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- July 2013 -'Big leap' towards curing blindness in stem cell study
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Frequently-asked questions (FAQs)
What gene therapy trials are currently taking place?
We are currently conducting a clinical trial of gene therapy for Leber congenital amaurosis (LCA) caused by damage to the RPE65 gene - the first of its kind in the world. Recruitment for this trial has finished and early results suggest that the therapy is safe, and can restore vision in some cases (Bainbridge et al. NEJM 2011).
What stem cell trials are currently taking place?
We recently began Europe's first safety trial of the transplantation into the eye of retinal cells derived from stem cells. We are assessing the safety of this technique in patients with advanced visual impairment due to Stargardt disease.
- I have been diagnosed with RP, am I eligible for the trial?
Our current gene therapy clinical trial is for patients with Leber congenital amaurosis (LCA) caused by damage to the RPE65 gene. We hope to begin similar clinical trials for RP in the next two years, having established proof-of-concept that gene therapy is effective for some forms of RP.
I have been diagnosed with Leber’s Hereditary Optic Neuropathy (LHON), am I eligible for the trial?
While there are no trials currently planned for LHON, research is ongoing to develop treatments for this condition
- Contact us if you would like to be informed of future developments
- I have been diagnosed with Stargardts, am I eligible for the trial?
The current stem cell trial is to test the safety of cell transplantation in patients with advanced visual impairment caused by Stargardt disease; if you are otherwise healthy and would like more information on whether you are eligible for this trial, please contact Dr. Michel Michaelides
- I have Macular Degeneration, am I eligible for the trial?
If you have age-related macular degeneration (AMD), the commonest form of macular degeneration, please contact your GP or ophthalmologist to discuss whether you are eligible for any of the drug treatments currently available.
We are developing both gene and cell therapies that we hope will be effective for AMD in the future - please contact us if you would like to be informed of future developments.
If you have been diagnosed with the rare, early-onset Stargardt macular degeneration, you may be eligible for the current stem cell-derived cell transplantation trial - please contact Dr. Michel Michaelides
- I have retinal detachments, are there any suitable gene or cell therapies for me?
Retinal detachment can be managed through surgery in most cases. The experimental gene and cell therapies we are currently developing are not suitable for treating this cause of sight loss, but there may be similar treatments developed in the future that benefit those with retinal detachment.
- Can you put my details on a database in case I am suitable for a future trial?
Please contact us so we can add you to our growing database and can contact you regarding any future trials for which you may be eligible.
How long will it be until all patients with LCA can be treated using gene therapy?
The current gene therapy trial for LCA caused by mutations in the RPE65 gene will tell us how safe and effective this treatment is in this form of the disease; we are planning several trials to use similar technology to treat LCA caused by damage to other genes.
Because each form of the disease requires a different gene to be replaced, it will be a number of years before clinical trials are completed for all the known forms, and it is unlikely that gene therapy will prove suitable for all LCA patients.
Nonetheless progress is being made towards the treatment of the commonest forms of LCA and we hope to have safe and effective treatments available in the near future.
- How can I find out what gene mutation cause my condition?
We recommend that you are referred to Moorfields Eye Hospital, where a specialist may be able to arrange a blood test that identifies which gene mutation is causing your loss of vision. To be seen under the NHS, please ask for a referral from your General Practitioner. To arrange a private appointment, please refer to the private practice of either Prof. James Bainbridge or Dr. Michel Michaelides.
- When will trials begin on patients with other eye diseases?
There are several gene therapy trials planned as part of our pipeline of therapies for sight loss, for which proof of principle has been established in models of disease. Pending approval from the regulatory authorities, and the manufacture of clinical-grade gene therapy virus, we plan to begin treating the first patients in late 2013/early 2014.
- Is it possible to have an eye transplant?
It is not, although some parts of the eye such as the cornea (that forms the transparent window at the front of the eye) can be transplanted. We are developing cell transplants that would help repair the light-sensitive cells in the retina at the back of the eye, and have started an important clinical trial testing the safety of transplanting RPE cells (that support the light-sensitive photoreceptor cells) that are grown from embryonic stem cells. More research is required before such treatments can be tested more widely in the clinic.
Are there any medicines, vitamins or treatments you can recommend to prevent any further deterioration in my sight?
There are some medicines that can slow the rate of vision loss in age-related macular degeneration and diabetic retinopathy, please contact your GP or ophthalmologist to see whether you may benefit from them.
