaws provides a wide range of services for people who have sight difficulties. They aim to enable visually impaired people of all ages to maximise their independence and enjoy a high quality of life. They have centres in: Harrogate, Knaresborough, Liverpool, Llandudno, Manchester, Newcastle upon Tyne, Salford, Southport and Trafford.


THE GUIDE DOGS FOR THE BLIND ASSOCIATION (GDBA)
Burghfield Common
Reading
RG7 3YG
Tel: 0118 983 5555
Email: guidedogs@guidedogs.org.uk
Web: www.guidedogs.org.uk
The GDBA’s mission is to provide guide dogs, mobility and other rehabilitation services that meet the needs of blind and partially sighted people.


ACTION FOR BLIND PEOPLE
14-16 Verney Road
London
SE16 3DZ
Tel: 0800 915 4666 (info & advice)
Web: www.afbp.org
Registered charity with national cover that provides practical support in the areas of housing, holidays, information, employment and training, cash grants and welfare rights for blind and partially-sighted people. Leaflets and booklets are available.


NATIONAL LEAGUE OF THE BLIND AND DISABLED
Central Office
Swinton House
324 Grays Inn Road
London
WC1X 8DD
Tel: 020 7837 6103
Textphone: 020 7837 6103
National League of the Blind and Disabled is a registered trade union and is involved in all issues regarding the employment of blind and disabled people in the UK.


NATIONAL LIBRARY FOR THE BLIND (NLB)
RNIB Customer Services on 0845 762 6843
Email: cservices@rnib.org.uk
Web: www.nlb-online.org
The NLB is a registered charity which helps visually impaired people throughout the country continue to enjoy the same access to the world of reading as people who are fully sighted.

Trustees from the Royal National Institute of the Blind (RNIB) and the National Library for the Blind (NLB) have agreed to merge the library services of both charities as of 1 January 2007, creating the new RNIB National Library Service.


EQUALITY AND HUMAN RIGHTS COMMISSION DISABILITY HELPLINE (England)
FREEPOST MID02164
Stratford upon Avon
CV37 9BR
Tel: 08457 622 633
Textphone: 08457 622 644
Fax: 08457 778 878
Mon, Tue, Thu, Fri 9:00 am-5:00 pm; Wed 8:00 am-8:00 pm.
Enquiry: englandhelpline2@equalityhumanrights.com
www.equalityhumanrights.com


Equality and Human Rights Commission Helpline Wales
Freepost RRLR-UEYB-UYZL
1st Floor
3 Callaghan Square
Cardiff
CF10 5BT
0845 604 8810 - Wales main number
0845 604 8820 - Wales textphone
0845 604 8830 - Wales fax

9:00 am-5:00 pm, Monday to Friday (an out-of-hours service will start running soon)

Enquiry: waleshelpline@equalityhumanrights.com
www.equalityhumanrights.com





Equality and Human Rights Commission Helpline Scotland
Freepost RRLL-GYLB-UJTA
The Optima Building
58 Robertson Street
Glasgow
G2 8DU
0845 604 5510 - Scotland Main
0845 604 5520 - Scotland Textphone
0845 604 5530 - Scotland – Fax

9:00 am-5:00 pm, Monday to Friday (an out-of-hours service will start running soon)

Enquiry: scotlandhelpline@equalityhumanrights.com
www.equalityhumanrights.com


DISABLED LIVING FOUNDATION
380-384 Harrow Road
London
W9 2HU
Tel: 0845 130 9177
Web: www.dlf.org.uk
The Disabled Living Foundation provide information and advice on disability equipment.



The BBC is not responsible for external websites 

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Transcript

IN TOUCH

TX: 29.04.08 2040-2100


PRESENTER: PETER WHITE

PRODUCER: CHERYL GABRIEL



White
Good evening. Magic bullet or yet more false hope - today we try to sort out fact from fiction when it comes to gene therapy and eye disease. Or are the answers to be found more in politics, we ask whether 600 organisations coming together in a talking shop can make a real difference.

First though the news over the weekend that a team of ophthalmologists had successfully injected genetic material into the retina of an 18-year-old man with the form of retinal disease known as Lebers. Successfully in the sense that he was reporting substantially improved night vision, poor night vision is a feature of this kind of condition. Well the excitement is quite understandable, particularly for the young man in question, Steven Howarth. But we wondered, as we often tend to on this programme, what yet another rash of headlines, trumpeting revolutionary treatments and thousands saved from sight loss mean to the many people who already suffer from conditions like retinitis pigmentosa or RP or who know that they're likely to. So before we hear from one of the surgeons involved in the research about its implications, two reactions from people at the sharp end. First Fred Brown, who's been diagnosed with RP for many years, and is a self confessed cynic.

