An ambition to give sight to blind children

Mr Wade will take to the podium on May 22nd at St John’s Smith Square in London to conduct  Saint-Saëns’ Symphony No. 3: “Organ Symphony” at a private concert for 200 friends to mark his 60th birthday.

He ultimately hopes to raise £400,000 for the development of a gene therapy treatment for a form of Leber’s Congenital Amaurosis, known as LCA4, which is a type of congenital blindness.

The treatment will be developed at the National Institute for Health Research (NIHR) Biomedical Research Centre at Moorfields Eye Hospital and the UCL Institute of Ophthalmology. It will be led by Professor James Bainbridge, a consultant surgeon at Moorfields Eye Hospital and Professor of Retinal Studies at University College London, who is working alongside Professor Robin Ali, on a research programme to develop gene therapy for inherited eye disease.

In the last few years Mr Wade had detached retinas in both his eyes but thanks to the swift and excellent treatment he received at Moorfields Eye Hospital, his sight has been saved.  Following the success of his sight-saving operations, Mr Wade felt he wanted “to give something back to the world of eye care” and has worked with Professor Bainbridge, Moorfields Eye Charity and UCL Institute of Ophthalmology on his plans to celebrate his 60th birthday and raise significant funds for this important research.

Mr Wade has funded the concert and plans to invite friends and family to mark his debut as a conductor and help him fulfil a lifetime’s dream for his 60th birthday.

Professor Bainbridge said: “Mr Wade’s innovative fundraising appeal is a major boost for the next piece of our gene therapy programme to develop an effective treatment for LCA4 – a rare, but particularly severe, form of congenital blindness, that affects children who have not inherited the required complement of genes required for normal sight. 

Previous trials have shown that sight can be improved in children with a different form of LCA by injecting a modified virus – or 'vector' – into the retina to target delivery of the missing gene to the light-sensitive ‘photoreceptor’ cells.

Mr Wade’s fundraising will help us to manufacture a different vector specifically for LCA4.”

Mr Wade said: “I am delighted to be involved in this fundraising project to make this crucial research happen.  Discussions with Professor Bainbridge and Moorfields Eye Charity have identified that raising £400,000 should pay for the production and release of sufficient vector to treat up to 100 affected children. This includes the cost of obtaining a manufacturing license, vector manufacture itself and the costs associated with release for clinical use.”

Production and quality testing of the vector is expected to take about 18 months. The impact on children who receive the vector may be measurable within one or two years.

Effective gene therapy for conditions that affect photoreceptor cells will not only benefit the children involved in the trial but will expand significantly the range of sight disorders potentially amenable to gene therapy and pave the way for the treatment of other severe blinding disorders that affect both children and adults.

Please note that the concert is a private event and not open to the public.

 Editors Notes

  • Professor James Bainbridge, Professor Robin Ali and the Gene and Cell Therapy group at the UCL Institute of Ophthalmology have established a programme of research to develop gene and cell therapies for inherited eye disease with the aim of protecting children and adults against sight loss. Substantial funding has already been secured for this programme of experimental medicine from a wide range of sources, including government bodies, such as the National Institute for Health Research (NIHR), charities and industry. This funding supports the essential infrastructure including laboratory and clinical facilities and maintains the unique expertise of our multidisciplinary team of scientific, technical, clinical and administrative staff required to achieve our ambition. For more information go to www.ucl.ac.uk/ioo/genetics/gene-and-cell-therapy and www.brcophthalmology.org.
  • Moorfields is one of the world’s leading eye hospitals, providing expertise in clinical care, research and education. We have provided excellence in eye care for more than 200 years and we continue to be at the forefront of new breakthroughs and developments.  We are an integral part of one of the UK’s first academic health science centres, UCL Partners, and also one of the new academic health science networks. We were one of the first NHS organisations to achieve foundation trust status in 2004. 
  • We treat the entire range of eye diseases, from common complaints to rare conditions which require treatments not available anywhere else in the UK. We dealt with more than 528,824 attendances in 2012/13 at our main hospital base in London’s City Road and at 21 other sites in and around the capital, enabling us to provide expert care closer to patients’ homes.
  • With our research partners at the UCL Institute of Ophthalmology, we run one of the largest ophthalmic research programmes in the world and have the highest measure of scientific productivity and impact in the world for our research activity.

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