There is some evidence. although it is not very strong, that specific combinations of vitamin supplements can slow the rate of vision loss in some patients who at high risk of developing, or already have, age-related macular degeneration. There are side effects caused by taking high levels of antioxidants and zinc supplements that must also be considered.
There is no evidence that taking supplements can prevent vision loss in any other conditions, and we do not recommend that you take any supplements before contacting your GP or ophthalmologist.
There is also no evidence that alternative therapies, untested stem cell treatments or other 'miracle cures' that you may come across are effective - in fact they may well be dangerous.
There is, however, strong evidence that stopping smoking and preventing obesity and high blood pressure through changing you diet and exercising more can reduce your chances of severe vision loss.
- Will gene or stem cell therapy ever be suitable for people who are colour blind?
The commonest form of colour-blindness is X-linked red-green colour blindness, and there are no plans to tackle this using gene or stem cell therapy. We have developed a gene therapy for an inherited disease called achromatopsia, in which patients lose colour vision as well as their central detailed vision because their cone photoreceptor cells stop functioning properly. We hope to commence clinical trials to test for this condition within the next two years.
- If I am not eligible for the gene therapy trial, what about stem cell therapies?
If you have advanced vision loss from either an inherited condition or a complex disease like AMD, gene therapy is unlikely to be effective once the light-sensitive rod and cone photoreceptor cells are lost. Stem cell therapies may be effective in this case, although the first trials of stem cell-derived transplants are only designed to assess safety and are not expected to result in benefit to patients' vision. To find out if you may be eligible for the current stem cell trial involving Stargardt disease, or for future studies, contact us.
Would the stem cell injections being offered privately be of any benefit to me?
It is vital that you only choose treatments and trials that are safe, scientifically plausible and carried out with proper oversight.
Many stem cell treatments being offered are potentially dangerous and extremely unlikely to provide benefit.
We urge you to read more on how to avoid bogus treatments, and speak to your GP or contact us if you are considering private therapy of any kind.
- What is the difference between gene therapy and stem cell therapy?
Gene therapy is where we deliver a working copy of a damaged gene, using a modified virus as a vehicle or vector, to cells in the eye. This allows cells that aren't functioning well to recover and to prevent loss of sight. In most cases different forms of blindness need to be treated with a different vector carrying the specific gene we want to deliver. Gene therapy is also being developed as a way of silencing mutant genes that cause dominant disease, and to introduce new genes that may help prevent the loss of cells or the abnormal growth of blood vessels.Stem cell therapy is where we replace lost cells in the advanced stages of disease. The transplanted cells originate from stem cells, and are grown into either light-sensitive photoreceptor cells or retinal pigment epithelium (RPE) cells. Further laboratory work is required before photoreceptor transplantation can be applied in the clinic, but we have begun Europe's first safety trial involving the transplantation of stem cell-derived RPE cells for Stargardt macular dystrophy.
- How can I request an appointment with Prof. Ali?
Professor Ali heads the research group and does not routinely meet with
patients. If you are interested in seeing one of our clinical specialists,
please either ask your General Practitioner for an NHS referral or contact the
private practice of either Prof. James Bainbridge or Dr. Michel Michaelides
- How can I request an appointment with a specialist at Moorfields Eye Hospital?
Please ask your General Practitioner for a referral or contact the private practice of either Prof. James Bainbridge or Dr. Michel Michaelides
When will gene therapy be available as treatment in hospitals?
Each gene therapy treatment must be thoroughly assessed in rigorous clinical trials to assess safety and efficacy. If these trials are successful, treatments can be made available through the NHS - this can take up to ten years, although we are hopeful the gene therapy for some eye conditions may be available sooner.
- Can you email me with details of any updates on the RPE65 gene therapy clinical trial?
Whilst we are unable to provide individual updates, we do have a quarterly newsletter that will keep you up-to-date with developments in all our clinical and laboratory studies. Sign up by contacting us.
- Are you conducting any other clinical trials for eye disease at the moment?Apart from the world's first clinical trial for gene therapy in the eye, and Europe's first safety trial of stem cells in eye disease, we are planning several other clinical trials as part of a pipeline of therapies for diseases that cause blindness.
- Should we consider storing stem cells from our new child’s umbilical cord?
Whilst we cannot be confident that such stem cells would be useful in the future to treat eye disease, we cannot advise on such matters and recommend seeking the opinion of your obstetrician.
Page last modified on 06 feb 13 13:56