Brown
I always get the feeling that these facilities throughout the world - America especially and to a certain degree our country - that they're doing it for publicity to gain funding, but that's just me.

White
But from an emotional point of view I mean I'm just wondering how you react - the extent to which almost against your will there's an element of hope when you ...

Brown
Oh there's always an element of hope at these things but I tend not to get sort of carried along with it after having my condition for the last 12 or 13 years.

White
If you were asking questions now of the team what would you most like to know?

Brown
My first question - why did you pick Lebers as your study case, who's paying for it all and another question I'd like to ask is whether there's any connection between stem cell research and this particular treatment.

White
And then this e-mail from Rowena Forbes who lives in Edinburgh:

"Exciting stuff, however, what I haven't been able to discern is whether the treatment is likely to ameliorate the condition of someone with a relatively advanced case of RP, such as me? I'm well aware that the pigmentation of the retina leads to the irreversible death of cells and it's unlikely that this treatment, at least in its present form, could do anything about that but I'd like to hear more about how they think the treatment might develop and whether it's likely to lead to treatments for folk with already quite advanced sight loss before I get too excited."

Rowena Forbes. Well we're joined from the United States by James Bainbridge, consultant ophthalmic surgeon from Moorfields Eye Hospital in London and also a Wellcome Trust Fellow at UCL's Institute of Ophthalmology, who are collaborating with this project.

James Bainbridge, I'll put Fred and Rowena's points to you in a moment but first of all just explain what you've done in kind of layman's terms.

Bainbridge
Well we started a study to investigate the effects of gene replacement treatment in people with a form of Leber's congenital amaurosis, that's the form of severe childhood blindness. People with this particular condition have poor vision because they lack one of the genes critical for normal functioning of the retina. And so what we've done is a study to find out whether replacing that gene can have a beneficial effect on their vision.

White
And to put Fred's question to you where it seems relevant, why did you pick that one when of course there are a number of conditions which are less rare and would therefore help more people?

Bainbridge
Indeed. There are a number of features of this particular condition that make it a very good model system for addressing the questions that we have in mind. Leber's is unusual because the retinal cells are relatively healthy but they can't work properly because they lack the gene that's critical for retinal function. So in this particular case we can hope that by replacing the missing gene in the healthy cells we can improve the function.

White
But does that mean that you chose this because you'd got a better chance of getting success?

Bainbridge
Absolutely, we were interested, in the first instance, to find out whether in the most optimistic set of circumstances whether it could work at all and we were delighted to find that in one of the patients that we treated in only the first stage of the trial we've seen a significant improvement in night vision.

White
And I should say that we've had to change phones, so you might notice a slight technical difference. But James Bainbridge how measurable are your results? I mean we know that self-reporting of improved sight can be, for very understandable reasons, quite subjective and what about peripheral vision, how can you measure what you've achieved?

Bainbridge
I think it's very important to be sceptical about any of the results and we performed a number of different tests. We found that in two of the tests of retinal sensitivity that we've detected improvement in retinal sensitivity in Steven's eye, up to 25-100 times and this improvement was robust and measurable. We also asked Steven to perform a challenge by navigating a maze in dim light and found that he was substantially better at performing that test after the operation than before.

White
Crucially who could this affect and what about people who've had the condition - like Rowena and Fred - for quite a long time?

Bainbridge
We believe that these results suggest in the best case scenario this particular kind of treatment can be useful for certain individuals. It's very important to realise that this is a best case scenario and we're very pleased that it's worked, even in older patients and are more optimistic that it's going to work even better in children. But whether this kind of technology would be applicable to people with other retinal conditions - conditions other than Leber's due to RPE65 - will need to be explored in further clinical trials.

White
So I mean if people like Fred and Rowena came knocking on your door what would be the best you could offer them do you think?

Bainbridge
I think that what this offers them is the possible hope that similar treatments might be available for them in the future. These results suggest that the treatment for certain kinds of inherited retinal disease may be available within the next 5-10 years. For people who have relatively advanced degeneration and where many of the cells of the retina are already dead then this is treatment is not going to help. In that situation it would be necessary to replace those cells and there are a number of different lines of research looking at cell transplantation techniques and stem cell techniques that may well offer them some hope, people who have substantial cell loss, the possibility of stem cell treatment and other cell transplantation treatments does offer some real hope for the future.

