Professor Robin Ali, "a brilliant young scientist"
Statement 27 April 2008
27th April 2008 23:25
RP Fighting Blindness' statement on the announcement from Moorfields Eye Hospital of initial results of Gene Therapy clinical trials to treat Lebers Congenital Amaurosis (a form of retinitis pigmentosa affecting the young)
Note: Detailed information about RP, about research to date, and about the charity can be found at www.brps.org.uk.
All of us at The British Retinitis Pigmentosa Society (RP Fighting Blindness) are excited by the progress that has been made and announced today. The demonstrable difference this trial has already made to just one person’s life is an important milestone on our way to achieving our key objective – a treatment for RP.
The charity is proud to have supported Professor Robin Ali’s work for several years. We provided grant money from our Research Fund to enable him to prove the principles behind retinal gene therapy and now, in collaboration with Foresight (a similar RP research charity based in Dubai), we are supporting the current trials which run until 2010.
We hope that the great results achieved by Robin and his team will encourage the development, funding and commissioning of similar projects tackling this and other flawed genes that cause RP, as we must point out that these trials are about just one.
Our member and the clinical trial patient whose vision has improved, Steven Howarth, describes “a small change – but (one that) makes a big difference to me.” A fellow patient describes all the clinical trial volunteers as “pioneers”. Both of these messages are important. The first tells us how even a small improvement in vision can really help someone maintain their independence, and that our investment in this work is undoubtedly worthwhile. The second illustrates very well the stage we are at with Gene Therapy - a very early one.
This organisation has been striving for 32 years for a cure for RP and represents some 2,500 members, all of whom will be given fresh hope from this week’s news. However we urge that optimism, though justified, is tinged with caution, because these are still ‘early days’. Robin himself said “These results give us great confidence that the technique is safe and can bring real benefit to patients with impaired vision. While we’re very excited about the improvement in Steven’s vision, it’s important to emphasise that gene therapy is still an experimental treatment not yet generally available to patients. The technique will be tested in other patients with LCA and we also hope to begin trials for other forms of retinal disease in the future.”
Professor Tony Moore, of the RP Fighting Blindness Medical Advisory Board, and a Trustee of the charity, added “It is very encouraging to see that this treatment can work, even in young adults who have severely advanced disease. We anticipate an even better outcome in the younger patients we are now beginning to involve as the trial proceeds, as we will be treating the disease in the early stages of its development.”
We are renewing our own appeals for contributions to our own Research Fund, in order that we can continue to influence the research agenda. We also urge the Department of Health and other agencies to make further monies available for this type of work. At this stage we do not know what Professor Ali and his team may propose as ‘the next step’, but whatever that might be, we would be very disappointed if the science, and further trials, were not able to continue simply because of a shortage of funds.
Recent advances in stem cell therapy, the announcement last week of the successful insertion of electronic retinal implants at Moorfields, and now these groundbreaking results, make this a truly exciting time for us at RP Fighting Blindness.
We would like to take this opportunity to thank Professor Ali’s team, and all the scientists whose work has preceded and contributed to the trials, for their hard work and dedication.
David Head, Chief Executive, said "“The initial results from Professor Ali’s project offer a ray of hope for people with retinitis pigmentosa (RP). The charity has been fighting RP for over 30 years and we are all really excited by this progress. In particular we have been supporting Professor Ali’s work for several years and to see it paying dividends like this is such fantastic news.
“Professor Ali is an outstanding eye scientist and I am sure we will continue to support his work. Gene therapy is one of the most promising avenues of research in our hunt for a treatment for RP and we are privileged to have such a brilliant young researcher striving for the same result.
“Of course, we must temper our excitement and enthusiasm with an acknowledgment that these are very early days, and the trial is working on one flawed gene. There are many others that are responsible for RP. However to have such a promising result from the first trials bode well for the future and for the commissioning of further trials. This is a crucial milestone."
Lynda Cantor MBE who founded the charity when she was diagnosed with RP in the mid-70’s and has driven the search for a cure ever since, is absolutely delighted. When told of the news, she responded “I’ve said many times over the years that if I lived to see the day that one person’s sight was improved as a result of our work I would be happy.”
Lynda, who is blind as a result of the disease, also spoke about Professor Ali. “Robin is a brilliant young scientist and I am so proud of him, and of the fact that we have supported his work from an early stage. His dedication to this research has been outstanding, he deserves the accolades.
“Proving the principles behind gene therapy, and that it can be used safely in the retina, is fantastic news. But don’t forget, this trial was about one particular gene, we have treated 1 person. There are some 25,000 people – and their families – dealing with sight loss through RP in the UK alone. We desperately need more funding to be able to back those scientists, like Robin, that have proved they can do this. Only then can we really expect to be able to ‘break the chain’ of inherited blindness.”
