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Publication Date: 22 September 2011
Stem cell trials for Stargardt disease (inherited juvenile macular degeneration) – information for patients
Background
- Advanced Cell Technology (ACT), the company that has developed a potential treatment for Stargardt disease, announced approval on Thursday 22 September by the Medicines and Healthcare Products Regulatory Agency (MHRA) to start clinical trials in the UK
- The clinical trials will be led by Professor James Bainbridge, one of Moorfields’ retinal surgeons, at the NIHR biomedical research centre for ophthalmology, based at Moorfields and the UCL Institute of Ophthalmology
- The trials are due to start in the next few months
Key points
- The trial aims to test the safety of a potential treatment for Stargardt disease, a form of macular degeneration that causes disabling loss of sight in young people
- The trial is designed to test whether transplanting retinal cells into people with Stargardt disease is safe
- There is currently no treatment for Stargardt disease, either privately or
on the NHS
Conditions that can be treated using retinal stem
cell therapy
- This trial is specifically related to Stargardt disease, a form of macular degeneration that causes disabling loss of sight in young people
- It is possible that it could be adapted in future for other blinding
disorders of the retina, including age-related macular degeneration (AMD)
Criteria for suitability to participate in the
trial
- Participants in the trial need to be adults with Stargardt disease and severe sight impairment, but who are otherwise healthy
Further information
- Clinical trial queries: please email mol.therapy@ucl.ac.uk
- Journalists and broadcasters: please contact the press office on 020 7566 2628
- General information: please email info@moorfields.nhs.uk
