It can take many years and millions of pounds to develop a new treatment from start to finish. Even after laboratory studies and tests in animal models are complete, most new treatments have to go through a series of clinical trials to prove that they are acceptably safe and offer meaningful benefit. Each one of these trials can take a few years by the time participants are recruited, treated and followed up and all the data is analysed.
However, there is an increase in momentum in the development of treatments for inherited sight loss. Our website has more information about the treatment approaches being explored. Successful therapy may eventually depend on a combination of these.
Genetic testing may help identify the gene that is causing your sight loss. Some future treatments for retinal conditions may be specific to certain genes, and some clinical trials require participants to have a particular faulty gene.
Many new genes associated with sight loss have been discovered in recent years and it may be possible for your gene to be identified now, even if this was not possible when you were diagnosed.
You can read more about the issues surrounding genetic testing and how you can access it on the Unlock Genetics site: www.RetinaUK.org.uk/genetics.
Clinical trials are experiments that confirm the safety of potential new treatments and test their effectiveness in slowing sight loss. As with any treatment, there will be associated risks and there is a possibility that the trial will reveal unpleasant side effects from the treatment.
The risks of any particular trial will be fully explained to you by the researchers and you should feel able to ask as many questions as you want. Asking for preliminary information about the trial or going to an initial appointment does not mean you are committing to taking part and you are free to withdraw your child from the trial at any time. This will not in any way affect their normal care.
You may come across clinic websites claiming to offer treatments that will slow or stop the progress of sight loss. These claims are often not supported by reliable scientific evidence and the treatments offered have not gone through thorough testing for safety and effectiveness.
To become proven, a new treatment must undergo a rigorous clinical trials process. The process not only tests whether a treatment works, it also ensures that the treatment is safe. A treatment that has not undergone proper clinical trials may have unacceptably serious side effects and at worst may even speed up the progression of sight loss.
Some clinics may claim to have performed clinical trials. We would encourage anyone who is considering an unproven treatment to discuss all the implications with their ophthalmologist before making a decision, as he or she will also be able to tell the difference between a reliable clinical trial and a poor one. You might also like to download the leaflet “I’ve got nothing to lose by trying it” from the organisation Sense About Science.
Our website also has more information about stem cell research in inherited sight loss.