Search Underway for Treatment of Night Blindness

Treatment Being Sought for Night Blindness Sabancı University, Faculty of Engineering and Natural Sciences faculty member Cavit Ağca is conducting research on the "Conversion of cGMP Analogues for Retinitis Pigmentosa (RP) Treatment" project to enable drug treatment for patients experiencing blindness due to night blindness disease. Other project coordinators running the consortium project within the scope of the European Union Rare Diseases Project Work include Valeria Marigo (principal investigator) from UNIMORE University in Italy, Vittoria Murro from Careggi University Hospital AOUC, Nicolaas Schipper from Swedish Research Institutes RISE, Francois Paquet-Durand from Mireca Medicines in Germany, Heiko von der Leyen from Hannover Clinical Research Center HCTC, and Arto Urtti from University of Eastern Finland. The initial goal in the project is completion of preliminary clinical studies. The studies in question consist of processes including formulation development, pharmacokinetic and toxicity tests, completion of clinical design, modelling, clinical expectations, patient identification and dosage regimens. Parallel to completion of this phase of the project, commencement of clinical trials is planned. Sabancı University, Faculty of Engineering and Natural Sciences faculty member Cavit Ağca, who reported that they have begun animal testing in their project aimed at stopping or slowing blindness caused by Retinitis Pigmentosa (RP) found in the retina or, as commonly used, night blindness or chicken blindness disease, stated the following: "With the transition to animal testing in clinical trials, we have reached an important stage in terms of converting the project into a product. Our goal is to begin phase one studies at the end of 3 years and subsequently begin treatment in patients within the scope of clinical phases in the following years; and after completing clinical trials, to use it as a general drug treatment against RP."   Patients from Turkey Will Also Be Included in Research Ağca, noting that they plan to make arrangements for participation of patients from Turkey in the selection of patients to participate in clinical research, continued as follows: "Retinitis pigmentosa is an inherited eye disease and over time causes retinal damage and severe vision loss. In the retina are photoreceptor cells that detect light and produce vision signals. In early stages of RP, rod photoreceptors are more severely affected than cone photoreceptors. As the rods die, people experience night blindness and progressive loss of visual field. In late stages of RP, people begin to lose more of their visual field and develop tunnel vision. This causes them to struggle with basic tasks of daily life, such as reading, driving, walking without assistance or recognizing objects with faces."

Disease Prevalence

Noting that RP affects 1 out of every 3,000 to 4,000 people in the world, Ağca stated, "In the world population estimated to be over 7.7 billion in 2020, approximately 1.9 to 2.5 million people are estimated to have RP disorder. This rate means that in Turkey with a population of approximately 83 million, 20,000 to 27,000 people have RP. In recent years, treatments such as neuroprotective gene therapy, optogenetics, chip technology and photoreceptor transplantation, which have been intensively studied in our laboratory for the disease, are still in development stages. Unfortunately, currently there is no treatment that can cure this disease or halt its progression. Instead, devices and methods that make vision more efficient are used, namely special glasses and magnifiers for more effective use of remaining vision." he said.

Easy Diagnostic Methods an Important Aspect of Research

Drawing attention to the fact that more than 90 different gene mutations in RP can be caused by, and the importance of targeting common processes so that patients with different mutations can benefit from the same treatment, Ağca stated the following: "The purpose of this project is actually the development of a drug and treatment protocol for different forms of RP. In this context, a liposomal formulation has been developed so far within the scope of the project, a treatment method that prevents cell photoreceptor death. This formulation has been successful in 3 different animal mutations causing RP so far. We have begun studies at Sabancı University for the 4th mutation. Another purpose of the project is to find markers for easy diagnosis and, using these markers, to quickly interpret patients' responses to treatment during treatment. The project is currently at a very advanced level and is promising."