RP Fighting Blindness' statement on the announcement from Moorfields Eye Hospital of initial results of Gene Therapy clinical trials to treat Lebers Congenital Amaurosis (a form of retinitis pigmentosa affecting the young)
Note: Detailed information about RP, about research to date, and about the charity can be found at www.brps.org.uk.
All of us at The British Retinitis Pigmentosa Society (RP Fighting Blindness) are excited by the progress that has been made and announced today. The demonstrable difference this trial has already made to just one person’s life is an important milestone on our way to achieving our key objective – a treatment for RP.
The charity is proud to have supported Professor Robin Ali’s work for several years. We provided grant money from our Research Fund to enable him to prove the principles behind retinal gene therapy and now, in collaboration with Foresight (a similar RP research charity based in Dubai), we are supporting the current trials which run until 2010.
We hope that the great results achieved by Robin and his team will encourage the development, funding and commissioning of similar projects tackling this and other flawed genes that cause RP, as we must point out that these trials are about just one.
Our member and the clinical trial patient whose vision has improved, Steven Howarth, describes “a small change – but (one that) makes a big difference to me.” A fellow patient describes all the clinical trial volunteers as “pioneers”. Both of these messages are important. The first tells us how even a small improvement in vision can really help someone maintain their independence, and that our investment in this work is undoubtedly worthwhile. The second illustrates very well the stage we are at with Gene Therapy - a very early one.
This organisation has been striving for 32 years for a cure for RP and represents some 2,500 members, all of whom will be given fresh hope from this week’s news. However we urge that optimism, though justified, is tinged with caution, because these are still ‘early days’. Robin himself said “These results give us great confidence that the technique is safe and can bring real benefit to patients with impaired vision. While we’re very excited about the improvement in Steven’s vision, it’s important to emphasise that gene therapy is still an experimental treatment not yet generally available to patients. The technique will be tested in other patients with LCA and we also hope to begin trials for other forms of retinal disease in the future.”
Professor Tony Moore, of the RP Fighting Blindness Medical Advisory Board, and a Trustee of the charity, added “It is very encouraging to see that this treatment can work, even in young adults who have severely advanced disease. We anticipate an even better outcome in the younger patients we are now beginning to involve as the trial proceeds, as we will be treating the disease in the early stages of its development.”
We are renewing our own appeals for contributions to our own Research Fund, in order that we can continue to influence the research agenda. We also urge the Department of Health and other agencies to make further monies available for this type of work. At this stage we do not know what Professor Ali and his team may propose as ‘the next step’, but whatever that might be, we would be very disappointed if the science, and further trials, were not able to continue simply because of a shortage of funds.
Recent advances in stem cell therapy, the announcement last week of the successful insertion of electronic retinal implants at Moorfields, and now these groundbreaking results, make this a truly exciting time for us at RP Fighting Blindness.
We would like to take this opportunity to thank Professor Ali’s team, and all the scientists whose work has preceded and contributed to the trials, for their hard work and dedication.
David Head, Chief Executive, said "“The initial results from Professor Ali’s project offer a ray of hope for people with retinitis pigmentosa (RP). The charity has been fighting RP for over 30 years and we are all really excited by this progress. In particular we have been supporting Professor Ali’s work for several years and to see it paying dividends like this is such fantastic news.
“Professor Ali is an outstanding eye scientist and I am sure we will continue to support his work. Gene therapy is one of the most promising avenues of research in our hunt for a treatment for RP and we are privileged to have such a brilliant young researcher striving for the same result.
“Of course, we must temper our excitement and enthusiasm with an acknowledgment that these are very early days, and the trial is working on one flawed gene. There are many others that are responsible for RP. However to have such a promising result from the first trials bode well for the future and for the commissioning of further trials. This is a crucial milestone."
Lynda Cantor MBE who founded the charity when she was diagnosed with RP in the mid-70’s and has driven the search for a cure ever since, is absolutely delighted. When told of the news, she responded “I’ve said many times over the years that if I lived to see the day that one person’s sight was improved as a result of our work I would be happy.”
Lynda, who is blind as a result of the disease, also spoke about Professor Ali. “Robin is a brilliant young scientist and I am so proud of him, and of the fact that we have supported his work from an early stage. His dedication to this research has been outstanding, he deserves the accolades.
“Proving the principles behind gene therapy, and that it can be used safely in the retina, is fantastic news. But don’t forget, this trial was about one particular gene, we have treated 1 person. There are some 25,000 people – and their families – dealing with sight loss through RP in the UK alone. We desperately need more funding to be able to back those scientists, like Robin, that have proved they can do this. Only then can we really expect to be able to ‘break the chain’ of inherited blindness.”
