Report by: Martin Smedstad, Dep. Chairman, RP-association in Norway
Research on a growing number of different approaches to treat RP and other retinal degeneration is evolving at an exponential rate. Patients can with justified hope glimpse the light of treatments “at the end of the tunnel vision”. In my opinion, these people; dedicating their careers to develop the treatments that can potentially eradicate vision loss due to retinal degeneration in the future - they are my idols. My rockstars. And I hope they keep rocking ‘till the wheels fall off.
Keynote speech was given by Professor Robin Ali from University College in London on the topic of gene therapy in general and his trials on the RPE65-gene specifically. Later on in the program, his fellow countryman professor Robert Maclaren from University of Oxford shared experiences from his own trial on gene therapy involving Choroideremia and the CHM-gene.
President of Retina International, Christina Fasser from Switzerland, talked about the formation of a European Reference Network for Rare Eye Disease (ERN-EYE). She stressed the importance of lobbying for collaborative research on treatments for rare eye diseases in Europe.
.Associate professor Per Ekström fra University of Lund, Sweden has been involved in the DRUGSFORD project; a multilateral European effort seeking to understand the mechanisms behind apoptosis and develop compounds and delivery systems to prevent photoreceptor damage.
Norway was represented on the program twice by professor Cecilie Bredrup from Haukeland University Hospital and Josephine Prener Holtan MD, Ph.D from Oslo University Hospital Ullevål. Professor Bredrup is conducting Next Generation Sequencing DNA-analysis and has located several novel mutations in her work. Dr. Prener Holtan is responsible for developing and doing research on a registry currently containing genotype/phenotype data on 800 patients with 24 different retinal degenerative diseases.
Professor Morten C. Moe from Oslo University Hospital is head of the department of ophthalmology, in addition to being a member of the Norwegian Centre for Stem Cell Research, and gave a lecture on stem cell treatments.
Professor Henry Klassen from University of Irvine, USA is a stem cell expert currently conducting a clinical trial involving retinal progenitor cells as treatment for RP.
The Norwegian RP-association has twice earlier, in 2011 and 2013, hosted symposiums where researchers in the front-running of the development shared their experiences from cutting-edge research with national health practitioners and patients. Our intention has been inspired by our object clause; to raise awareness and knowledge level in national academic institutions about RP and RP-related research to benefit Norwegian patients in the future.
The success of our symposium, taking place on the 25th of November 2016, was the result of a collaboration between the RP-association and OUH Ullevål. This allowed us to schedule the event just after another retina specialist course taking place in Oslo. Since they already were in town, and since the Norwegian Medical Association (NMA) had approved the symposium as “advanced training” for ophthalmologists, we were happy to welcome 50 future retina specialists from all over Norway.
The national radio NRK conducted interviews for a long segment covering RP and gene therapy that aired in December. Also in the audience were patients and representatives from Norway and other Nordic RP-associations, a retina specialist from Sweden and a senior counselor from a national advisory panel currently relevant in changing legislation to allow gene therapy trials on non-lethal diseases in Norway. And all this came to fruition because of a big initiative from a small RP-association in Norway with help from some good friends.
The RP-association would like to thank the dep. Of ophthalmology at Oslo University Hospital Ullevål for their dedication and collaboration on this project. We would also like to acknowledge the financial contributions from Norwegian Association for the Blind and Partially Sighted (NABP), Blindemissionen IL, the national association for deafblindness (LSHDB) and the BBS-association in Norway (fBBS).