White
Presumably in order to think in terms of doing this kind of work with the kind of numbers of people that the newspapers talk about again, you know we're talking in thousands, that would have pretty big financial implications?

Bainbridge
Yes indeed it would and it would need to be - the financial implications are big and they would need to be justified. But I'm optimistic that that can work.

White
And what about the implications for other conditions, including the big one in terms of numbers, Age-related Macular Degeneration?

Bainbridge
Age-related Macular Degeneration is not a simple genetic condition, in the sense that we're facing a single gene is not likely to cure the condition. However, the technology that we're developing may well have some application in terms of treatments for AMD in the future because technology offers one way to deliver drugs into the eye in a sustained way after a single injection, so that as opposed to many of the current treatments where repeated injections are required, this could potentially work after a single injection long term.

White
Can I ask you how you balance the need for publicity with - giving publicity which obviously helps with funding with the avoidance of raising false hope, the kind of false hope that this interview in a way is designed to allay?

Bainbridge
Well I'm very pleased to have the opportunity to discuss it on this programme and I think, as you say, there is a balance between publicity which is critical for funding bodies and raising awareness, even at government level for funding. And it's important to qualify that with the descriptions that I've been making, that this represents a small but significant incremental step in the progress towards some new treatments that offer real hope for people with low vision and blindness.

White
Right, but even with the best will in the world small incremental effect wasn't the impression given, even by some of the more serious newspapers yesterday.

Bainbridge
Although it's a small step it's a significant one because it's the first time that this has - this technique has been shown to be robustly effective in humans, in people.

White
How excited are you by this?

Bainbridge
It's very exciting for us because it's the culmination of 10-15 years work.

White
But you understand perhaps the difference between that excitement and allaying individual excitement among people who've perhaps been waiting for a solution to this for years, this may not yet be it?

Bainbridge
Yes absolutely and it's great to have the opportunity to explain that although this is a significant step, it does not mean that we can promise patients cures in the near future.

White
James Bainbridge, thank you very much indeed and sorry the telecommunications didn't quite match up with the surgery. There'll be more information about that, including links to the RP website, on our action line, more details later.

And from the search to help people one at a time to the equally important task of helping them in large numbers with politics. The loftily named Vision 20/20 UK Strategy was launched earlier this month with three main aims. To eradicate all preventable sight loss by 2020; improving healthcare for people who are losing their sight and increasing participation in society by blind and partially sighted people. The strategy is a collaboration between some of the giants of the visual impairment charity world - the RNIB, Action for Blind People, Guide Dogs - as well as over 600 societies and associations focusing on particular aspects of visual-impairment. Well they've all pledged to put their differences aside for the common good but will it make a difference?

Our reporter Mani Djazmi was at the launch:

Vox Pops
I would like to see greater information about what services are available on a whole range of issues. Being able to find information about emotional support - I'm talking about what my local organisations are doing, what my local social services departments are doing. For me that's important.

Accessible crossings. Being able to independently move around underground. I'd be getting spoken messages when I want them. Knowing what buses are coming. Getting information in an accessible form for our local authority. Having websites that I can easily use. Those for me, as someone who doesn't have a health condition in relation to sight loss but is totally blind, those are the key things for me.

The most important thing is when people are referred or they're diagnosed that they've got a visual-impairment that they should have help with low visual aids and be referred to local societies where they can be helped. But unfortunately not all the societies are as good as they should be.

Eight years ago I was working for RNIB as an individual support project manager and looking at the individual support needs of older people. Where I am now is that I'm a mum at home looking after a child, I don't want to come along in eight years time and hear the same stuff but in a different jargon.

White
Well Mani is here with me. Mani, having heard those comments everyone will have their own personal wish list of course but this is a pretty ambitious project.

Djazmi
It is and they're aiming to achieve all those aims in the next five years. It's the first time that such a wide ranging group of professionals and service users, as we're called, have come together and basically come up with an action plan on the whole spectrum of sight loss. One of the aims you mentioned is to increase participation in society by blind and partially-sighted people, this obviously includes getting more of us into work and reducing that much touted statistic that 75% of visually-impaired people of working age are unemployed.

