5TH EURETINAWINTER MEETING

Professor Jan van Meurs, of Rotterdam Eye Hospital and Erasmus University, The Netherlands, told delegates at the 5th EURETINA Winter Meeting that while anti-VEGF therapies may work well for a proportion of patients, there are specific groups for which anti-VEGF therapy does not work.

According to Prof van Meurs, stem cell therapeutics, while of significant promise, continue to have serious challenges including both complex surgery and the quality of “product” that can be produced in the lab.

The future for many stem cell therapies may involve the development of robust sheets of RPE derived from stem cells, however creating a reproducible and predictable quality control method for the production of such cells represents a significant engineering and biological challenge, he said.

He showed data of recent surgeries with autolous transplants, emphasising that transplantation of both autologous and stem cell derived RPE sheets in patients with exudative AMD will be accompanied by trauma and tissue reactions to trauma (the danger model of Polly Matzinger).

Prof James Bainbridge, of Moorfields Eye Hospital NHS Foundation Trust, London, UK, provided a brief update on the ongoing Octata Therapeutics (previously ACT Inc.) trial under way at centres in the EU. Prof Bainbridge provided details on the Phase I/II, open-label, multicentre, prospective study involving the subretinal transplantation of hESC-RPE cells in 12 subjects. The trial includes four cohorts of three Stargardt’s patients, each cohort receiving increasing doses of the RPE between 50,000 and 200,000 cells, together with immunosuppression medication withdrawn after 12 weeks.

To date, Prof Bainbridge confirmed that the procedure appeared to be well tolerated, with areas of new pigmentation in the fundi of recipients suggesting survival of transplanted cells.

While a number of speakers discussed successes and challenges with AAV gene therapy, alternative vectors for the delivery of therapeutic genes were presented by Dr Kyriacos Mitrophanous from Oxford Biomedica.

According to Dr Mitrophanous, lentiviruses overcome a significant limitation of AAV, namely the size restriction on what size gene may be delivered. Lentiviral vectors can incorporate 9-10kb of DNA and can therefore comfortably accommodate the 6.8kb transgene required to treat Stargardt’s disease. Clinical trials, sponsored by the company, are currently under way and formal results are expected by the end of 2015.

Dr Ursula Schmidt-Erfurth gave a presentation on computational image analysis, introducing the concept of utilising big data made possible from the thousands of patients seen at clinics. Computational analyses allow for a deeper understanding of disease mechanisms in all major indications.For the entire patient population seen over time, disease models allow to identify subgroups and disease patterns already at the initial presentation.

Jan van Meurs: j.vanmeurs@oogziekenhuis.nl

James Bainbridge: j.bainbridge@ucl.ac.uk

Kyriacos Mitrophanous: enquiries@oxfordbiomedica.co.uk

Ursula Schmidt-Erfurth: ursula.schmidt-erfurth@meduniwien.ac.at