Well Mike Brace OBE, who's totally blind and a member of the Strategic Advisory Group, says that it's not just about getting blind people into work, but also making sure that people who are already working and lose their sight know where to find help to enable them to stay in employment. Mike says that the key battle for the strategy is winning over the hearts and minds of employers and making them aware that visually-impaired people are just as capable of doing most jobs as sighted people.

Brace
They are people themselves that may go blind. The issue that somehow it only happens to someone else is not true. So we're saying to them - what would you feel like if next week you had a sight loss issue, would you say then you didn't want to keep your job or that you would be put out to grass because you would be no longer of any use? Of course not. It's about how you would actually wish to be treated if the situation was reversed.

Djazmi
So in your opinion can this strategy increase the number of employed visually-impaired people?

Brace
I think we can. I think if it's perceptions, change, if people then are looking at what people can do, what their abilities are and not what their disability is I think that will help. And the third element is real practical information and advice about what's available to people that would make those adaptations and those variations actually meaningful to them to be employable, not as a temporary, not as a volunteer, but as a paid position making a contribution.

White
Mani, people like to say these things can be changed by attitude, it's not all about money, but we all know money's involved, so how much support is the strategy getting from the government?

Djazmi
Well the health secretary - Alan Johnson - was out of the country so he sent a recorded message on DVD:

Johnson
The vision strategy has already involved an impressive array of cross-sector working in its development. I'm sure this will continue and that the NHS and other agencies will work together to deliver the benefits to patients that we all want to achieve. I commend this strategy and thank all those who've contributed towards its launch today.

Mr Johnson also said that the strategy makes a valuable contribution to the emerging debate on the future of adult social care and that it's an addition to the government's policy considerations. Pick the bones out of that. So possibly not quite the ringing endorsement of the strategy that the likes of Lesley Ann Alexander would have wanted. Lesley Ann is chief executive of RNIB and chair of the group leading the development of this strategy.

Alexander
Well he is actually supportive of it, we've had a lot of support from all of the governments across the UK, we've certainly got Ann Keen, the minister for health, coming to speak at an event that we're hosting to bring together all of the English parts of the strategy implementation. And of course it will take that very senior leadership from government to ensure that proper resources are put in place.

Djazmi
And presumably those resources that you refer to basically comes down to money, so how much will it cost to implement this strategy?

Alexander
Well it's not just money, it's money and time and energy and effort. At the moment I know that the money there is available isn't being spent properly because of the duplication in the sector and because we don't always view people as individual. Undoubtedly there needs to be more money but if we can present a united front to government I'm more confident that we will be able to get proper resources in place than I would be if we were very fragmented and all asking for different things.

Djazmi
So how valuable is the government's money to you, I mean - in five years time if the government doesn't come up with what you want from them how much progress do you think will have been made?

Alexander
If government don't come up with new money to fill the gaps that there are after we've all made better use of the resources that currently exist the outcomes of the strategy won't be as good as they can be. But the outcomes of the strategy will be good in any event because we're determined, as a group, to make the very best of what we have already.

White
Of course charities like the RNIB have asked the government for money before without very much success haven't they.

Djazmi
Indeed, there's the good old right to read campaign, which is still trying to get more books in Braille and large print after more than five years of hammering away. In fact I asked Lesley Ann Alexander if she could give any assurances that the UK vision strategy will be more fruitful?

Alexander
I can't guarantee anything, I think there are very few things in this life that you can guarantee. But I have confidence that because the sector has come together to develop the UK vision strategy that we have a much better chance of people taking us seriously.

Djazmi
Do you expect this conference to - by this afternoon - to give an answer to how the strategy's to be implemented and the way forward?

Alexander
The conference today is very much about people who geographically work in a particular region, area, or country of the UK, it's very much about the specialists in certain fields coming together to debate how we're going to implement it. So I would be very disappointed if we came out at the end of today without some much clearer plans around implementation. We all know where we want to go and now we have to work out how.

Djazmi
Well the conference is over, Lesley Ann Alexander, how is the strategy going to be implemented?

Alexander
I think we're much, much closer than we were at the start of the day. I think the workshops where people have met in geographical groups to work out what they're going to do locally have been very, very successful because after all we could have all the words in the world but unless local people get a grip of the local issues then we're never going to deliver the aims of the strategy.

White
Lesley Ann Alexander ending that report from Mani. Mani, thanks very much.

Now that's it for today but you can call our action line on 0800 044 044. From me, Peter White, my producer Cheryl Gabriel and the team, goodbye.



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